T. Friedmann and R. Roblin, Gene Therapy for Human Genetic Disease?, Science, vol.175, issue.4025, pp.949-955, 1972.
DOI : 10.1126/science.175.4025.949

S. H. Orkin and A. G. Motulsky, Report and recommendations of the panel to assess the NIH investment in research on gene therapy, 1995.

A. Asokan, D. V. Schaffer, and R. J. Samulski, The AAV Vector Toolkit: Poised at the Clinical Crossroads, Molecular Therapy, vol.20, issue.4, pp.699-708, 2012.
DOI : 10.1038/mt.2011.287

R. Mann, R. C. Mulligan, and D. Baltimore, Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus, Cell, vol.33, issue.1, pp.153-159, 1983.
DOI : 10.1016/0092-8674(83)90344-6

L. Naldini, U. Blomer, P. Gallay, D. Ory, R. Mulligan et al., In Vivo Gene Delivery and Stable Transduction of Nondividing Cells by a Lentiviral Vector, Science, vol.272, issue.5259, pp.263-267, 1996.
DOI : 10.1126/science.272.5259.263

M. Cavazzana-calvo, S. Hacein-bey, G. De-saint-basile, F. Gross, E. Yvon et al., Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease, Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease, pp.669-672, 2000.
DOI : 10.1126/science.288.5466.669

A. Andolfi, F. Tabucchi, E. Carlucci, F. Marinello, S. Cattaneo et al., Correction of ADA- SCID by stem cell gene therapy combined with nonmyeloablative conditioning, Science, vol.296, pp.2410-2413, 2002.

S. Hacein-bey-abina, J. Hauer, A. Lim, C. Picard, G. P. Wang et al., Cavazzana-Calvo, Efficacy of gene therapy for X-linked severe combined immunodeficiency, N. Engl. J. Med, pp.363-355, 2010.

H. B. Gaspar, S. Cooray, K. C. Gilmour, K. L. Parsley, S. Adams et al., Long-Term Persistence of a Polyclonal T Cell Repertoire After Gene Therapy for X-Linked Severe Combined Immunodeficiency, Science Translational Medicine, vol.3, issue.97, p.79, 2011.
DOI : 10.1126/scitranslmed.3002715

A. Sorensen, P. Forster, J. I. Fraser, G. Cohen, I. De-saint-basile et al., Cavazzana-Calvo, LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1, Science, vol.302, pp.415-419, 2003.

S. I. Thornhill, A. Schambach, S. J. Howe, M. Ulaganathan, E. Grassman et al., Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined Immunodeficiency, Molecular Therapy, vol.16, issue.3, pp.590-598, 2008.
DOI : 10.1038/sj.mt.6300393

J. Blanche, J. Bleesing, H. Blondeau, K. F. De-boer, L. Buckland et al., A modified gamma-retrovirus vector for X-linked severe combined immunodeficiency, N. Engl. J. Med, pp.371-1407, 2014.

A. Aiuti, L. Biasco, S. Scaramuzza, F. Ferrua, M. P. Cicalese et al., Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome, Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome, p.1233151, 2013.
DOI : 10.1126/science.1233151

C. Xu-bayford, F. Rivat, F. Touzot, A. Mavilio, J. M. Lim et al., Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome, JAMA, vol.313, pp.1550-1563, 2015.

M. Blanche, E. Audit, P. Payen, B. Leboulch, P. Homme et al., Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy, Science, vol.326, pp.818-823, 2009.
URL : https://hal.archives-ouvertes.fr/cea-00817492

A. Biffi, C. C. Bartolomae, D. Cesana, N. Cartier, P. Aubourg et al., Lentiviral vector common integration sites in preclinical models and a clinical trial reflect a benign integration bias and not oncogenic selection, Blood, vol.117, issue.20, pp.5332-5339, 2011.
DOI : 10.1182/blood-2010-09-306761

R. Girot, G. J. Dorazio, A. Mulder, A. Polack, J. Bank et al., Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia, Nature, vol.467, pp.318-322, 2010.

L. Mingozzi, H. C. Couto, K. A. Ertl, M. A. High, and . Kay, Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response, Nat. Med, pp.12-342, 2006.

A. C. Nathwani, U. M. Reiss, E. G. Tuddenham, C. Rosales, P. Chowdary et al., Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B, New England Journal of Medicine, vol.371, issue.21, p.371, 1994.
DOI : 10.1056/NEJMoa1407309

S. G. Jacobson, A. V. Cideciyan, A. J. Roman, A. Sumaroka, S. B. Schwartz et al., Improvement and Decline in Vision with Gene Therapy in Childhood Blindness, New England Journal of Medicine, vol.372, issue.20, pp.372-1920, 2015.
DOI : 10.1056/NEJMoa1412965

J. W. Bainbridge, M. S. Mehat, V. Sundaram, S. J. Robbie, S. E. Barker et al., Long-Term Effect of Gene Therapy on Leber???s Congenital Amaurosis, New England Journal of Medicine, vol.372, issue.20, pp.372-1887, 2015.
DOI : 10.1056/NEJMoa1414221

A. F. Wright, Long-Term Effects of Retinal Gene Therapy in Childhood Blindness, New England Journal of Medicine, vol.372, issue.20, pp.1954-1955, 2015.
DOI : 10.1056/NEJMe1503419

L. Naldini, Gene therapy returns to centre stage, Nature, vol.32, issue.7573, pp.351-360, 2015.
DOI : 10.1038/nature15818

S. L. Maude, N. Frey, P. A. Shaw, R. Aplenc, D. M. Barrett et al., Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia, New England Journal of Medicine, vol.371, issue.16, pp.371-1507, 2014.
DOI : 10.1056/NEJMoa1407222

D. W. Lee, J. N. Kochenderfer, M. Stetler-stevenson, Y. K. Cui, C. Delbrook et al., T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial, Mackall, T cells expressing CD19 chimeric antigen receptors for acute lymphoblastic leukaemia in children and young adults: a phase 1 dose-escalation trial, pp.517-528, 2015.
DOI : 10.1016/S0140-6736(14)61403-3

J. A. Doudna and E. Charpentier, The new frontier of genome engineering with CRISPR-Cas9, Science, vol.346, issue.6213, p.1258096, 2014.
DOI : 10.1126/science.1258096

C. H. Levine and . June, Gene editing of CCR5 in autologous CD4 T cells of persons infected with HIV, N. Engl. J. Med, vol.370, pp.901-910, 2014.

C. Long, L. Amoasii, A. A. Mireault, J. R. Mcanally, H. Li et al., Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy, Science, vol.351, issue.6271, pp.400-403, 2016.
DOI : 10.1126/science.aad5725

P. Genovese, G. Schiroli, G. Escobar, T. Di-tomaso, C. Firrito et al., Targeted genome editing in human repopulating haematopoietic stem cells, Targeted genome editing in human repopulating haematopoietic stem cells, pp.235-240, 2014.
DOI : 10.1038/nature13420

H. Yin, C. Q. Song, J. R. Dorkin, L. J. Zhu, Y. Li et al., Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo, Nature Biotechnology, vol.33, issue.3, pp.34-328, 2016.
DOI : 10.1371/journal.pone.0108424

Y. Yang, L. Wang, P. Bell, D. Mcmenamin, Z. He et al., A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice, Nature Biotechnology, vol.246, issue.3, pp.34-334, 2016.
DOI : 10.1038/gt.2011.111