ABOUT US

The liver gene transfer and immunology laboratory focuses on the development of in vivo therapies targeting hepatocytes with AAV vectors and on the study of immune responses in gene transfer. Our work combines the disciplines of virology and vector design, immunology, cell biology, and basic studies on inherited diseases to develop innovative therapies aim at establishing long-lasting correction of a variety of diseases including metabolic diseases and protein deficiencies. The research program in our lab integrates our experience in gene transfer and immunology to develop safe and effective gene therapies for diseases affecting pediatric and adult subjects.

Currently we are moving towards the initiation of a multicenter phase I/II clinical trial of AAV vector mediated liver gene transfer for Crigler-Najjar syndrome. This program is the result of the combined efforts of several laboratories and clinical centers in Europe in close collaboration with the Patients’ Organizations.

HIGHLIGHTS

A. Meliani, C. Leborgne et al.. 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:25819687].  

A. C. Nathwani et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N. Engl. J. Med., 371(21):1994-2004, Nov 2014. [PubMed:25409372].

F. Mingozzi et al. Overcoming preexisting humoral immunity to AAV using capsid decoys. Sci Transl Med, 5(194):194ra92, Jul 2013.[PubMed:23863832].      

F. Mingozzi and K. A. High. Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges. Nat. Rev. Genet., 12(5):341-355, May 2011. [PubMed:21499295].

F. Mingozzi et al. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat. Med., 13(4):419-422, Apr 2007. [PubMed:17369837].

F. Mingozzi et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Invest., 111(9):1347-1356, May 2003. [PubMed:12727926].

SUPPORT

• European Union - European Research Council Consolidator Grant MoMAAV.
• European Union - FP7-PEOPLE-2012-CIG. Marie Curie Career Integration Grant NosMod.
• European Union - H2020 Marie Curie project GLYCODIS3
• Bayer Hemophilia award program, Early Career Investigator Award
• DIM Biothérapies - Région Ile-de-France.