, , 2015.
, Promoterless gene targeting without nucleases ameliorates haemophilia B in mice, Nature, vol.517, pp.360-364
An unbiased genome-wide analysis of zinc-finger nuclease specificity, Nat Biotechnol, vol.29, pp.816-823, 2011. ,
CRISPR/ Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse, 2016. ,
, EMBO Mol Med, vol.8, pp.477-488
Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human, Cancer Gene Ther, vol.15, pp.225-230, 2008. ,
In vivo genome editing restores haemostasis in a mouse model of haemophilia, Nature, vol.475, pp.217-221, 2011. ,
Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response, Nat Med, vol.12, pp.342-347, 2006. ,
, EMBO Molecular Medicine, vol.8, issue.5, 2016.
Homology-directed repair in rodent zygotes using Cas9 and TALEN engineered proteins, Sci Rep, vol.5, p.14410, 2015. ,
Longterm safety and efficacy of factor IX gene therapy in hemophilia B, N Engl J Med, vol.371, pp.1994-2004, 2014. ,
Improved genome editing efficiency and flexibility using modified oligonucleotides with TALEN and CRISPR-Cas9 nucleases, Cell Rep, vol.14, pp.2263-2272, 2016. ,
URL : https://hal.archives-ouvertes.fr/hal-01371505
Safety and efficacy of regional intravenous (RI) versus intramuscular (IM) delivery of rAAV1 and rAAV 1 and rAAV 8 to nonhuman primate skeletal muscle, Mol Ther, vol.16, pp.1291-1299, 2008. ,
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype, Nat Biotechnol, vol.32, pp.551-553, 2014. ,
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