GENOME EDITING

 

 

 


Post Doctoral position available in Hematopoietic Stem Cell Gene therapy

(See application conditions)

ABOUT US

 

Our laboratory is interested in developing safe and effective genome editing and classical gene therapy approaches to ex vivo modify hematopoietic stem cells (HSC) for the treatment of rare genetic disorders.

 

The current projects are mainly focused on:

o Correcting hemoglobinopathies, in particular sickle-cell disease and β-thalassemia, by exploiting artificial nucleases to repair the underlying disease causing mutations or to reactivate fetal globin expression.

o Developing new gene therapy approaches for the treatment of clotting disorders, in particular hemophilia A.

o Developing alternative ways to exploit HSC as therapeutic delivery vehicles, due to the ability of their progeny to extensively home to many tissues, including the central nervous system.

o Developing new genetic approaches to provide a fitness advantage to genetically corrected HSC over the affected ones.

 

PhD student, postdoc, bioinformatician position might be available in our group. If you wish to apply, please send your application to mamendola@genethon.fr.

HIGHLIGHTS

 

S. Droz-Georget Lathion, A. Rochat, G. Knott, A. Recchia, D. Martinet et al. A single epidermal stem cell strategy for safe ex vivo gene therapy. EMBO Mol Med, 7(4):380-393, Apr 2015.[PubMed:25724200]

 

B. Marini, A. Kertesz-Farkas, H. Ali, B. Lucic, K. Lisek, L. Manganaro et al. Nuclear architecture dictates HIV-1 integration site selection. Nature, 521(7551):227-231, May 2015. [PubMed:25731161]

 

V. Poletti, A. Delli Carri, G. Malagoli Tagliazucchi, A. Faedo, L. Petiti, E. M. Mazza et al. Genome-Wide Definition of Promoter and Enhancer Usage during Neural Induction of Human Embryonic Stem Cells. PLoS ONE, 10(5):e0126590, 2015. [PubMed:25978676]

 

F. Mavilio. Repairing without cutting: a safer alternative to gene correction? Mol. Ther., 22(4):690-691, Apr 2014. [PubMed:24691177]

 

A. Coluccio, F. Miselli et al. Targeted gene addition in human epithelial stem cells by zinc-finger nuclease-mediated homologous recombination. Mol. Ther., 21(9):1695-1704, Sep 2013. [PubMed:23760447]

SUPPORT

  • ERC Advanced Grant: Gene Therapy of Skin Adhesion Diseases (GT-SKIN)
  • Fondagen grant (PI Amendola) Genopole, Evry