| [38] | N. Telailia, S. Fisson, and H. Frih. Compression of The Sciatic Nerve May not Contribute to Ipsilateral Hyperalgesia Development in Ovariectomized Female Rats! Cell J, 22(4):548-555, Jan 2021. [PubMed Central:PMC7211275] [DOI:10.22074/cellj.2021.6907] [PubMed:19041344]. |
| [37] | L. Buscara, D. A. Gross, and N. Daniele. Of rAAV and Men: From Genetic Neuromuscular Disorder Efficacy and Toxicity Preclinical Studies to Clinical Trials and Back. J Pers Med, 10(4), Nov 2020. [DOI:10.3390/jpm10040258] [PubMed:33260623]. |
| [36] | H. Jmii, S. Fisson, M. Aouni, and H. Jaidane. Type B coxsackieviruses and central nervous system disorders: critical review of reported associations. Rev Med Virol, page e2191, Nov 2020. [DOI:10.1002/rmv.2191] [PubMed:33159700]. |
| [35] | Z. Yablonka-Reuveni, F. Stockdale, U. Nudel, D. Israeli, H. M. Blau, A. Shainberg, S. Neuman, G. Kessler-Icekson, E. M. Krull, B. Paterson, O. S. Fuchs, D. Greenberg, R. Sarig, O. Halevy, E. Ozawa, and D. J. Katcoff. Farewell to Professor David Yaffe - A pillar of the myogenesis field. Eur J Transl Myol, 30(3):9306, Sep 2020. [PubMed Central:PMC7582454] [DOI:10.4081/ejtm.2020.9306] [PubMed:1701405]. |
| [34] | P. Mu?oz, M. Trist?n-Manzano, A. S?nchez-Gilabert, G. Santilli, A. Galy, A. J. Thrasher, and F. Martin. WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation. Mol Ther Methods Clin Dev, 19:220-235, Dec 2020. [PubMed Central:PMC7558809] [DOI:10.1016/j.omtm.2020.09.006] [PubMed:30332548]. |
| [33] | R. Guimar?es-Costa, G. Fern?ndez-Eulate, K. Wahbi, F. Leturcq, E. Malfatti, A. Behin, S. Leonard-Louis, I. Desguerre, C. Barnerias, M. C. Nougues, A. Isapof, B. Estournet-Mathiaud, S. Quijano-Roy, A. Fayssoil, D. Orlikowski, B. Fauroux, I. Richard, C. Semplicini, N. B. Romero, G. Querin, B. Eymard, P. Lafor?t, and T. Stojkovic. Clinical correlations and long-term follow-up in 100 patients with sarcoglycanopathies. Eur J Neurol, Oct 2020. [DOI:10.1111/ene.14592] [PubMed:33051934]. |
| [32] | P. Colella, P. Sellier, M. J. Gomez, M. G. Biferi, G. Tanniou, N. Guerchet, M. Cohen-Tannoudji, M. Moya-Nilges, L. van Wittenberghe, N. Daniele, B. Gjata, J. Krijnse-Locker, F. Collaud, M. Simon-Sola, S. Charles, U. Cagin, and F. Mingozzi. Gene therapy with secreted acid alpha-glucosidase rescues Pompe disease in a novel mouse model with early-onset spinal cord and respiratory defects. EBioMedicine, 61:103052, Nov 2020. [PubMed Central:PMC7553357] [DOI:10.1016/j.ebiom.2020.103052] [PubMed:33039711]. |
| [31] | C. Vaubourg, E. Gicquel, I. Richard, W. Lostal, and J. Bellec. Minimal Consequences of CMAH and DBA/2J Background on a FKRP Deficient Model. J Neuromuscul Dis, Aug 2020. [DOI:10.3233/JND-200487] [PubMed:32925084]. |
| [30] | L. Weber, G. Frati, T. Felix, G. Hardouin, A. Casini, C. Wollenschlaeger, V. Meneghini, C. Masson, A. De Cian, A. Chalumeau, F. Mavilio, M. Amendola, I. Andre-Schmutz, A. Cereseto, W. El Nemer, J. P. Concordet, C. Giovannangeli, M. Cavazzana, and A. Miccio. Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype. Sci Adv, 6(7), Feb 2020. [PubMed Central:PMC7015694] [DOI:10.1126/sciadv.aay9392] [PubMed:30287096]. |
| [29] | J. Alonso-P?rez, L. Gonz?lez-Quereda, L. Bello, M. Guglieri, V. Straub, P. Gallano, C. Semplicini, E. Pegoraro, V. Zangaro, A. Nascimento, C. Ortez, G. P. Comi, L. T. Dam, M. De Visser, A. J. van der Kooi, C. Garrido, M. Santos, U. Schara, A. Gangfu?, N. L?kken, J. H. Storgaard, J. Vissing, B. Schoser, G. Dekomien, B. Udd, J. Palmio, A. D'Amico, L. Politano, V. Nigro, C. Bruno, C. Panicucci, A. Sarkozy, O. Abdel-Mannan, A. Alonso-Jimenez, K. G. Claeys, D. Gomez-Andr?s, F. Munell, L. Costa-Comellas, J. Haberlov?, M. Rohlenov?, V. Elke, J. L. De Bleecker, C. Dominguez-Gonz?lez, G. Tasca, C. Weiss, N. Deconinck, R. Fern?ndez-Torr?n, A. L?pez de Munain, A. Camacho-Salas, B. Melegh, K. Hadzsiev, L. Leonardis, B. Koritnik, M. Garibaldi, J. C. de Leon-Hern?ndez, E. Malfatti, A. Fraga-Bau, I. Richard, I. Illa, and J. D?az-Manera. New genotype-phenotype correlations in a large European cohort of patients with sarcoglycanopathy. Brain, 143(9):2696-2708, Sep 2020. [DOI:10.1093/brain/awaa228] [PubMed:32875335]. |
| [28] | S. Cavallero, R. Neves Granito, D. Stockholm, P. Azzolin, M. T. Martin, and N. O. Fortunel. Exposure of Human Skin Organoids to Low Genotoxic Stress Can Promote Epithelial-to-Mesenchymal Transition in Regenerating Keratinocyte Precursor Cells. Cells, 9(8), 08 2020. [PubMed Central:PMC7466070] [DOI:10.3390/cells9081912] [PubMed:29131160]. |
| [27] | G. Pavani, M. Laurent, A. Fabiano, E. Cantelli, A. Sakkal, G. Corre, P. J. Lenting, J. P. Concordet, M. Toueille, A. Miccio, and M. Amendola. Author Correction: Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins. Nat Commun, 11(1):4146, 08 2020. [PubMed Central:PMC7426957] [DOI:10.1038/s41467-020-18036-0] [PubMed:32792546]. |
| [26] | G. Pavani, M. Laurent, A. Fabiano, E. Cantelli, A. Sakkal, G. Corre, P. J. Lenting, J. P. Concordet, M. Toueille, A. Miccio, and M. Amendola. Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins. Nat Commun, 11(1):3778, 07 2020. [PubMed Central:PMC7391635] [DOI:10.1038/s41467-020-17552-3] [PubMed:23992846]. |
| [25] | V. Mariot, C. Le Guiner, I. Barth?l?my, M. Montus, S. Blot, S. Torelli, J. Morgan, F. Muntoni, T. Voit, and J. Dumonceaux. Myostatin Is a Quantifiable Biomarker for Monitoring Pharmaco-gene Therapy in Duchenne Muscular Dystrophy. Mol Ther Methods Clin Dev, 18:415-421, Sep 2020. [PubMed Central:PMC7363622] [DOI:10.1016/j.omtm.2020.06.016] [PubMed:28796573]. |
| [24] | S. J. Aronson, R. S. Bakker, S. Moenis, R. van Dijk, G. Bortolussi, F. Collaud, X. Shi, S. Duijst, L. Ten Bloemendaal, G. Ronzitti, A. F. Muro, F. Mingozzi, U. Beuers, and P. J. Bosma. A Quantitative In Vitro Potency Assay for Adeno-Associated Virus Vectors Encoding for the UGT1A1 Transgene. Mol Ther Methods Clin Dev, 18:250-258, Sep 2020. [PubMed Central:PMC7327880] [DOI:10.1016/j.omtm.2020.06.002] [PubMed:14529832]. |
| [23] | M. V. Petkova, A. Stantzou, A. Morin, O. Petrova, S. Morales-Gonzalez, F. Seifert, J. Bellec-Dyevre, T. Manoliu, A. Goyenvalle, L. Garcia, I. Richard, C. Laplace-Builh?, M. Schuelke, and H. Amthor. Live-imaging of revertant and therapeutically restored dystrophin in the DmdEGFP-mdx mouse model for Duchenne muscular dystrophy. Neuropathol Appl Neurobiol, 46(6):602-614, Oct 2020. [DOI:10.1111/nan.12639] [PubMed:32573804]. |
| [22] | J. Vissing, J. R. Dahlqvist, C. Roudaut, J. Poupiot, I. Richard, M. Duno, and T. Krag. A single c.1715G>C calpain 3 gene variant causes dominant calpainopathy with loss of calpain 3 expression and activity. Hum Mutat, 41(9):1507-1513, Sep 2020. [DOI:10.1002/humu.24066] [PubMed:32557990]. |
| [21] | U. Cagin, F. Puzzo, M. J. Gomez, M. Moya-Nilges, P. Sellier, C. Abad, L. Van Wittenberghe, N. Daniele, N. Guerchet, B. Gjata, F. Collaud, S. Charles, M. S. Sola, O. Boyer, J. Krijnse-Locker, G. Ronzitti, P. Colella, and F. Mingozzi. Rescue of Advanced Pompe Disease in Mice with Hepatic Expression of Secretable Acid α-Glucosidase. Mol Ther, 28(9):2056-2072, 09 2020. [PubMed Central:PMC7474269] [DOI:10.1016/j.ymthe.2020.05.025] [PubMed:16860134]. |
| [20] | M. Sanson, A. Vu Hong, E. Massourides, N. Bourg, L. Suel, F. Amor, G. Corre, P. B?nit, I. Barth?l?my, S. Blot, A. Bigot, C. Pinset, P. Rustin, L. Servais, T. Voit, I. Richard, and D. Israeli. miR-379 links glucocorticoid treatment with mitochondrial response in Duchenne muscular dystrophy. Sci Rep, 10(1):9139, 06 2020. [PubMed Central:PMC7272451] [DOI:10.1038/s41598-020-66016-7] [PubMed:32499563]. |
| [19] | C. Leborgne, E. Barbon, J. M. Alexander, H. Hanby, S. Delignat, D. M. Cohen, F. Collaud, S. Muraleetharan, D. Lupo, J. Silverberg, K. Huang, L. van Wittengerghe, B. Marolleau, A. Miranda, A. Fabiano, V. Daventure, H. Beck, X. M. Anguela, G. Ronzitti, S. M. Armour, S. Lacroix-Desmazes, and F. Mingozzi. IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies. Nat Med, 26(7):1096-1101, 07 2020. [DOI:10.1038/s41591-020-0911-7] [PubMed:32483358]. |
| [18] | G. Ronzitti, D. A. Gross, and F. Mingozzi. Human Immune Responses to Adeno-Associated Virus (AAV) Vectors. Front Immunol, 11:670, 2020. [PubMed Central:PMC7181373] [DOI:10.3389/fimmu.2020.00670] [PubMed:28284982]. |
| [17] | H. Jmii, A. Halouani, M. Maatouk, L. Chekir-Ghedira, M. Aouni, S. Fisson, and H. Ja?dane. Coxsackievirus B4 infection and interneuronal spread in primary cultured neurons. Microb Pathog, 145:104235, Aug 2020. [DOI:10.1016/j.micpath.2020.104235] [PubMed:32360191]. |
| [16] | L. Caputo, A. Granados, J. Lenzi, A. Rosa, S. Ait-Si-Ali, P. L. Puri, and S. Albini. Acute conversion of patient-derived Duchenne muscular dystrophy iPSC into myotubes reveals constitutive and inducible over-activation of TGFβ-dependent pro-fibrotic signaling. Skelet Muscle, 10(1):13, 05 2020. [PubMed Central:PMC7195779] [DOI:10.1186/s13395-020-00224-7] [PubMed:32359374]. |
| [15] | A. Perrin, C. Metay, M. Villanova, R. Y. Carlier, E. Pegoraro, R. Juntas Morales, T. Stojkovic, I. Richard, P. Richard, N. B. Romero, H. Granzier, M. Koenig, E. Malfatti, and M. Coss?e. A new congenital multicore titinopathy associated with fast myosin heavy chain deficiency. Ann Clin Transl Neurol, 7(5):846-854, 05 2020. [PubMed Central:PMC7261750] [DOI:10.1002/acn3.51031] [PubMed:30471092]. |
| [14] | O. Romano, L. Petiti, T. Felix, V. Meneghini, M. Portafax, C. Antoniani, M. Amendola, S. Bicciato, C. Peano, and A. Miccio. GATA Factor-Mediated Gene Regulation in Human Erythropoiesis. iScience, 23(4):101018, Apr 2020. [PubMed Central:PMC7155206] [DOI:10.1016/j.isci.2020.101018] [PubMed:32283524]. |
| [13] | C. B. Breuer, K. S. Hanlon, J. S. Natasan, A. Volak, A. Meliani, F. Mingozzi, B. P. Kleinstiver, J. J. Moon, and C. A. Maguire. In vivo engineering of lymphocytes after systemic exosome-associated AAV delivery. Sci Rep, 10(1):4544, 03 2020. [PubMed Central:PMC7066196] [DOI:10.1038/s41598-020-61518-w] [PubMed:29121014]. |
| [12] | E. Barbon, G. Ayme, A. Mohamadi, J. F. Ottavi, C. Kawecki, C. Casari, S. Verhenne, S. Marmier, L. van Wittenberghe, S. Charles, F. Collaud, C. V. Denis, O. D. Christophe, F. Mingozzi, and P. J. Lenting. Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors. EMBO Mol Med, 12(4):e11298, 04 2020. [PubMed Central:PMC7136963] [DOI:10.15252/emmm.201911298] [PubMed:28632502]. |
| [11] | M. Carotti, M. Scano, I. Fancello, I. Richard, G. Risato, M. Bensalah, M. Soardi, and D. Sandon? Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery. Int J Mol Sci, 21(5), Mar 2020. [PubMed Central:PMC7084537] [DOI:10.3390/ijms21051813] [PubMed:11812794]. |
| [10] | E. Six, A. Guilloux, A. Denis, A. Lecoules, A. Magnani, R. Vilette, F. Male, N. Cagnard, M. Delville, E. Magrin, L. Caccavelli, C. Roudaut, C. Plantier, S. Sobrino, J. Gregg, C. L. Nobles, J. K. Everett, S. Hacein-Bey-Abina, A. Galy, A. Fischer, A. J. Thrasher, I. Andr?, M. Cavazzana, and F. D. Bushman. Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs. Blood, 135(15):1219-1231, 04 2020. [PubMed Central:PMC7146019] [DOI:10.1182/blood.2019002350] [PubMed:20410504]. |
| [9] | D. B. Kohn, C. Booth, E. M. Kang, S. Y. Pai, K. L. Shaw, G. Santilli, M. Armant, K. F. Buckland, U. Choi, S. S. De Ravin, M. J. Dorsey, C. Y. Kuo, D. Leon-Rico, C. Rivat, N. Izotova, K. Gilmour, K. Snell, J. X. Dip, J. Darwish, E. C. Morris, D. Terrazas, L. D. Wang, C. A. Bauser, T. Paprotka, D. B. Kuhns, J. Gregg, H. E. Raymond, J. K. Everett, G. Honnet, L. Biasco, P. E. Newburger, F. D. Bushman, M. Grez, H. B. Gaspar, D. A. Williams, H. L. Malech, A. Galy, A. J. Thrasher, K. F. Buckland, C. A. Bauser, G. Honnet, M. Grez, H. B. Gaspar, A. Galy, and A. J. Thrasher. Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med, 26(2):200-206, 02 2020. [PubMed Central:PMC7115833] [DOI:10.1038/s41591-019-0735-5] [PubMed:24161820]. |
| [8] | H. C. Verdera, K. Kuranda, and F. Mingozzi. AAV Vector Immunogenicity in Humans: A Long Journey to Successful Gene Transfer. Mol Ther, 28(3):723-746, 03 2020. [PubMed Central:PMC7054726] [DOI:10.1016/j.ymthe.2019.12.010] [PubMed:31972133]. |
| [7] | B. Bertin, P. Veron, C. Leborgne, J. Y. Deschamps, S. Moullec, Y. Fromes, F. Collaud, S. Boutin, V. Latournerie, L. van Wittenberghe, B. Delache, R. Le Grand, N. Dereuddre-Bosquet, O. Benveniste, P. Moullier, C. Masurier, O. Merten, and F. Mingozzi. Capsid-specific removal of circulating antibodies to adeno-associated virus vectors. Sci Rep, 10(1):864, 01 2020. [PubMed Central:PMC6972890] [DOI:10.1038/s41598-020-57893-z] [PubMed:28078733]. |
| [6] | H. Jmii, A. Halouani, M. Abdeli, M. Aouni, S. Fisson, and H. Ja?dane. Immunopathology in the brain of mice following vertical transmission of Coxsackievirus B4. Microb Pathog, 140:103965, Mar 2020. [DOI:10.1016/j.micpath.2020.103965] [PubMed:31904449]. |
| [5] | A. Marquette, C. Leborgne, V. Schartner, E. Salnikov, B. Bechinger, and A. Kichler. Peptides derived from the C-terminal domain of HIV-1 Viral Protein R in lipid bilayers: Structure, membrane positioning and gene delivery. Biochim Biophys Acta Biomembr, 1862(2):183149, 02 2020. [DOI:10.1016/j.bbamem.2019.183149] [PubMed:31816324]. |
| [4] | J. B. Dupont, J. Guo, E. Renaud-Gabardos, K. Poulard, V. Latournerie, M. W. Lawlor, R. W. Grange, J. T. Gray, A. Buj-Bello, M. K. Childers, and D. L. Mack. AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy. Mol Ther, 28(2):382-393, 02 2020. [PubMed Central:PMC7000997] [DOI:10.1016/j.ymthe.2019.10.018] [PubMed:28361972]. |
| [3] | S. Espinoza, M. Scarpato, D. Damiani, F. Manag?, M. Mereu, A. Contestabile, O. Peruzzo, P. Carninci, C. Santoro, F. Papaleo, F. Mingozzi, G. Ronzitti, S. Zucchelli, and S. Gustincich. SINEUP Non-coding RNA Targeting GDNF Rescues Motor Deficits and Neurodegeneration in a Mouse Model of Parkinson's Disease. Mol Ther, 28(2):642-652, 02 2020. [PubMed Central:PMC7000958] [DOI:10.1016/j.ymthe.2019.08.005] [PubMed:10877911]. |
| [2] | I. Evnouchidou, P. Chappert, S. Benadda, A. Zucchetti, M. Weimershaus, M. Bens, V. Caillens, D. Koumantou, S. Lotersztajn, P. van Endert, J. Davoust, P. Guermonprez, C. Hivroz, D. A. Gross, and L. Saveanu. IRAP-dependent endosomal T cell receptor signalling is essential for T cell responses. Nat Commun, 11(1):2779, 06 2020. [PubMed Central:PMC7265453] [DOI:10.1038/s41467-020-16471-7] [PubMed:32487999]. |
| [1] | H. Taheri, M. A. Unal, M. Sevim, C. Gurcan, O. Ekim, A. Ceylan, Z. Syrgiannis, K. C. Christoforidis, S. Bosi, O. Ozgen?, M. J. G?mez, M. Turktas Erken, ?. Soydal, Z. Ero?lu, C. V. Bitirim, U. Cagin, F. Ar?, A. Ozen, O. Ku?uk, L. G. Delogu, M. Prato, ?. Metin, and A. Yilmazer. Photocatalytically Active Graphitic Carbon Nitride as an Effective and Safe 2D Material for In Vitro and In Vivo Photodynamic Therapy. Small, 16(10):e1904619, Mar 2020. [DOI:10.1002/smll.201904619] [PubMed:31971659]. |
| [36] | L. Bartolo, S. Li Chung Tong, P. Chappert, D. Urbain, F. Collaud, P. Colella, I. Richard, G. Ronzitti, J. Demengeot, D. A. Gross, F. Mingozzi, and J. Davoust. Dual muscle-liver transduction imposes immune tolerance for muscle transgene engraftment despite preexisting immunity. JCI Insight, 4(11), Jun 2019. [PubMed Central:PMC6629094] [DOI:10.1172/jci.insight.127008] [PubMed:9311887]. |
| [35] | S. J. Aronson, P. Veron, F. Collaud, A. Hubert, V. Delahais, G. Honnet, R. J. de Knegt, N. Junge, U. Baumann, A. Di Giorgio, L. D'Antiga, V. M. Ginocchio, N. Brunetti-Pierri, P. Labrune, U. Beuers, P. J. Bosma, and F. Mingozzi. Prevalence and Relevance of Pre-Existing Anti-Adeno-Associated Virus Immunity in the Context of Gene Therapy for Crigler-Najjar Syndrome. Hum. Gene Ther., 30(10):1297-1305, 10 2019. [PubMed Central:PMC6763963] [DOI:10.1089/hum.2019.143] [PubMed:25072305]. |
| [34] | M. Annoussamy, C. Lilien, T. Gidaro, E. Gargaun, V. Ch?, U. Schara, A. Gangfu?, A. D'Amico, J. J. Dowling, B. T. Darras, A. Daron, A. Hernandez, C. de Lattre, J. M. Arnal, M. Mayer, J. M. Cuisset, C. Vuillerot, S. Fontaine, R. Bellance, V. Biancalana, A. Buj-Bello, J. Y. Hogrel, H. Landy, and L. Servais. X-linked myotubular myopathy: A prospective international natural history study. Neurology, 92(16):e1852-e1867, 04 2019. [PubMed Central:PMC6550499] [DOI:10.1212/WNL.0000000000007319] [PubMed:10900271]. |
| [33] | William Lostal and Isabelle Richard. Therapeutic Approaches for Dysferlinopathy in Animal Models. In Muscle Gene Therapy, pages 439-451. Springer International Publishing, Cham, 2019. [ DOI | http ] |
| [32] | S. Pignani, F. Zappaterra, E. Barbon, A. Follenzi, M. Bovolenta, F. Bernardi, A. Branchini, and M. Pinotti. Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts. Biochim Biophys Acta Gene Regul Mech, 1862(6):619-624, 06 2019. [DOI:10.1016/j.bbagrm.2019.04.002] [PubMed:31005673]. |
| [31] | A. Moussy, N. Papili Gao, G. Corre, V. Poletti, S. Majdoul, D. Fenard, R. Gunawan, D. Stockholm, and A. P?ldi. Constraints on Human CD34+ Cell Fate due to Lentiviral Vectors Can Be Relieved by Valproic Acid. Hum. Gene Ther., 30(8):1023-1034, 08 2019. [DOI:10.1089/hum.2019.009] [PubMed:30977420]. |
| [30] | S. J. Aronson, R. S. Bakker, X. Shi, S. Duijst, L. Ten Bloemendaal, D. R. de Waart, J. Verheij, G. Ronzitti, R. P. Oude Elferink, U. Beuers, C. C. Paulusma, and P. J. Bosma. Liver-directed gene therapy results in long-term correction of progressive familial intrahepatic cholestasis type 3 in mice. J. Hepatol., 71(1):153-162, Jul 2019. [DOI:10.1016/j.jhep.2019.03.021] [PubMed:30935993]. |
| [29] | D. A. Gross, C. Leborgne, P. Chappert, C. Masurier, M. Leboeuf, V. Monteilhet, S. Boutin, F. A. Lemonnier, J. Davoust, and A. Kichler. Induction of tumor-specific CTL responses using the C-terminal fragment of Viral protein R as cell penetrating peptide. Sci Rep, 9(1):3937, 03 2019. [PubMed Central:PMC6408526] [DOI:10.1038/s41598-019-40594-7] [PubMed:21704113]. |
| [28] | D. Boudeffa, B. Bertin, A. Biek, M. Mormin, F. Leseigneur, A. Galy, and O. W. Merten. Toward a Scalable Purification Protocol of GaLV-TR-Pseudotyped Lentiviral Vectors. Hum Gene Ther Methods, 30(5):153-171, 10 2019. [DOI:10.1089/hgtb.2019.076] [PubMed:31516018]. |
| [27] | H. B?ning, F. Bosch, and F. Mingozzi. Breaking the Barriers of Genetic and Metabolic Disorders. Hum. Gene Ther., 30(10):1177-1179, 10 2019. [DOI:10.1089/hum.2019.29093.hbu] [PubMed:31573872]. |
| [26] | S. Charrier, C. Lagresle-Peyrou, V. Poletti, M. Rothe, G. C?drone, B. Gjata, F. Mavilio, A. Fischer, A. Schambach, J. P. de Villartay, M. Cavazzana, S. Hacein-Bey-Abina, and A. Galy. Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Mol Ther Methods Clin Dev, 15:232-245, Dec 2019. [PubMed Central:PMC6838972] [DOI:10.1016/j.omtm.2019.08.014] [PubMed:27611239]. |
| [25] | P. Colella and F. Mingozzi. Gene Therapy for Pompe Disease: The Time is now. Hum. Gene Ther., 30(10):1245-1262, 10 2019. [DOI:10.1089/hum.2019.109] [PubMed:31298581]. |
| [24] | P. Colella, P. Sellier, H. Costa Verdera, F. Puzzo, L. van Wittenberghe, N. Guerchet, N. Daniele, B. Gjata, S. Marmier, S. Charles, M. Simon Sola, I. Ragone, C. Leborgne, F. Collaud, and F. Mingozzi. AAV Gene Transfer with Tandem Promoter Design Prevents Anti-transgene Immunity and Provides Persistent Efficacy in Neonate Pompe Mice. Mol Ther Methods Clin Dev, 12:85-101, Mar 2019. [PubMed Central:PMC6299151] [DOI:10.1016/j.omtm.2018.11.002] [PubMed:9668092]. |
| [23] | F. Collaud, G. Bortolussi, L. Guianvarc'h, S. J. Aronson, T. Bordet, P. Veron, S. Charles, P. Vidal, M. S. Sola, S. Rundwasser, D. G. Dufour, F. Lacoste, C. Luc, L. V. Wittenberghe, S. Martin, C. Le Bec, P. J. Bosma, A. F. Muro, G. Ronzitti, M. Hebben, and F. Mingozzi. Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome. Mol Ther Methods Clin Dev, 12:157-174, Mar 2019. [PubMed Central:PMC6348934] [DOI:10.1016/j.omtm.2018.12.011] [PubMed:9382955]. |
| [22] | S. Da Rocha, J. Bigot, F. Onodi, J. Cosette, G. Corre, J. Poupiot, D. Fenard, B. Gjata, A. Galy, and T. M. A. Neildez-Nguyen. Temporary Reduction of Membrane CD4 with the Antioxidant MnTBAP Is Sufficient to Prevent Immune Responses Induced by Gene Transfer. Mol Ther Methods Clin Dev, 14:285-299, Sep 2019. [PubMed Central:PMC6718808] [DOI:10.1016/j.omtm.2019.06.011] [PubMed:31001252]. |
| [21] | S. F. Henriques, E. Gicquel, J. Marsolier, and I. Richard. Functional and cellular localization diversity associated with Fukutin-related protein patient genetic variants. Hum. Mutat., 40(10):1874-1885, 10 2019. [DOI:10.1002/humu.23827] [PubMed:31268217]. |
| [20] | P. Lafor?t, M. Inoue, E. Goillot, C. Lefeuvre, U. Cagin, N. Streichenberger, S. Leonard-Louis, G. Brochier, A. Madelaine, C. Labasse, C. Hedberg-Oldfors, T. Krag, L. Jauze, J. Fabregue, P. Labrune, J. Milisenda, A. Nadaj-Pakleza, S. Sacconi, F. Mingozzi, G. Ronzitti, F. Petit, B. Schoser, A. Oldfors, J. Vissing, N. B. Romero, I. Nishino, and E. Malfatti. Deep morphological analysis of muscle biopsies from type III glycogenesis (GSDIII), debranching enzyme deficiency, revealed stereotyped vacuolar myopathy and autophagy impairment. Acta Neuropathol Commun, 7(1):167, 10 2019. [PubMed Central:PMC6819650] [DOI:10.1186/s40478-019-0815-2] [PubMed:23318145]. |
| [19] | S. Le Quellec, A. P. Dane, E. Barbon, J. C. Bordet, F. Mingozzi, Y. Dargaud, T. Marais, M. G. Biferi, C. N?grier, A. C. Nathawani, and N. Enjolras. Recombinant Adeno-Associated Viral Vectors Expressing Human Coagulation FIX-E456H Variant in Hemophilia B Mice. Thromb. Haemost., 119(12):1956-1967, Dec 2019. [DOI:10.1055/s-0039-1697658] [PubMed:31659733]. |
| [18] | J. Poupiot, H. Costa Verdera, R. Hardet, P. Colella, F. Collaud, L. Bartolo, J. Davoust, P. Sanatine, F. Mingozzi, I. Richard, and G. Ronzitti. Role of Regulatory T Cell and Effector T Cell Exhaustion in Liver-Mediated Transgene Tolerance in Muscle. Mol Ther Methods Clin Dev, 15:83-100, Dec 2019. [PubMed Central:PMC6804827] [DOI:10.1016/j.omtm.2019.08.012] [PubMed:28284982]. |
| [17] | C. Radek, O. Bernadin, K. Drechsel, N. Cordes, R. Pfeifer, P. Str??er, M. Mormin, A. Gutierrez-Guerrero, F. L. Cosset, A. D. Kaiser, T. Schaser, A. Galy, E. Verhoeyen, and I. C. D. Johnston. Vectofusin-1 Improves Transduction of Primary Human Cells with Diverse Retroviral and Lentiviral Pseudotypes, Enabling Robust, Automated Closed-System Manufacturing. Hum. Gene Ther., 30(12):1477-1493, 12 2019. [PubMed Central:PMC6919281] [DOI:10.1089/hum.2019.157] [PubMed:30224381]. |
| [16] | P. R?o, S. Navarro, W. Wang, R. S?nchez-Dom?nguez, R. M. Pujol, J. C. Segovia, M. Bogliolo, E. Merino, N. Wu, R. Salgado, M. L. Lamana, R. M. Ya?ez, J. A. Casado, Y. Gim?nez, F. J. Rom?n-Rodr?guez, L. ?lvarez, O. Alberquilla, A. Raimbault, G. Guenechea, M. L. Lozano, L. Cerrato, M. Hernando, E. G?lvez, R. Hladun, I. Giralt, J. Barquinero, A. Galy, N. Garc?a de Ando?n, R. L?pez, A. Catal?, J. D. Schwartz, J. Surrall?s, J. Soulier, M. Schmidt, C. D?az de Heredia, J. Sevilla, and J. A. Bueren. Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nat. Med., 25(9):1396-1401, 09 2019. [DOI:10.1038/s41591-019-0550-z] [PubMed:31501599]. |
| [15] | N. S. Orefice, B. Souchet, J. Braudeau, S. Alves, F. Piguet, F. Collaud, G. Ronzitti, S. Tada, P. Hantraye, F. Mingozzi, F. Ducong?, and N. Cartier. Real-Time Monitoring of Exosome Enveloped-AAV Spreading by Endomicroscopy Approach: A New Tool for Gene Delivery in the Brain. Mol Ther Methods Clin Dev, 14:237-251, Sep 2019. [PubMed Central:PMC6699252] [DOI:10.1016/j.omtm.2019.06.005] [PubMed:9813665]. |
| [14] | G. Ronzitti, F. Collaud, P. Laforet, and F. Mingozzi. Progress and challenges of gene therapy for Pompe disease. Ann Transl Med, 7(13):287, Jul 2019. [PubMed Central:PMC6642941] [DOI:10.21037/atm.2019.04.67] [PubMed:9829534]. |
| [13] | L. Jauze, L. Monteillet, G. Mithieux, F. Rajas, and G. Ronzitti. Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III. Hum. Gene Ther., 30(10):1263-1273, 10 2019. [DOI:10.1089/hum.2019.102] [PubMed:31319709]. |
| [12] | E. Casajus Pelegay, F. Puzzo, A. Yilmazer, and U. Cagin. Targeting Mitochondrial Defects to Increase Longevity in Animal Models of Neurodegenerative Diseases. Adv. Exp. Med. Biol., 1134:89-110, 2019. [DOI:10.1007/978-3-030-12668-1_5] [PubMed:30919333]. |
| [11] | M. Lo Scrudato, K. Poulard, C. Sourd, S. Tom?, A. F. Klein, G. Corre, A. Huguet, D. Furling, G. Gourdon, and A. Buj-Bello. Genome Editing of Expanded CTG Repeats within the Human DMPK Gene Reduces Nuclear RNA Foci in the Muscle of DM1 Mice. Mol. Ther., 27(8):1372-1388, 08 2019. [PubMed Central:PMC6697452] [DOI:10.1016/j.ymthe.2019.05.021] [PubMed:29056323]. |
| [10] | D. W. Scott and F. Mingozzi. Balancing immunity and tolerance in gene therapy for inherited and acquired diseases. Cell. Immunol., 342:103945, 08 2019. [DOI:10.1016/j.cellimm.2019.103945] [PubMed:31229195]. |
| [9] | D. Israeli, J. Cosette, G. Corre, F. Amor, J. Poupiot, D. Stockholm, M. Montus, B. Gjata, and I. Richard. An AAV-SGCG Dose-Response Study in a γ-Sarcoglycanopathy Mouse Model in the Context of Mechanical Stress. Mol Ther Methods Clin Dev, 13:494-502, Jun 2019. [PubMed Central:PMC6545357] [DOI:10.1016/j.omtm.2019.04.007] [PubMed:9732288]. |
| [8] | L. Hoch, S. F. Henriques, C. Bruge, J. Marsolier, M. Benabides, N. Bourg, J. Tournois, G. Mah?, L. Morizur, M. Jarrige, A. Bigot, I. Richard, and X. Nissan. Identification of thiostrepton as a pharmacological approach to rescue misfolded alpha-sarcoglycan mutant proteins from degradation. Sci Rep, 9(1):6915, 05 2019. [PubMed Central:PMC6502821] [DOI:10.1038/s41598-019-43399-w] [PubMed:31061434]. |
| [7] | C. Kutchukian, P. Szentesi, B. Allard, A. Buj-Bello, L. Csernoch, and V. Jacquemond. Ca2+-induced sarcoplasmic reticulum Ca2+ release in myotubularin-deficient muscle fibers. Cell Calcium, 80:91-100, 06 2019. [DOI:10.1016/j.ceca.2019.04.004] [PubMed:30999217]. |
| [6] | W. Lostal, D. Stockholm, and I. Richard. In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer. Methods Mol. Biol., 1915:57-66, 2019. [DOI:10.1007/978-1-4939-8988-1_5] [PubMed:30617795]. |
| [5] | A. Lattanzi, V. Meneghini, G. Pavani, F. Amor, S. Ramadier, T. Felix, C. Antoniani, C. Masson, O. Alibeu, C. Lee, M. H. Porteus, G. Bao, M. Amendola, F. Mavilio, and A. Miccio. Optimization of CRISPR/Cas9 Delivery to Human Hematopoietic Stem and Progenitor Cells for Therapeutic Genomic Rearrangements. Mol. Ther., 27(1):137-150, 01 2019. [PubMed Central:PMC6318785] [DOI:10.1016/j.ymthe.2018.10.008] [PubMed:25733580]. |
| [4] | C. Leborgne, V. Latournerie, S. Boutin, D. Desgue, A. Qu?r?, E. Pignot, F. Collaud, S. Charles, M. Simon Sola, E. Masat, F. Jouen, O. Boyer, C. Masurier, F. Mingozzi, and P. Veron. Prevalence and long-term monitoring of humoral immunity against adeno-associated virus in Duchenne Muscular Dystrophy patients. Cell. Immunol., 342:103780, 08 2019. [DOI:10.1016/j.cellimm.2018.03.004] [PubMed:29571923]. |
| [3] | W. Lostal, C. Roudaut, M. Faivre, K. Charton, L. Suel, N. Bourg, H. Best, J. E. Smith, J. Gohlke, G. Corre, X. Li, Z. Elbeck, R. Kn?ll, J. Y. Deschamps, H. Granzier, and I. Richard. Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A. Sci Transl Med, 11(520), 11 2019. [DOI:10.1126/scitranslmed.aat6072] [PubMed:31776291]. |
| [2] | R. Cabrera-P?rez, F. Vila-Juli?, M. Hirano, F. Mingozzi, J. Torres-Torronteras, and R. Mart? Alpha-1-Antitrypsin Promoter Improves the Efficacy of an Adeno-Associated Virus Vector for the Treatment of Mitochondrial Neurogastrointestinal Encephalomyopathy. Hum. Gene Ther., 30(8):985-998, 08 2019. [DOI:10.1089/hum.2018.217] [PubMed:30900470]. |
| [1] | E. Danese, S. Raimondi, M. Montagnana, A. Tagetti, T. Langaee, P. Borgiani, C. Ciccacci, A. J. Carcas, A. M. Borobia, H. Y. Tong, C. D?vila-Fajardo, M. Rodrigues Botton, S. Bourgeois, P. Deloukas, M. D. Caldwell, J. K. Burmester, R. L. Berg, L. H. Cavallari, K. Drozda, M. Huang, L. Z. Zhao, H. J. Cen, R. Gonzalez-Conejero, V. Roldan, Y. Nakamura, T. Mushiroda, I. Y. Gong, R. B. Kim, K. Hirai, K. Itoh, C. Isaza, L. Beltr?n, E. Jim?nez-Varo, M. Ca?adas-Garre, A. Giontella, M. K. Kringen, K. B. F. Haug, H. S. Gwak, K. E. Lee, P. Minuz, M. T. M. Lee, S. A. Lubitz, S. Scott, C. Mazzaccara, L. Sacchetti, E. Gen?, M. ?zer, A. Pathare, R. Krishnamoorthy, A. Paldi, V. Siguret, M. A. Loriot, V. K. Kutala, G. Suarez-Kurtz, J. Perini, J. C. Denny, A. H. Ramirez, B. Mittal, S. S. Rathore, H. Sagreiya, R. Altman, M. H. A. Shahin, S. I. Khalifa, N. A. Limdi, C. Rivers, A. Shendre, C. Dillon, I. M. Suriapranata, H. H. Zhou, S. L. Tan, V. Tatarunas, V. Lesauskaite, Y. Zhang, A. H. Maitland-van der Zee, T. I. Verhoef, A. de Boer, M. Taljaard, C. F. Zambon, V. Pengo, J. E. Zhang, M. Pirmohamed, J. A. Johnson, and C. Fava. Effect of CYP4F2, VKORC1, and CYP2C9 in Influencing Coumarin Dose: A Single-Patient Data Meta-Analysis in More Than 15,000 Individuals. Clin. Pharmacol. Ther., 105(6):1477-1491, 06 2019. [PubMed Central:PMC6542461] [DOI:10.1002/cpt.1323] [PubMed:29238065]. |
| [31] | V. Poletti, F. Urbinati, S. Charrier, G. Corre, R. P. Hollis, B. Campo Fernandez, S. Martin, M. Rothe, A. Schambach, D. B. Kohn, and F. Mavilio. Pre-clinical Development of a Lentiviral Vector Expressing the Anti-sickling βAS3 Globin for Gene Therapy for Sickle Cell Disease. Mol Ther Methods Clin Dev, 11:167-179, Dec 2018. [PubMed Central:PMC6276308] [DOI:10.1016/j.omtm.2018.10.014] [PubMed:12933581]. |
| [30] | R. Avila-Polo, E. Malfatti, X. Lornage, C. Cheraud, I. Nelson, J. Nectoux, J. Bohm, R. Schneider, C. Hedberg-Oldfors, B. Eymard, S. Monges, F. Lubieniecki, G. Brochier, M. Thao Bui, A. Madelaine, C. Labasse, M. Beuvin, E. Lacene, A. Boland, J. F. Deleuze, J. Thompson, I. Richard, A. L. Taratuto, B. Udd, F. Leturcq, G. Bonne, A. Oldfors, J. Laporte, and N. B. Romero. Loss of Sarcomeric Scaffolding as a Common Baseline Histopathologic Lesion in Titin-Related Myopathies. J. Neuropathol. Exp. Neurol., 77(12):1101-1114, Dec 2018. [DOI:10.1093/jnen/nly095] [PubMed:30365001]. |
| [29] | C. C. Weihl, B. Udd, M. Hanna, A. Ben-Zvi, T. Blaettler, R. Bryson-Richardson, S. Carra, M. Dimachkie, A. Findlay, L. Greensmith, S. Greenspan, M. Hanna, J. Hohfled, P. H. Jonson, H. Kampinga, L. Larsson, W. Linke, G. Lynch, P. Machado, L. Orlando, I. Richard, A. Roos, J. Sarparanta, V. Timmerman, B. Udd, C. Weihl, and L. Zah. 234th ENMC International Workshop: Chaperone dysfunction in muscle disease Naarden, The Netherlands, 8-10 December 2017. Neuromuscul. Disord., 28(12):1022-1030, Dec 2018. [DOI:10.1016/j.nmd.2018.09.004] [PubMed:30424919]. |
| [28] | R. Mark Wightman, N. Dominguez, and R. Borges. How intravesicular composition affects exocytosis. Pflugers Arch., 470(1):135-141, 01 2018. [DOI:10.1007/s00424-017-2035-6] [PubMed:18369955]. |
| [27] | K. Kuranda, P. Jean-Alphonse, C. Leborgne, R. Hardet, F. Collaud, S. Marmier, H. Costa Verdera, G. Ronzitti, P. Veron, and F. Mingozzi. Exposure to wild-type AAV drives distinct capsid immunity profiles in humans. J. Clin. Invest., 128(12):5267-5279, Dec 2018. [PubMed Central:PMC6264647] [DOI:10.1172/JCI122372] [PubMed:22643865]. |
| [26] | M. R. Lidonnici, Y. Paleari, F. Tiboni, G. Mandelli, C. Rossi, M. Vezzoli, A. Aprile, C. W. Lederer, A. Ambrosi, F. Chanut, F. Sanvito, A. Calabria, V. Poletti, F. Mavilio, E. Montini, L. Naldini, P. Cristofori, and G. Ferrari. Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia. Mol Ther Methods Clin Dev, 11:9-28, Dec 2018. [PubMed Central:PMC6178212] [DOI:10.1016/j.omtm.2018.09.001] [PubMed:8453619]. |
| [25] | A. Meliani, F. Boisgerault, R. Hardet, S. Marmier, F. Collaud, G. Ronzitti, C. Leborgne, H. Costa Verdera, M. Simon Sola, S. Charles, A. Vignaud, L. van Wittenberghe, G. Manni, O. Christophe, F. Fallarino, C. Roy, A. Michaud, P. Ilyinskii, T. K. Kishimoto, and F. Mingozzi. Antigen-selective modulation of AAV immunogenicity with tolerogenic rapamycin nanoparticles enables successful vector re-administration. Nat Commun, 9(1):4098, 10 2018. [PubMed Central:PMC6173722] [DOI:10.1038/s41467-018-06621-3] [PubMed:19956269]. |
| [24] | F. Mingozzi. AAV Immunogenicity: A Matter of Sensitivity. Mol. Ther., 26(10):2335-2336, Oct 2018. [PubMed Central:PMC6171166] [DOI:10.1016/j.ymthe.2018.09.001] [PubMed:19441963]. |
| [23] | F. Urbinati, B. Campo Fernandez, K. E. Masiuk, V. Poletti, R. P. Hollis, C. Koziol, M. L. Kaufman, D. Brown, F. Mavilio, and D. B. Kohn. Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study. Hum. Gene Ther., 29(10):1153-1166, Oct 2018. [DOI:10.1089/hum.2018.061] [PubMed:30198339]. |
| [22] | A. Izmiryan, C. Ganier, M. Bovolenta, A. Schmitt, F. Mavilio, and A. Hovnanian. Ex Vivo COL7A1 Correction for Recessive Dystrophic Epidermolysis Bullosa Using CRISPR/Cas9 and Homology-Directed Repair. Mol Ther Nucleic Acids, 12:554-567, Sep 2018. [PubMed Central:PMC6077132] [DOI:10.1016/j.omtn.2018.06.008] [PubMed:24837660]. |
| [21] | L. Weber, V. Poletti, E. Magrin, C. Antoniani, S. Martin, C. Bayard, H. Sadek, T. Felix, V. Meneghini, M. N. Antoniou, W. El-Nemer, F. Mavilio, M. Cavazzana, I. Andre-Schmutz, and A. Miccio. An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype. Mol Ther Methods Clin Dev, 10:268-280, Sep 2018. [PubMed Central:PMC6105766] [DOI:10.1016/j.omtm.2018.07.012] [PubMed:20665635]. |
| [20] | H. Khabou, C. Cordeau, L. Pacot, S. Fisson, and D. Dalkara. Dosage Thresholds and Influence of Transgene Cassette in Adeno-Associated Virus-Related Toxicity. Hum. Gene Ther., 29(11):1235-1241, Nov 2018. [DOI:10.1089/hum.2018.144] [PubMed:30132368]. |
| [19] | Z. Fitzpatrick, C. Leborgne, E. Barbon, E. Masat, G. Ronzitti, L. van Wittenberghe, A. Vignaud, F. Collaud, S. Charles, M. Simon Sola, F. Jouen, O. Boyer, and F. Mingozzi. Influence of Pre-existing Anti-capsid Neutralizing and Binding Antibodies on AAV Vector Transduction. Mol Ther Methods Clin Dev, 9:119-129, Jun 2018. [PubMed Central:PMC5948224] [DOI:10.1016/j.omtm.2018.02.003] [PubMed:16178850]. |
| [18] | V. Poletti, S. Charrier, G. Corre, B. Gjata, A. Vignaud, F. Zhang, M. Rothe, A. Schambach, H. B. Gaspar, A. J. Thrasher, and F. Mavilio. Preclinical Development of a Lentiviral Vector for Gene Therapy of X-Linked Severe Combined Immunodeficiency. Mol Ther Methods Clin Dev, 9:257-269, Jun 2018. [PubMed Central:PMC5918176] [DOI:10.1016/j.omtm.2018.03.002] [PubMed:20660403]. |
| [17] | I. Donadon, M. Pinotti, K. Rajkowska, G. Pianigiani, E. Barbon, E. Morini, H. Motaln, B. Rogelj, F. Mingozzi, S. A. Slaugenhaupt, and F. Pagani. Exon-specific U1 snRNAs improve ELP1 exon 20 definition and rescue ELP1 protein expression in a familial dysautonomia mouse model. Hum. Mol. Genet., 27(14):2466-2476, 07 2018. [PubMed Central:PMC6030917] [DOI:10.1093/hmg/ddy151] [PubMed:11179008]. |
| [16] | E. C. Oates, K. J. Jones, S. Donkervoort, A. Charlton, S. Brammah, J. E. Smith, J. S. Ware, K. S. Yau, L. C. Swanson, N. Whiffin, A. J. Peduto, A. Bournazos, L. B. Waddell, M. A. Farrar, H. A. Sampaio, H. L. Teoh, P. J. Lamont, D. Mowat, R. B. Fitzsimons, A. J. Corbett, M. M. Ryan, G. L. O'Grady, S. A. Sandaradura, R. Ghaoui, H. Joshi, J. L. Marshall, M. A. Nolan, S. Kaur, J. Punetha, A. Topf, E. Harris, M. Bakshi, C. A. Genetti, M. Marttila, U. Werlauff, N. Streichenberger, A. Pestronk, I. Mazanti, J. R. Pinner, C. Vuillerot, C. Grosmann, A. Camacho, P. Mohassel, M. E. Leach, A. R. Foley, D. Bharucha-Goebel, J. Collins, A. M. Connolly, H. R. Gilbreath, S. T. Iannaccone, D. Castro, B. B. Cummings, R. I. Webster, L. Lazaro, J. Vissing, S. Coppens, N. Deconinck, H. M. Luk, N. H. Thomas, N. C. Foulds, M. A. Illingworth, S. Ellard, C. A. McLean, R. Phadke, G. Ravenscroft, N. Witting, P. Hackman, I. Richard, S. T. Cooper, E. J. Kamsteeg, E. P. Hoffman, K. Bushby, V. Straub, B. Udd, A. Ferreiro, K. N. North, N. F. Clarke, M. Lek, A. H. Beggs, C. G. Bonnemann, D. G. MacArthur, H. Granzier, M. R. Davis, and N. G. Laing. Congenital Titinopathy: Comprehensive characterization and pathogenic insights. Ann. Neurol., 83(6):1105-1124, Jun 2018. [PubMed Central:PMC6105519] [DOI:10.1002/ana.25241] [PubMed:10625306]. |
| [15] | C. Brendel, M. Rothe, G. Santilli, S. Charrier, S. Stein, H. Kunkel, D. Abriss, U. Muller-Kuller, B. Gaspar, U. Modlich, A. Galy, A. Schambach, A. J. Thrasher, and M. Grez. Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease. Hum Gene Ther Clin Dev, 29(2):69-79, 06 2018. [DOI:10.1089/humc.2017.245] [PubMed:29664709]. |
| [14] | J. Vendomele, S. Dehmani, Q. Khebizi, A. Galy, and S. Fisson. Subretinal Injection of HY Peptides Induces Systemic Antigen-Specific Inhibition of Effector CD4+ and CD8+ T-Cell Responses. Front Immunol, 9:504, 2018. [PubMed Central:PMC5890180] [DOI:10.3389/fimmu.2018.00504] [PubMed:17264480]. |
| [13] | W. Lostal, J. A. Urtizberea, I. Richard, A. Alonso-Jimenez, R. Y. Carlier, V. Carson, J. Diaz-Manera, B. Eymard, M. Fardeau, M. L. Gourlay, M. Guglieri, J. Y. Hogrel, B. Kullmann, J. Levy, W. Lostal, Y. Ono, H. Prigent, I. Richard, A. Saenz, C. Semplicini, J. A. Urtizberea, M. Vainzof, J. Vissing, and M. Walter. 233rd ENMC International Workshop:: Clinical Trial Readiness for Calpainopathies, Naarden, The Netherlands, 15-17 September 2017. Neuromuscul. Disord., 28(6):540-549, Jun 2018. [DOI:10.1016/j.nmd.2018.03.010] [PubMed:29655529]. |
| [12] | C. Antoniani, V. Meneghini, A. Lattanzi, T. Felix, O. Romano, E. Magrin, L. Weber, G. Pavani, S. El Hoss, R. Kurita, Y. Nakamura, T. J. Cradick, A. S. Lundberg, M. Porteus, M. Amendola, W. El Nemer, M. Cavazzana, F. Mavilio, and A. Miccio. Induction of fetal hemoglobin synthesis by CRISPR/Cas9-mediated editing of the human β-globin locus. Blood, 131(17):1960-1973, Apr 2018. [DOI:10.1182/blood-2017-10-811505] [PubMed:29519807]. |
| [11] | M. Holstein, C. Mesa-Nunez, C. Miskey, E. Almarza, V. Poletti, M. Schmeer, E. Grueso, J. C. Ordonez Flores, D. Kobelt, W. Walther, M. K. Aneja, J. Geiger, H. B. Bonig, Z. Izsvak, M. Schleef, C. Rudolph, F. Mavilio, J. A. Bueren, G. Guenechea, and Z. Ivics. Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors. Mol. Ther., 26(4):1137-1153, Apr 2018. [PubMed Central:PMC6079369] [DOI:10.1016/j.ymthe.2018.01.012] [PubMed:23745681]. |
| [10] | P. H. Jonson, J. Palmio, M. Johari, S. Penttila, A. Evila, I. Nelson, G. Bonne, N. Wiart, V. Meyer, A. Boland, J. F. Deleuze, C. Masson, T. Stojkovic, F. Chapon, N. B. Romero, G. Sole, X. Ferrer, A. Ferreiro, P. Hackman, I. Richard, and B. Udd. Novel mutations in DNAJB6 cause LGMD1D and distal myopathy in French families. Eur. J. Neurol., 25(5):790-794, 05 2018. [DOI:10.1111/ene.13598] [PubMed:29437287]. |
| [9] | N. Daniele, C. Moal, L. Julien, M. Marinello, T. Jamet, S. Martin, A. Vignaud, M. W. Lawlor, and A. Buj-Bello. Intravenous Administration of a MTMR2-Encoding AAV Vector Ameliorates the Phenotype of Myotubular Myopathy in Mice. J. Neuropathol. Exp. Neurol., 77(4):282-295, Apr 2018. [PubMed Central:PMC5939852] [DOI:10.1093/jnen/nly002] [PubMed:22645112]. |
| [8] | P. Vidal, S. Pagliarani, P. Colella, H. Costa Verdera, L. Jauze, M. Gjorgjieva, F. Puzzo, S. Marmier, F. Collaud, M. Simon Sola, S. Charles, S. Lucchiari, L. van Wittenberghe, A. Vignaud, B. Gjata, I. Richard, P. Laforet, E. Malfatti, G. Mithieux, F. Rajas, G. P. Comi, G. Ronzitti, and F. Mingozzi. Rescue of GSDIII Phenotype with Gene Transfer Requires Liver- and Muscle-Targeted GDE Expression. Mol. Ther., 26(3):890-901, 03 2018. [PubMed Central:PMC5910667] [DOI:10.1016/j.ymthe.2017.12.019] [PubMed:15585406]. |
| [7] | S. F. Henriques, C. Patissier, N. Bourg, C. Fecchio, D. Sandona, J. Marsolier, and I. Richard. Different outcome of sarcoglycan missense mutation between human and mouse. PLoS ONE, 13(1):e0191274, 2018. [PubMed Central:PMC5779665] [DOI:10.1371/journal.pone.0191274] [PubMed:16722804]. |
| [6] | M. Carotti, J. Marsolier, M. Soardi, E. Bianchini, C. Gomiero, C. Fecchio, S. F. Henriques, R. Betto, R. Sacchetto, I. Richard, and D. Sandona. Repairing folding-defective α-sarcoglycan mutants by CFTR correctors, a potential therapy for limb-girdle muscular dystrophy 2D. Hum. Mol. Genet., 27(6):969-984, 03 2018. [PubMed Central:PMC5886177] [DOI:10.1093/hmg/ddy013] [PubMed:9146999]. |
| [5] | P. Colella, G. Ronzitti, and F. Mingozzi. Emerging Issues in AAV-Mediated In Vivo Gene Therapy. Mol Ther Methods Clin Dev, 8:87-104, Mar 2018. [PubMed Central:PMC5758940] [DOI:10.1016/j.omtm.2017.11.007] [PubMed:23939025]. |
| [4] | G. Ronzitti and F. Mingozzi. Combination Therapy Is the New Gene Therapy? Mol. Ther., 26(1):12-14, 01 2018. [PubMed Central:PMC5763149] [DOI:10.1016/j.ymthe.2017.12.008] [PubMed:25568350]. |
| [3] | V. Poletti and F. Mavilio. Interactions between Retroviruses and the Host Cell Genome. Mol Ther Methods Clin Dev, 8:31-41, Mar 2018. [PubMed Central:PMC5684498] [DOI:10.1016/j.omtm.2017.10.001] [PubMed:16959972]. |
| [2] | A. Paldi. Conceptual Challenges of the Systemic Approach in Understanding Cell Differentiation. Methods Mol. Biol., 1702:27-39, 2018. [DOI:10.1007/978-1-4939-7456-6_3] [PubMed:29119500]. |
| [1] | N. K. Paulk, K. Pekrun, E. Zhu, S. Nygaard, B. Li, J. Xu, K. Chu, C. Leborgne, A. P. Dane, A. Haft, Y. Zhang, F. Zhang, C. Morton, M. B. Valentine, A. M. Davidoff, A. C. Nathwani, F. Mingozzi, M. Grompe, I. E. Alexander, L. Lisowski, and M. A. Kay. Bioengineered AAV Capsids with Combined High Human Liver Transduction In Vivo and Unique Humoral Seroreactivity. Mol. Ther., 26(1):289-303, 01 2018. [PubMed Central:PMC5763027] [DOI:10.1016/j.ymthe.2017.09.021] [PubMed:18400866]. |
| [35] | A. Boucheham, V. Sommard, F. Zehraoui, A. Boualem, M. Batouche, A. Bendahmane, D. Israeli, and F. Tahi. IpiRId: Integrative approach for piRNA prediction using genomic and epigenomic data. PLoS ONE, 12(6):e0179787, 2017. [PubMed Central:PMC5473586] [DOI:10.1371/journal.pone.0179787] [PubMed:28622364]. |
| [34] | T. Soheili, A. Durand, F. E. Sepulveda, J. Riviere, C. Lagresle-Peyrou, H. Sadek, G. de Saint Basile, S. Martin, F. Mavilio, M. Cavazzana, and I. Andre-Schmutz. Gene transfer into hematopoietic stem cells reduces HLH manifestations in a murine model of Munc13-4 deficiency. Blood Adv, 1(27):2781-2789, Dec 2017. [PubMed Central:PMC5745141] [DOI:10.1182/bloodadvances.2017012088] [PubMed:29296930]. |
| [33] | A. Meliani, F. Boisgerault, Z. Fitzpatrick, S. Marmier, C. Leborgne, F. Collaud, M. Simon Sola, S. Charles, G. Ronzitti, A. Vignaud, L. van Wittenberghe, B. Marolleau, F. Jouen, S. Tan, O. Boyer, O. Christophe, A. R. Brisson, C. A. Maguire, and F. Mingozzi. Enhanced liver gene transfer and evasion of preexisting humoral immunity with exosome-enveloped AAV vectors. Blood Adv, 1(23):2019-2031, Oct 2017. [PubMed Central:PMC5728288] [DOI:10.1182/bloodadvances.2017010181] [PubMed:29296848]. |
| [32] | J. Vendomele, Q. Khebizi, and S. Fisson. Cellular and Molecular Mechanisms of Anterior Chamber-Associated Immune Deviation (ACAID): What We Have Learned from Knockout Mice. Front Immunol, 8:1686, 2017. [PubMed Central:PMC5714853] [DOI:10.3389/fimmu.2017.01686] [PubMed:29250068]. |
| [31] | F. Puzzo, P. Colella, M. G. Biferi, D. Bali, N. K. Paulk, P. Vidal, F. Collaud, M. Simon-Sola, S. Charles, R. Hardet, C. Leborgne, A. Meliani, M. Cohen-Tannoudji, S. Astord, B. Gjata, P. Sellier, L. van Wittenberghe, A. Vignaud, F. Boisgerault, M. Barkats, P. Laforet, M. A. Kay, D. D. Koeberl, G. Ronzitti, and F. Mingozzi. Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid α-glucosidase. Sci Transl Med, 9(418), Nov 2017. [PubMed Central:PMC5826611] [DOI:10.1126/scitranslmed.aam6375] [PubMed:29187643]. |
| [30] | Patrice Vidal, Serena Pagliarani, Pasqualina Colella, Helena Costa Verdera, Louisa Jauze, Monika Gjorgjieva, Francesco Puzzo, Solenne Marmier, Fanny Collaud, Marcelo Simon Sola, et al. Rescue of gsdiii phenotype with gene transfer requires liver-and muscle-targeted gde expression. Molecular Therapy, 2017. |
| [29] | A Vihola, H Luque, M Savarese, S Penttilä, M Lindfors, F Leturcq, B Eymard, G Tasca, B Brais, T Conte, et al. Diagnostic anoctamin-5 protein defect in patients with ano5-mutated muscular dystrophy. Neuropathology and applied neurobiology, 2017. |
| [28] | L. S. Vermeer, L. Hamon, A. Schirer, M. Schoup, J. Cosette, S. Majdoul, D. Pastre, D. Stockholm, N. Holic, P. Hellwig, A. Galy, D. Fenard, and B. Bechinger. Vectofusin-1, a potent peptidic enhancer of viral gene transfer forms pH-dependent α-helical nanofibrils, concentrating viral particles. Acta Biomater, 64:259-268, Dec 2017. [DOI:10.1016/j.actbio.2017.10.009] [PubMed:29017974]. |
| [27] | F. Mingozzi and K. A. High. Overcoming the Host Immune Response to Adeno-Associated Virus Gene Delivery Vectors: The Race Between Clearance, Tolerance, Neutralization, and Escape. Annu Rev Virol, 4(1):511-534, Sep 2017. [DOI:10.1146/annurev-virology-101416-041936] [PubMed:28961410]. |
| [26] | Virginie Mariot, Romain Joubert, Christophe Hourdé, Léonard Féasson, Michael Hanna, Francesco Muntoni, Thierry Maisonobe, Laurent Servais, Caroline Bogni, Rozen Panse, et al. Downregulation of myostatin pathway in neuromuscular diseases may explain challenges of anti-myostatin therapeutic approaches. Nature communications, 8(1):1859, 2017. |
| [25] | S. Majdoul, J. Cosette, A. K. Seye, E. Bernard, S. Frin, N. Holic, N. Chazal, L. Briant, L. Espert, A. Galy, and D. Fenard. Peptides derived from evolutionarily conserved domains in Beclin-1 and Beclin-2 enhance the entry of lentiviral vectors into human cells. J. Biol. Chem., 292(45):18672-18681, 11 2017. [PubMed Central:PMC5682973] [DOI:10.1074/jbc.M117.800813] [PubMed:28928217]. |
| [24] | G. Ferrari, M. Cavazzana, and F. Mavilio. Gene Therapy Approaches to Hemoglobinopathies. Hematol. Oncol. Clin. North Am., 31(5):835-852, 10 2017. [DOI:10.1016/j.hoc.2017.06.010] [PubMed:28895851]. |
| [23] | C. Vandamme, O. Adjali, and F. Mingozzi. Unraveling the Complex Story of Immune Responses to AAV Vectors Trial After Trial. Hum. Gene Ther., 28(11):1061-1074, Nov 2017. [PubMed Central:PMC5649404] [DOI:10.1089/hum.2017.150] [PubMed:28835127]. |
| [22] | A. Galy. Major Advances in the Development of Vectors for Clinical Gene Therapy of Hematopoietic Stem Cells from European Groups over the Last 25 Years. Hum. Gene Ther., 28(11):964-971, Nov 2017. [DOI:10.1089/hum.2017.152] [PubMed:28806879]. |
| [21] | Oriana Romano, Ingrid Cifola, Valentina Poletti, Marco Severgnini, Clelia Peano, Gianluca De Bellis, Fulvio Mavilio, and Annarita Miccio. Retroviral scanning: Mapping mlv integration sites to define cell-specific regulatory regions. Journal of visualized experiments: JoVE, (123), 2017. |
| [20] | P. Rio, S. Navarro, G. Guenechea, R. Sanchez-Dominguez, M. L. Lamana, R. Yanez, J. A. Casado, P. A. Mehta, M. R. Pujol, J. Surralles, S. Charrier, A. Galy, J. C. Segovia, C. Diaz de Heredia, J. Sevilla, and J. A. Bueren. Engraftment and in vivo proliferation advantage of gene-corrected mobilized CD34+ cells from Fanconi anemia patients. Blood, 130(13):1535-1542, 09 2017. [DOI:10.1182/blood-2017-03-774174] [PubMed:28801449]. |
| [19] | A. Matet, C. Kostic, A. P. Bemelmans, A. Moulin, S. G. Rosolen, S. Martin, F. Mavilio, V. Amirjanians, K. Stieger, B. Lorenz, F. Behar-Cohen, and Y. Arsenijevic. Evaluation of tolerance to lentiviral LV-RPE65 gene therapy vector after subretinal delivery in non-human primates. Transl Res, 188:40-57, 10 2017. [DOI:10.1016/j.trsl.2017.06.012] [PubMed:28754419]. |
| [18] | A. Moussy, J. Cosette, R. Parmentier, C. da Silva, G. Corre, A. Richard, O. Gandrillon, D. Stockholm, and A. Paldi. Integrated time-lapse and single-cell transcription studies highlight the variable and dynamic nature of human hematopoietic cell fate commitment. PLoS Biol., 15(7):e2001867, Jul 2017. [PubMed Central:PMC5531424] [DOI:10.1371/journal.pbio.2001867] [PubMed:28749943]. |
| [17] | Caroline Le Guiner, Laurent Servais, Marie Montus, Thibaut Larcher, Bodvaël Fraysse, Sophie Moullec, Marine Allais, Virginie François, Maeva Dutilleul, Alberto Malerba, et al. Long-term microdystrophin gene therapy is effective in a canine model of duchenne muscular dystrophy. Nature communications, 8:16105, 2017. |
| [16] | Gilles Moulay, Christian Leborgne, A James Mason, Christopher Aisenbrey, Antoine Kichler, and Burkhard Bechinger. Histidine-rich designer peptides of the lah4 family promote cell delivery of a multitude of cargo. Journal of Peptide Science, 23(4):320-328, 2017. |
| [15] | E. C. Morris, T. Fox, R. Chakraverty, R. Tendeiro, K. Snell, C. Rivat, S. Grace, K. Gilmour, S. Workman, K. Buckland, K. Butler, R. Chee, A. D. Salama, H. Ibrahim, H. Hara, C. Duret, F. Mavilio, F. Male, F. D. Bushman, A. Galy, S. O. Burns, H. B. Gaspar, and A. J. Thrasher. Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. Blood, 130(11):1327-1335, 09 2017. [PubMed Central:PMC5813727] [DOI:10.1182/blood-2017-04-777136] [PubMed:28716862]. |
| [14] | A. Lattanzi, S. Duguez, A. Moiani, A. Izmiryan, E. Barbon, S. Martin, K. Mamchaoui, V. Mouly, F. Bernardi, F. Mavilio, and M. Bovolenta. Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System. Mol Ther Nucleic Acids, 7:11-19, Jun 2017. [PubMed Central:PMC5363679] [DOI:10.1016/j.omtn.2017.02.004] [PubMed:28624187]. |
| [13] | F. Mavilio. Developing gene and cell therapies for rare diseases: an opportunity for synergy between academia and industry. Gene Ther., 24(9):590-592, Sep 2017. [DOI:10.1038/gt.2017.36] [PubMed:28485723]. |
| [12] | C. Piovan, V. Marin, C. Scavullo, S. Corna, E. Giuliani, S. Bossi, A. Galy, D. Fenard, C. Bordignon, G. P. Rizzardi, and C. Bovolenta. Vectofusin-1 Promotes RD114-TR-Pseudotyped Lentiviral Vector Transduction of Human HSPCs and T Lymphocytes. Mol Ther Methods Clin Dev, 5:22-30, Jun 2017. [PubMed Central:PMC5415310] [DOI:10.1016/j.omtm.2017.02.003] [PubMed:28480301]. |
| [11] | M. Elverman, M. A. Goddard, D. Mack, J. M. Snyder, M. W. Lawlor, H. Meng, A. H. Beggs, A. Buj-Bello, K. Poulard, A. P. Marsh, R. W. Grange, V. E. Kelly, and M. K. Childers. Long-term effects of systemic gene therapy in a canine model of myotubular myopathy. Muscle Nerve, 56(5):943-953, Nov 2017. [PubMed Central:PMC5620115] [DOI:10.1002/mus.25658] [PubMed:28370029]. |
| [10] | Christian Leborgne, Debborah Alimi-Guez, Nelly El Shafey, Laetitia van Wittenberghe, Pascal Bigey, Daniel Scherman, and Antoine Kichler. The absorption enhancer sodium deoxycholate promotes high gene transfer in skeletal muscles. International journal of pharmaceutics, 523(1):291-299, 2017. |
| [9] | S. O. Han, G. Ronzitti, B. Arnson, C. Leborgne, S. Li, F. Mingozzi, and D. Koeberl. Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction. Mol Ther Methods Clin Dev, 4:126-136, Mar 2017. [PubMed Central:PMC5363303] [DOI:10.1016/j.omtm.2016.12.010] [PubMed:28344998]. |
| [8] | E. Gicquel, N. Maizonnier, S. J. Foltz, W. J. Martin, N. Bourg, F. Svinartchouk, K. Charton, A. M. Beedle, and I. Richard. AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression. Hum. Mol. Genet., 26(10):1952-1965, 05 2017. [DOI:10.1093/hmg/ddx066] [PubMed:28334834]. |
| [7] | N. Nagy, R. J. Nonneman, T. Llanga, C. F. Dial, N. V. Riddick, T. Hampton, S. S. Moy, K. K. Lehtimaki, T. Ahtoniemi, J. Puolivali, H. Windish, D. Albrecht, I. Richard, and M. L. Hirsch. Hip region muscular dystrophy and emergence of motor deficits in dysferlin-deficient Bla/J mice. Physiol Rep, 5(6), Mar 2017. [PubMed Central:PMC5371557] [DOI:10.14814/phy2.13173] [PubMed:28320887]. |
| [6] | M. Hosel, A. Huber, S. Bohlen, J. Lucifora, G. Ronzitti, F. Puzzo, F. Boisgerault, U. T. Hacker, W. J. Kwanten, N. Kloting, M. Bluher, A. Gluschko, M. Schramm, O. Utermohlen, W. Bloch, F. Mingozzi, O. Krut, and H. Buning. Autophagy determines efficiency of liver-directed gene therapy with adeno-associated viral vectors. Hepatology, 66(1):252-265, 07 2017. [PubMed Central:PMC5518300] [DOI:10.1002/hep.29176] [PubMed:28318036]. |
| [5] | J. Cosette, A. Moussy, A. Paldi, and D. Stockholm. Combination of imaging flow cytometry and time-lapse microscopy for the study of label-free morphology dynamics of hematopoietic cells. Cytometry A, 91(3):254-260, Mar 2017. [DOI:10.1002/cyto.a.23064] [PubMed:28248454]. |
| [4] | D. L. Mack, K. Poulard, M. A. Goddard, V. Latournerie, J. M. Snyder, R. W. Grange, M. R. Elverman, J. Denard, P. Veron, L. Buscara, C. Le Bec, J. Y. Hogrel, A. G. Brezovec, H. Meng, L. Yang, F. Liu, M. O'Callaghan, N. Gopal, V. E. Kelly, B. K. Smith, J. L. Strande, F. Mavilio, A. H. Beggs, F. Mingozzi, M. W. Lawlor, A. Buj-Bello, and M. K. Childers. Systemic AAV8-Mediated Gene Therapy Drives Whole-Body Correction of Myotubular Myopathy in Dogs. Mol. Ther., 25(4):839-854, 04 2017. [PubMed Central:PMC5383631] [DOI:10.1016/j.ymthe.2017.02.004] [PubMed:28237839]. |
| [3] | R. Hardet and F. Mingozzi. Oral Tolerance: Another Reason to Eat Your Veggies! Mol. Ther., 25(2):311-313, 02 2017. [PubMed Central:PMC5368840] [DOI:10.1016/j.ymthe.2017.01.001] [PubMed:28109961]. |
| [2] | N. Holic, S. Frin, A. K. Seye, A. Galy, and D. Fenard. Improvement of De Novo Cholesterol Biosynthesis Efficiently Promotes the Production of Human Immunodeficiency Virus Type 1-Derived Lentiviral Vectors. Hum Gene Ther Methods, 28(2):67-77, 04 2017. [DOI:10.1089/hgtb.2016.150] [PubMed:28042946]. |
| [1] | I. Dalichaouche, Y. Sifi, C. Roudaut, K. Sifi, A. Hamri, L. Rouabah, N. Abadi, and I. Richard. γ-sarcoglycan and dystrophin mutation spectrum in an Algerian cohort. Muscle Nerve, 56(1):129-135, Jul 2017. [DOI:10.1002/mus.25443] [PubMed:27759885]. |
| [32] | L. S. Vermeer, A. Marquette, M. Schoup, D. Fenard, A. Galy, and B. Bechinger. Simultaneous Analysis of Secondary Structure and Light Scattering from Circular Dichroism Titrations: Application to Vectofusin-1. Sci Rep, 6:39450, Dec 2016. [DOI:10.1038/srep39450] [PubMed:28004740]. |
| [31] | C. Kutchukian, M. Lo Scrudato, Y. Tourneur, K. Poulard, A. Vignaud, C. Berthier, B. Allard, M. W. Lawlor, A. Buj-Bello, and V. Jacquemond. Phosphatidylinositol 3-kinase inhibition restores Ca2+ release defects and prolongs survival in myotubularin-deficient mice. Proc. Natl. Acad. Sci. U.S.A., 113(50):14432-14437, Dec 2016. [PubMed Central:PMC5167204] [DOI:10.1073/pnas.1604099113] [PubMed:27911767]. |
| [30] | E. Barbon, M. Ferrarese, L. van Wittenberghe, P. Sanatine, G. Ronzitti, F. Collaud, P. Colella, M. Pinotti, and F. Mingozzi. Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics. Mol Ther Nucleic Acids, 5(11):e392, Nov 2016. [PubMed Central:PMC5155329] [DOI:10.1038/mtna.2016.97] [PubMed:27898092]. |
| [29] | N. El Shafey, M. Guesnon, F. Simon, E. Deprez, J. Cosette, D. Stockholm, D. Scherman, P. Bigey, and A. Kichler. Inhibition of the myostatin/Smad signaling pathway by short decorin-derived peptides. Exp. Cell Res., 341(2):187-195, Feb 2016. [DOI:10.1016/j.yexcr.2016.01.019] [PubMed:26844629]. |
| [28] | F. Nefzi, C. Lambert, A. Gautheret-Dejean, S. Fisson, Q. Khebizi, A. Khelif, H. Agut, and M. Aouni. The cytokines and cellular responses to human herpesvirus-6B in patients with B-acute lymphoblastic leukemia. Microbiol. Immunol., Nov 2016. [DOI:10.1111/1348-0421.12452] [PubMed:27862208]. |
| [27] | E. Hudry, C. Martin, S. Gandhi, B. Gyorgy, D. I. Scheffer, D. Mu, S. F. Merkel, F. Mingozzi, Z. Fitzpatrick, H. Dimant, M. Masek, T. Ragan, S. Tan, A. R. Brisson, S. H. Ramirez, B. T. Hyman, and C. A. Maguire. Exosome-associated AAV vector as a robust and convenient neuroscience tool. Gene Ther., 23(11):819, Nov 2016. [DOI:10.1038/gt.2016.65] [PubMed:27808124]. |
| [26] | E. Masat, P. Laforet, M. De Antonio, G. Corre, B. Perniconi, N. Taouagh, K. Mariampillai, D. Amelin, W. Mauhin, J. Y. Hogrel, C. Caillaud, G. Ronzitti, F. Puzzo, K. Kuranda, P. Colella, R. Mallone, O. Benveniste, F. Mingozzi, G. Bassez, A. L. Bedat-Millet, A. Behin, B. Eymard, S. Leonard-Louis, T. Stojkovic, A. Canal, V. Decostre, F. Bouhour, F. Boyer, Y. Castaing, F. Chapon, P. Cintas, I. Durieu, A. Echaniz-Laguna, L. Feasson, A. Furby, D. Hamroun, X. Ferrer, G. Sole, R. Froissart, M. Piraud, D. Germain, K. Benistan, N. Guffon-Fouilhoux, H. Journel, P. Labauge, A. Lacour, A. Levy, A. Magot, Y. Pereon, M. C. Minot-Myhie, A. Nadaj-Pakleza, C. Nathier, D. Orlikowski, N. Pellegrini, P. Petiot, J. Praline, F. Lofaso, H. Prigent, A. Dutry, D. Renard, S. Sacconi, C. Desnuelle, E. Salort-Campana, J. Pouget, V. Tiffreau, D. Vincent, and F. Zagnoli. Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients. Sci Rep, 6:36182, Nov 2016. [PubMed Central:PMC5096052] [DOI:10.1038/srep36182] [PubMed:27812025]. |
| [25] | I. Dalichaouche, Y. Sifi, C. Roudaut, K. Sifi, A. Hamri, L. Rouabah, N. Abadi, and I. Richard. γ-Sarcoglycan and Dystrophin Mutation Spectrum in an Algerian Cohort. Muscle Nerve, Oct 2016. [DOI:10.1002/mus.25443] [PubMed:27759885]. |
| [24] | G. Ronzitti, G. Bortolussi, R. van Dijk, F. Collaud, S. Charles, C. Leborgne, P. Vidal, S. Martin, B. Gjata, M. S. Sola, L. van Wittenberghe, A. Vignaud, P. Veron, P. J. Bosma, A. F. Muro, and F. Mingozzi. A translationally optimized AAV-UGT1A1 vector drives safe and long-lasting correction of Crigler-Najjar syndrome. Mol Ther Methods Clin Dev, 3:16049, 2016. [DOI:10.1038/mtm.2016.49] [PubMed:27722180]. |
| [23] | J. Martinet, G. Bourdenet, A. Meliani, L. Jean, S. Adriouch, J. L. Cohen, F. Mingozzi, and O. Boyer. Induction of Hematopoietic Microchimerism by Gene-Modified BMT Elicits Antigen-Specific B and T Cell Unresponsiveness toward Gene Therapy Products. Front Immunol, 7:360, 2016. [DOI:10.3389/fimmu.2016.00360] [PubMed:27695454]. |
| [22] | N. Armbruster, A. Lattanzi, M. Jeavons, L. Van Wittenberghe, B. Gjata, T. Marais, S. Martin, A. Vignaud, T. Voit, F. Mavilio, M. Barkats, and A. Buj-Bello. Efficacy and biodistribution analysis of intracerebroventricular administration of an optimized scAAV9-SMN1 vector in a mouse model of spinal muscular atrophy. Mol Ther Methods Clin Dev, 3:16060, 2016. [PubMed Central:PMC5022869] [DOI:10.1038/mtm.2016.60] [PubMed:27652289]. |
| [21] | A. Galy. Like Angler Fish, CAARs Lure Their Prey. Mol. Ther., 24(8):1339-1341, Aug 2016. [DOI:10.1038/mt.2016.165] [PubMed:27578283]. |
| [20] | S. Cire, S. Da Rocha, M. Ferrand, M. K. Collins, and A. Galy. In vivo gene delivery to lymph node stromal cells leads to transgene-specific CD8+ T cell anergy in mice. Mol. Ther., Aug 2016. [DOI:10.1038/mt.2016.168] [PubMed:27562586]. |
| [19] | K. Charton, L. Suel, S. F. Henriques, J. P. Moussu, M. Bovolenta, M. Taillepierre, C. Becker, K. Lipson, and I. Richard. Exploiting The CRISPR/CAS9 System to Study Alternative Splicing In Vivo: Application to Titin. Hum. Mol. Genet., Aug 2016. [DOI:10.1093/hmg/ddw280] [PubMed:27554112]. |
| [18] | N. Sabha, J. R. Volpatti, H. Gonorazky, A. Reifler, A. E. Davidson, X. Li, N. M. Eltayeb, C. Dall'Armi, G. Di Paolo, S. V. Brooks, A. Buj-Bello, E. L. Feldman, and J. J. Dowling. PIK3C2B inhibition improves function and prolongs survival in myotubular myopathy animal models. J. Clin. Invest., Aug 2016. [DOI:10.1172/JCI86841] [PubMed:27548528]. |
| [17] | J. Cosette, R. Ben Abdelwahed, S. Donnou-Triffault, C. Sautes-Fridman, P. Flaud, and S. Fisson. Bioluminescence-Based Tumor Quantification Method for Monitoring Tumor Progression and Treatment Effects in Mouse Lymphoma Models. J Vis Exp, (113), 2016. [DOI:10.3791/53609] [PubMed:27501019]. |
| [16] | O. W. Merten, M. Mezzina, and S. Fisson. Editorial (Thematic Issue: Proceedings from the EMBO Workshop: “Modern DNA Concepts and Tools for Safe Gene Transfer and Modificatio“). Curr Gene Ther, 16(3):153-155, 2016. [PubMed:27457888]. |
| [15] | E. Verhoeyen, S. Gomez, A. Galy, E. Ayuso, P. Midoux, M. Puceat, G. Vassaux, and P. Cordelier. Twelfth Annual Meeting of the French Society of Cell and Gene Therapy. Hum. Gene Ther., 27(7):555-558, Jul 2016. [DOI:10.1089/hum.2016.29031.eve] [PubMed:27428658]. |
| [14] | T. Aranyi, D. Stockholm, R. Yao, C. Poinsignon, T. Wiart, G. Corre, N. Touleimat, J. Tost, A. Galy, and A. Paldi. Systemic epigenetic response to recombinant lentiviral vectors independent of proviral integration. Epigenetics Chromatin, 9:29, 2016. [PubMed Central:PMC4940770] [DOI:10.1186/s13072-016-0077-1] [PubMed:27408621]. |
| [13] | J. Bennett, J. Wellman, K. A. Marshall, S. McCague, M. Ashtari, J. DiStefano-Pappas, O. U. Elci, D. C. Chung, J. Sun, J. F. Wright, D. R. Cross, P. Aravand, L. L. Cyckowski, J. L. Bennicelli, F. Mingozzi, A. Auricchio, E. A. Pierce, J. Ruggiero, B. P. Leroy, F. Simonelli, K. A. High, and A. M. Maguire. Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial. Lancet, Jun 2016. [DOI:10.1016/S0140-6736(16)30371-3] [PubMed:27375040]. |
| [12] | D. Israeli, J. Poupiot, F. Amor, K. Charton, W. Lostal, L. Jeanson-Leh, and I. Richard. Circulating miRNAs are generic and versatile therapeutic monitoring biomarkers in muscular dystrophies. Sci Rep, 6:28097, 2016. [DOI:10.1038/srep28097] [PubMed:27323895]. |
| [11] | N. Holic and D. Fenard. Production of Retrovirus-Based Vectors in Mildly Acidic pH Conditions. Methods Mol. Biol., 1448:41-48, 2016. [DOI:10.1007/978-1-4939-3753-0_3] [PubMed:27317171]. |
| [10] | O. Romano, C. Peano, G. M. Tagliazucchi, L. Petiti, V. Poletti, F. Cocchiarella, E. Rizzi, M. Severgnini, A. Cavazza, C. Rossi, P. Pagliaro, A. Ambrosi, G. Ferrari, S. Bicciato, G. De Bellis, F. Mavilio, and A. Miccio. Transcriptional, epigenetic and retroviral signatures identify regulatory regions involved in hematopoietic lineage commitment. Sci Rep, 6:24724, 2016. [PubMed Central:PMC4837375] [DOI:10.1038/srep24724] [PubMed:27095295]. |
| [9] | R. Mansour, S. Severin, J. M. Xuereb, M. P. Gratacap, J. Laporte, A. Buj-Bello, H. Tronchere, and B. Payrastre. Expression of myotubularins in blood platelets: Characterization and potential diagnostic of X-linked myotubular myopathy. Biochem. Biophys. Res. Commun., 476(3):167-173, Jul 2016. [DOI:10.1016/j.bbrc.2016.04.127] [PubMed:27155155]. |
| [8] | A. Cavazza, A. Miccio, O. Romano, L. Petiti, G. Malagoli Tagliazucchi, C. Peano, M. Severgnini, E. Rizzi, G. De Bellis, S. Bicciato, and F. Mavilio. Dynamic Transcriptional and Epigenetic Regulation of Human Epidermal Keratinocyte Differentiation. Stem Cell Reports, 6(4):618-632, Apr 2016. [PubMed Central:PMC4834057] [DOI:10.1016/j.stemcr.2016.03.003] [PubMed:27050947]. |
| [7] | M. Costopoulos, V. Touitou, J. L. Golmard, A. Darugar, S. Fisson, P. Bonnemye, M. L. Le Lez, C. Soussain, N. Cassoux, T. Lamy, P. Le Hoang, B. Bodaghi, H. Merle-Beral, and M. Le Garff-Tavernier. ISOLD: A New Highly Sensitive Interleukin Score for Intraocular Lymphoma Diagnosis. Ophthalmology, Mar 2016. [DOI:10.1016/j.ophtha.2016.01.037] [PubMed:26948307]. |
| [6] | I. Richard, J. Y. Hogrel, D. Stockholm, C. A. Payan, F. Fougerousse, B. Eymard, C. Mignard, A. Lopez de Munain, M. Fardeau, and J. A. Urtizberea. Natural history of LGMD2A for delineating outcome measures in clinical trials. Ann Clin Transl Neurol, 3(4):248-265, Apr 2016. [PubMed Central:PMC4818744] [DOI:10.1002/acn3.287] [PubMed:27081656]. |
| [5] | S. Gregoire, C. Terrada, G. H. Martin, G. Fourcade, A. Baeyens, G. Marodon, S. Fisson, F. Billiard, B. Lucas, R. Tadayoni, F. Behar-Cohen, B. Levacher, A. Galy, P. LeHoang, D. Klatzmann, B. Bodaghi, and B. L. Salomon. Treatment of Uveitis by In Situ Administration of Ex Vivo-Activated Polyclonal Regulatory T Cells. J. Immunol., 196(5):2109-2118, Mar 2016. [DOI:10.4049/jimmunol.1501723] [PubMed:26826251]. |
| [4] | G. Corre, M. Dessainte, J. B. Marteau, B. Dalle, D. Fenard, and A. Galy. "RCL-Pooling Assay": A Simplified Method for the Detection of Replication-Competent Lentiviruses in Vector Batches Using Sequential Pooling. Hum. Gene Ther., 27(2):202-210, Feb 2016. [DOI:10.1089/hum.2015.166] [PubMed:26886834]. |
| [3] | P. Rivera-Munoz, V. Abramowski, S. Jacquot, P. Andre, S. Charrier, K. Lipson-Ruffert, A. Fischer, A. Galy, M. Cavazzana, and J. P. de Villartay. Lymphopoiesis in transgenic mice over-expressing Artemis. Gene Ther., 23(2):176-186, Feb 2016. [DOI:10.1038/gt.2015.95] [PubMed:26361272]. |
| [2] | S. Majdoul, A. K. Seye, A. Kichler, N. Holic, A. Galy, B. Bechinger, and D. Fenard. Molecular Determinants of Vectofusin-1 and Its Derivatives for the Enhancement of Lentivirally Mediated Gene Transfer into Hematopoietic Stem/Progenitor Cells. J. Biol. Chem., 291(5):2161-2169, Jan 2016. [PubMed Central:PMC4732202] [DOI:10.1074/jbc.M115.675033] [PubMed:26668323]. |
| [1] | M. W. Lawlor, A. H. Beggs, A. Buj-Bello, M. K. Childers, J. J. Dowling, E. S. James, H. Meng, S. A. Moore, S. Prasad, B. Schoser, and C. A. Sewry. Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons. J. Neuropathol. Exp. Neurol., Jan 2016. [DOI:10.1093/jnen/nlv020] [PubMed:26823526]. |
| [29] | Richard, I., Hogrel, J-Y., Stockholm, D., Payan, C.A.M., Fougerousse, F., The calpainopathy study group, Eymard, B., Mignard, C., Lopez de Munain, A., Fardeau, M., Urtizberea, J-A. Natural history of LGMD2A indicates the need to select gender, mutation type and muscles to delineate the best outcome measures for clinical trials. Ann. Clin. Transl. Neurol.on press |
| [28] | A. Galy, G. Corre, M. Cavazzana, and S. Hacein-Bey-Abina. Efficacy and safety of gene therapy for Wiskott-Aldrich syndrome. Med Sci (Paris), 31(12):1066-1069, Dec 2015. [DOI:10.1051/medsci/20153112006] [PubMed:26672655]. |
| [27] | S. Majdoul, A. K. Seye, A. Kichler, N. Holic, A. Galy, B. Bechinger, and D. Fenard. Molecular determinants of Vectofusin-1 and its derivatives for the enhancement of lentiviral-mediated gene transfer into hematopoietic stem/progenitor cells. J. Biol. Chem., Dec 2015. [DOI:10.1074/jbc.M115.675033] [PubMed:26668323]. |
| [26] | J. Cosette, A. Moussy, F. Onodi, A. Auffret-Cariou, T. M. Neildez-Nguyen, A. Paldi, and D. Stockholm. Single Cell Dynamics Causes Pareto-Like Effect in Stimulated T Cell Populations. Sci Rep, 5:17756, 2015. [PubMed Central:PMC4673432] [DOI:10.1038/srep17756] [PubMed:26648396]. |
| [25] | M. K. Childers, A. H. Beggs, and A. Buj-Bello. Gene replacement rescues severe muscle pathology and prolongs survival in myotubularin-deficient mice and dogs. Ann Transl Med, 3(17):257, Oct 2015. [PubMed Central:PMC4620094] [DOI:10.3978/j.issn.2305-5839.2015.10.01] [PubMed:26605303]. |
| [24] | R. De Cid, R. Ben Yaou, C. Roudaut, K. Charton, S. Baulande, F. Leturcq, N. B. Romero, E. Malfatti, M. Beuvin, A. Vihola, A. Criqui, I. Nelson, J. Nectoux, L. Ben Aim, C. Caloustian, R. Olaso, B. Udd, G. Bonne, B. Eymard, and I. Richard. A new titinopathy: Childhood-juvenile onset Emery-Dreifuss-like phenotype without cardiomyopathy. Neurology, 85(24):2126-2135, Dec 2015. [DOI:10.1212/WNL.0000000000002200] [PubMed:26581302]. |
| [23] | D. J. Hui, S. C. Edmonson, G. M. Podsakoff, G. C. Pien, L. Ivanciu, R. M. Camire, H. Ertl, F. Mingozzi, K. A. High, and E. Basner-Tschakarjan. AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol Ther Methods Clin Dev, 2:15029, 2015. [PubMed Central:PMC4588448] [DOI:10.1038/mtm.2015.29] [PubMed:26445723]. |
| [22] | F. J. Molina-Estevez, A. Nowrouzi, M. Lozano, A. Galy, S. Charrier, C. V. Kalle, G. Guenechea, J. A. Bueren, and M. Schmidt. Lentiviral-Mediated Gene Therapy in Fanconi Anemia-A Mice Reveals Long-Term Engraftment and Continuous Turnover of Corrected HSCs. Curr Gene Ther, 15(6):550-562, 2015. [PubMed:26415575]. |
| [21] | P. Rivera-Munoz, V. Abramowski, S. Jacquot, P. Andre, S. Charrier, K. Lipson-Ruffert, A. Fischer, A. Galy, M. Cavazzana, and J. P. de Villartay. Lymphopoiesis in transgenic mice over-expressing Artemis. Gene Ther., Oct 2015. [DOI:10.1038/gt.2015.95] [PubMed:26361272]. |
| [20] | F. Boisgerault and F. Mingozzi. The Skeletal Muscle Environment and Its Role in Immunity and Tolerance to AAV Vector-Mediated Gene Transfer. Curr Gene Ther, 15(4):381-394, 2015. [PubMed Central:PMC4515578] [PubMed:26122097]. |
| [19] | C. J. Aalbers, L. Bevaart, S. Loiler, K. de Cortie, J. F. Wright, F. Mingozzi, P. P. Tak, and M. J. Vervoordeldonk. Preclinical Potency and Biodistribution Studies of an AAV 5 Vector Expressing Human Interferon-β (ART-I02) for Local Treatment of Patients with Rheumatoid Arthritis. PLoS ONE, 10(6):e0130612, 2015. [PubMed Central:PMC4479517] [DOI:10.1371/journal.pone.0130612] [PubMed:26107769]. |
| [18] | J. Rouillon, J. Poupiot, A. Zocevic, F. Amor, T. Leger, C. Garcia, J. M. Camadro, B. Wong, R. Pinilla, J. Cosette, A. M. Coenen-Stass, G. Mcclorey, T. C. Roberts, M. J. Wood, L. Servais, B. Udd, T. Voit, I. Richard, and F. Svinartchouk. Serum proteomic profiling reveals fragments of MYOM3 as potential biomarkers for monitoring the outcome of therapeutic interventions in muscular dystrophies. Hum. Mol. Genet., 24(17):4916-4932, Sep 2015. [PubMed Central:PMC4527491] [DOI:10.1093/hmg/ddv214] [PubMed:26060189]. |
| [17] | M. Pryadkina, W. Lostal, N. Bourg, K. Charton, C. Roudaut, M. L. Hirsch, and I. Richard. A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence. Mol Ther Methods Clin Dev, 2:15009, 2015. [PubMed Central:PMC4445010] [DOI:10.1038/mtm.2015.9] [PubMed:26029720]. |
| [16] | V. Poletti, A. Delli Carri, G. Malagoli Tagliazucchi, A. Faedo, L. Petiti, E. M. Mazza, C. Peano, G. De Bellis, S. Bicciato, A. Miccio, E. Cattaneo, and F. Mavilio. Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells. PLoS ONE, 10(5):e0126590, 2015. [PubMed Central:PMC4433211] [DOI:10.1371/journal.pone.0126590] [PubMed:25978676]. |
| [15] | S. Hacein-Bey Abina, H. B. Gaspar, J. Blondeau, L. Caccavelli, S. Charrier, K. Buckland, C. Picard, E. Six, N. Himoudi, K. Gilmour, A. M. McNicol, H. Hara, J. Xu-Bayford, C. Rivat, F. Touzot, F. Mavilio, A. Lim, J. M. Treluyer, S. Heritier, F. Lefrere, J. Magalon, I. Pengue-Koyi, G. Honnet, S. Blanche, E. A. Sherman, F. Male, C. Berry, N. Malani, F. D. Bushman, A. Fischer, A. J. Thrasher, A. Galy, and M. Cavazzana. Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. JAMA, 313(15):1550-1563, Apr 2015. [DOI:10.1001/jama.2015.3253] [PubMed:25898053]. |
| [14] | M. Ferrand, S. Da Rocha, G. Corre, A. Galy, and F. Boisgerault. Serotype-specific Binding Properties and Nanoparticle Characteristics Contribute to the Immunogenicity of rAAV1 Vectors. Mol. Ther., 23(6):1022-1033, Jun 2015. [DOI:10.1038/mt.2015.59] [PubMed:25881000]. |
| [13] | K. Charton, J. Sarparanta, A. Vihola, A. Milic, P. H. Jonson, L. Suel, H. Luque, I. Boumela, I. Richard, and B. Udd. CAPN3-mediated processing of C-terminal titin replaced by pathological cleavage in titinopathy. Hum. Mol. Genet., 24(13):3718-3731, Jul 2015. [DOI:10.1093/hmg/ddv116] [PubMed:25877298]. |
| [12] | F. Mingozzi and H. Buning. Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity. Front Immunol, 6:120, 2015. [PubMed Central:PMC4362342] [DOI:10.3389/fimmu.2015.00120] [PubMed:25852689]. |
| [11] | A. Meliani, C. Leborgne, S. Triffault, L. Jeanson-Leh, P. Veron, and F. Mingozzi. Determination of anti-adeno-associated virus vector neutralizing antibody titer with an in vitro reporter system. Hum Gene Ther Methods, 26(2):45-53, Apr 2015. [PubMed Central:PMC4403012] [DOI:10.1089/hgtb.2015.037] [PubMed:25819687]. |
| [10] | B. Marini, A. Kertesz-Farkas, H. Ali, B. Lucic, K. Lisek, L. Manganaro, S. Pongor, R. Luzzati, A. Recchia, F. Mavilio, M. Giacca, and M. Lusic. Nuclear architecture dictates HIV-1 integration site selection. Nature, 521(7551):227-231, May 2015. [DOI:10.1038/nature14226] [PubMed:25731161]. |
| [9] | S. Droz-Georget Lathion, A. Rochat, G. Knott, A. Recchia, D. Martinet, S. Benmohammed, N. Grasset, A. Zaffalon, N. Besuchet Schmutz, E. Savioz-Dayer, J. S. Beckmann, J. Rougemont, F. Mavilio, and Y. Barrandon. A single epidermal stem cell strategy for safe ex vivo gene therapy. EMBO Mol Med, 7(4):380-393, Apr 2015. [PubMed Central:PMC4403041] [DOI:10.15252/emmm.201404353] [PubMed:25724200]. |
| [8] | W. Mauhin, O. Lidove, E. Masat, F. Mingozzi, K. Mariampillai, J. M. Ziza, and O. Benveniste. Innate and Adaptive Immune Response in Fabry Disease. JIMD Rep, 22:1-10, 2015. [PubMed Central:PMC4486269] [DOI:10.1007/8904_2014_371] [PubMed:25690728]. |
| [7] | T. R. Cheever, D. Berkley, S. Braun, R. H. Brown, B. J. Byrne, J. S. Chamberlain, V. Cwik, D. Duan, H. J. Federoff, K. A. High, B. K. Kaspar, K. W. Klinger, J. Larkindale, J. Lincecum, F. Mavilio, C. L. McDonald, J. McLaughlin, B. Weiss McLeod, J. R. Mendell, G. Nuckolls, H. H. Stedman, D. A. Tagle, L. H. Vandenberghe, H. Wang, P. J. Wernett, J. M. Wilson, J. D. Porter, and A. K. Gubitz. Perspectives on best practices for gene therapy programs. Hum. Gene Ther., 26(3):127-133, Mar 2015. [PubMed Central:PMC4367233] [DOI:10.1089/hum.2014.147] [PubMed:25654329]. |
| [6] | N. Junge, F. Mingozzi, M. Ott, and U. Baumann. Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver. J. Pediatr. Gastroenterol. Nutr., 60(4):433-440, Apr 2015. [DOI:10.1097/MPG.0000000000000703] [PubMed:25594875]. |
| [5] | J. M. Crudele, J. D. Finn, J. I. Siner, N. B. Martin, G. P. Niemeyer, S. Zhou, F. Mingozzi, C. D. Lothrop, and V. R. Arruda. AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice. Blood, 125(10):1553-1561, Mar 2015. [PubMed Central:PMC4351503] [DOI:10.1182/blood-2014-07-588194] [PubMed:25568350]. |
| [4] | A. Ribera, V. Haurigot, M. Garcia, S. Marco, S. Motas, P. Villacampa, L. Maggioni, X. Leon, M. Molas, V. Sanchez, S. Munoz, C. Leborgne, X. Moll, M. Pumarola, F. Mingozzi, J. Ruberte, S. Anor, and F. Bosch. Biochemical, histological and functional correction of mucopolysaccharidosis type IIIB by intra-cerebrospinal fluid gene therapy. Hum. Mol. Genet., 24(7):2078-2095, Apr 2015. [DOI:10.1093/hmg/ddu727] [PubMed:25524704]. |
| [3] | S. O. Han, S. Li, E. D. Brooks, E. Masat, C. Leborgne, S. Banugaria, A. Bird, F. Mingozzi, H. Waldmann, and D. Koeberl. Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. Hum. Gene Ther., 26(1):26-35, Jan 2015. [PubMed Central:PMC4303018] [DOI:10.1089/hum.2014.115] [PubMed:25382056]. |
| [2] | J. Nectoux, R. de Cid, S. Baulande, F. Leturcq, J. A. Urtizberea, I. Penisson-Besnier, A. Nadaj-Pakleza, C. Roudaut, A. Criqui, L. Orhant, D. Peyroulan, R. Ben Yaou, I. Nelson, A. M. Cobo, M. C. Arne-Bes, E. Uro-Coste, P. Nitschke, M. Claustres, G. Bonne, N. Levy, J. Chelly, I. Richard, and M. Cossee. Detection of TRIM32 deletions in LGMD patients analyzed by a combined strategy of CGH array and massively parallel sequencing. Eur. J. Hum. Genet., 23(7):929-934, Jul 2015. [PubMed Central:PMC4463509] [DOI:10.1038/ejhg.2014.223] [PubMed:25351777]. |
| [1] | R. Ben Abdelwahed Bagga, S. Donnou, J. Cosette, C. Sautes-Fridman, M. Aouni, and S. Fisson. Mouse models of primary central nervous system lymphomas: tools for basing funding and therapeutic strategies. J. Neurooncol., 121(1):9-18, Jan 2015. [DOI:10.1007/s11060-014-1624-x] [PubMed:25300908]. |
| [17] | S. Cire, S. Da Rocha, R. Yao, S. Fisson, C. J. Buchholz, M. K. Collins, and A. Galy. Immunization of mice with lentiviral vectors targeted to MHC class II+ cells is due to preferential transduction of dendritic cells in vivo. PLoS ONE, 9(7):e101644, 2014. [PubMed Central:PMC4109917] [DOI:10.1371/journal.pone.0101644] [PubMed:25058148]. |
| [16] | A. Moiani, J. D. Suerth, F. Gandolfi, E. Rizzi, M. Severgnini, G. De Bellis, A. Schambach, and F. Mavilio. Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells. Genes (Basel), 5(2):415-429, 2014. [PubMed Central:PMC4094940] [DOI:10.3390/genes5020415] [PubMed:24840152]. |
| [15] | G. Corre, D. Stockholm, O. Arnaud, G. Kaneko, J. Vinuelas, Y. Yamagata, T. M. Neildez-Nguyen, J. J. Kupiec, G. Beslon, O. Gandrillon, and A. Paldi. Stochastic fluctuations and distributed control of gene expression impact cellular memory. PLoS ONE, 9(12):e115574, 2014. [PubMed Central:PMC4274012] [DOI:10.1371/journal.pone.0115574] [PubMed:25531401]. |
| [14] | G. Turchiano, M. C. Latella, A. Gogol-Doring, C. Cattoglio, F. Mavilio, Z. Izsvak, Z. Ivics, and A. Recchia. Genomic analysis of Sleeping Beauty transposon integration in human somatic cells. PLoS ONE, 9(11):e112712, 2014. [PubMed Central:PMC4229213] [DOI:10.1371/journal.pone.0112712] [PubMed:25390293]. |
| [13] | M. A. Goddard, E. Burlingame, A. H. Beggs, A. Buj-Bello, M. K. Childers, A. P. Marsh, and V. E. Kelly. Gait characteristics in a canine model of X-linked myotubular myopathy. J. Neurol. Sci., 346(1-2):221-226, Nov 2014. [PubMed Central:PMC4253544] [DOI:10.1016/j.jns.2014.08.032] [PubMed:25281397]. |
| [12] | A. Sarwal, M. S. Cartwright, F. O. Walker, E. Mitchell, A. Buj-Bello, A. H. Beggs, and M. K. Childers. Ultrasound assessment of the diaphragm: Preliminary study of a canine model of X-linked myotubular myopathy. Muscle Nerve, 50(4):607-609, Oct 2014. [PubMed Central:PMC4175136] [DOI:10.1002/mus.24294] [PubMed:24861988]. |
| [11] | T. M. Neildez-Nguyen, J. Bigot, S. Da Rocha, G. Corre, F. Boisgerault, A. Paldi, and A. Galy. Hypoxic culture conditions enhance the generation of regulatory T cells. Immunology, Sep 2014. [DOI:10.1111/imm.12388] [PubMed:25243909]. |
| [10] | N. Holic, A. K. Seye, S. Majdoul, S. Martin, O. W. Merten, A. Galy, and D. Fenard. Influence of mildly acidic pH conditions on the production of lentiviral and retroviral vectors. Hum Gene Ther Clin Dev, 25(3):178-185, Sep 2014. [DOI:10.1089/humc.2014.027] [PubMed:25073060]. |
| [9] | M. Ferrand, A. Galy, and F. Boisgerault. A dystrophic muscle broadens the contribution and activation of immune cells reacting to rAAV gene transfer. Gene Ther., 21(9):828-839, Sep 2014. [PubMed Central:PMC4283385] [DOI:10.1038/gt.2014.61] [PubMed:25030611]. |
| [8] | M. W. Lawlor, M. G. Viola, H. Meng, R. V. Edelstein, F. Liu, K. Yan, E. J. Luna, A. Lerch-Gaggl, R. G. Hoffmann, C. R. Pierson, A. Buj-Bello, J. L. Lachey, S. Pearsall, L. Yang, C. J. Hillard, and A. H. Beggs. Differential muscle hypertrophy is associated with satellite cell numbers and Akt pathway activation following activin type IIB receptor inhibition in Mtm1 p.R69C mice. Am. J. Pathol., 184(6):1831-1842, Jun 2014. [PubMed Central:PMC4044712] [DOI:10.1016/j.ajpath.2014.03.003] [PubMed:24726641]. |
| [7] | F. Monjaret, N. Bourg, L. Suel, C. Roudaut, F. Le Roy, I. Richard, and K. Charton. Cis-splicing and translation of the pre-trans-splicing molecule combine with efficiency in spliceosome-mediated RNA trans-splicing. Mol. Ther., 22(6):1176-1187, Jun 2014. [PubMed Central:PMC4048894] [DOI:10.1038/mt.2014.35] [PubMed:24618805]. |
| [6] | E. G. Rodriguez, R. Lefebvre, D. Bodnar, C. Legrand, P. Szentesi, J. Vincze, K. Poulard, J. Bertrand-Michel, L. Csernoch, A. Buj-Bello, and V. Jacquemond. Phosphoinositide substrates of myotubularin affect voltage-activated Ca2+ release in skeletal muscle. Pflugers Arch., 466(5):973-985, May 2014. [DOI:10.1007/s00424-013-1346-5] [PubMed:24022704]. |
| [5] | R. W. van Zwieten, S. Puttini, M. Lekka, G. Witz, E. Gicquel-Zouida, I. Richard, J. A. Lobrinus, F. Chevalley, H. Brune, G. Dietler, A. Kulik, T. Kuntzer, and N. Mermod. Assessing dystrophies and other muscle diseases at the nanometer scale by atomic force microscopy. Nanomedicine (Lond), 9(4):393-406, Apr 2014. [PubMed:24910872]. |
| [4] | F. Mavilio. Repairing without cutting: a safer alternative to gene correction? Mol. Ther., 22(4):690-691, Apr 2014. [PubMed Central:PMC3982485] [DOI:10.1038/mt.2014.40] [PubMed:24691177]. |
| [3] | A. Evila, A. Vihola, J. Sarparanta, O. Raheem, J. Palmio, S. Sandell, B. Eymard, I. Illa, R. Rojas-Garcia, K. Hankiewicz, L. Negrao, T. Lopponen, P. Nokelainen, M. Karppa, S. Penttila, M. Screen, T. Suominen, I. Richard, P. Hackman, and B. Udd. Atypical phenotypes in titinopathies explained by second titin mutations. Ann. Neurol., 75(2):230-240, Feb 2014. [DOI:10.1002/ana.24102] [PubMed:24395473]. |
| [2] | D. Ingrao, S. Majdoul, A. K. Seye, A. Galy, and D. Fenard. Concurrent measures of fusion and transduction efficiency of primary CD34+ cells with human immunodeficiency virus 1-based lentiviral vectors reveal different effects of transduction enhancers. Hum Gene Ther Methods, 25(1):48-56, Feb 2014. [DOI:10.1089/hgtb.2013.090] [PubMed:24152219]. |
| [1] | M. K. Childers, R. Joubert, K. Poulard, C. Moal, R. W. Grange, J. A. Doering, M. W. Lawlor, B. E. Rider, T. Jamet, N. Daniele, S. Martin, C. Riviere, T. Soker, C. Hammer, L. Van Wittenberghe, M. Lockard, X. Guan, M. Goddard, E. Mitchell, J. Barber, J. K. Williams, D. L. Mack, M. E. Furth, A. Vignaud, C. Masurier, F. Mavilio, P. Moullier, A. H. Beggs, and A. Buj-Bello. Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. Sci Transl Med, 6(220):220ra10, Jan 2014. [PubMed Central:PMC4105197] [DOI:10.1126/scitranslmed.3007523] [PubMed:24452262]. |
| [0] | G. Corre, D. Stockholm, and A. Paldi. Variabilité non-génétique dans les cultures cellulaires. In Culture de cellules animales (3e Éd.). Lavoisier, 2014. [link:Lavoisier]. |