Gene Correction by Homologous Recombination With Zinc Finger Nucleases in Primary Cells From a Mouse Model of a Generic Recessive Genetic Disease

Hacein-Bey-Abina, 2002, Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy, N Engl J Med, 346, 1185, 10.1056/NEJMoa012616 Aiuti, 2009, Gene therapy for immunodeficiency due to adenosine deaminase deficiency, N Engl J Med, 360, 447, 10.1056/NEJMoa0805817 Bainbridge, 2008, Effect of gene therapy on visual function in Leber's congenital amaurosis, N Engl J Med, 358, 2231, 10.1056/NEJMoa0802268 Maguire, 2009, Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial, Lancet, 374, 1597, 10.1016/S0140-6736(09)61836-5 Cartier, 2009, Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy, Science, 326, 818, 10.1126/science.1171242 Hacein-Bey-Abina, 2003, LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1, Science, 302, 415, 10.1126/science.1088547 Lombardo, 2007, Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery, Nat Biotechnol, 25, 1298, 10.1038/nbt1353 Porteus, 2005, Gene targeting using zinc finger nucleases, Nat Biotechnol, 23, 967, 10.1038/nbt1125 Doetschman, 1987, Targetted correction of a mutant HPRT gene in mouse embryonic stem cells, Nature, 330, 576, 10.1038/330576a0 Porteus, 2003, Chimeric nucleases stimulate gene targeting in human cells, Science, 300, 763, 10.1126/science.1078395 Donoho, 1998, Analysis of gene targeting and intrachromosomal homologous recombination stimulated by genomic double-strand breaks in mouse embryonic stem cells, Mol Cell Biol, 18, 4070, 10.1128/MCB.18.7.4070 Rouet, 1994, Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease, Mol Cell Biol, 14, 8096, 10.1128/MCB.14.12.8096 Choulika, 1995, Induction of homologous recombination in mammalian chromosomes by using the I-SceI system of Saccharomyces cerevisiae, Mol Cell Biol, 15, 1968, 10.1128/MCB.15.4.1968 Brenneman, 1996, Stimulation of intrachromosomal homologous recombination in human cells by electroporation with site-specific endonucleases, Proc Natl Acad Sci USA, 93, 3608, 10.1073/pnas.93.8.3608 Arnould, 2006, Engineering of large numbers of highly specific homing endonucleases that induce recombination on novel DNA targets, J Mol Biol, 355, 443, 10.1016/j.jmb.2005.10.065 Pâques, 2007, Meganucleases and DNA double-strand break-induced recombination: perspectives for gene therapy, Curr Gene Ther, 7, 49, 10.2174/156652307779940216 Ashworth, 2006, Computational redesign of endonuclease DNA binding and cleavage specificity, Nature, 441, 656, 10.1038/nature04818 Durai, 2005, Zinc finger nucleases: custom-designed molecular scissors for genome engineering of plant and mammalian cells, Nucleic Acids Res, 33, 5978, 10.1093/nar/gki912 Smith, 2000, Requirements for double-strand cleavage by chimeric restriction enzymes with zinc finger DNA-recognition domains, Nucleic Acids Res, 28, 3361, 10.1093/nar/28.17.3361 Bitinaite, 1998, FokI dimerization is required for DNA cleavage, Proc Natl Acad Sci USA, 95, 10570, 10.1073/pnas.95.18.10570 Cathomen, 2008, Zinc-finger nucleases: the next generation emerges, Mol Ther, 16, 1200, 10.1038/mt.2008.114 Bibikova, 2003, Enhancing gene targeting with designed zinc finger nucleases, Science, 300, 764, 10.1126/science.1079512 Carroll, 2008, Progress and prospects: zinc-finger nucleases as gene therapy agents, Gene Ther, 15, 1463, 10.1038/gt.2008.145 Doyon, 2008, Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases, Nat Biotechnol, 26, 702, 10.1038/nbt1409 Geurts, 2009, Knockout rats via embryo microinjection of zinc-finger nucleases, Science, 325, 433, 10.1126/science.1172447 Meng, 2008, Targeted gene inactivation in zebrafish using engineered zinc-finger nucleases, Nat Biotechnol, 26, 695, 10.1038/nbt1398 Morton, 2006, Induction and repair of zinc-finger nuclease-targeted double-strand breaks in Caenorhabditis elegans somatic cells, Proc Natl Acad Sci USA, 103, 16370, 10.1073/pnas.0605633103 Urnov, 2005, Highly efficient endogenous human gene correction using designed zinc-finger nucleases, Nature, 435, 646, 10.1038/nature03556 Shukla, 2009, Precise genome modification in the crop species Zea mays using zinc-finger nucleases, Nature, 459, 437, 10.1038/nature07992 Townsend, 2009, High-frequency modification of plant genes using engineered zinc-finger nucleases, Nature, 459, 442, 10.1038/nature07845 Hockemeyer, 2009, Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases, Nat Biotechnol, 27, 851, 10.1038/nbt.1562 Zou, 2009, Gene targeting of a disease-related gene in human induced pluripotent stem and embryonic stem cells, Cell Stem Cell, 5, 97, 10.1016/j.stem.2009.05.023 Perez, 2008, Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-finger nucleases, Nat Biotechnol, 26, 808, 10.1038/nbt1410 Zambrowicz, 1997, Disruption of overlapping transcripts in the ROSA beta geo 26 gene trap strain leads to widespread expression of beta-galactosidase in mouse embryos and hematopoietic cells, Proc Natl Acad Sci USA, 94, 3789, 10.1073/pnas.94.8.3789 Pruett-Miller, 2008, Comparison of zinc finger nucleases for use in gene targeting in mammalian cells, Mol Ther, 16, 707, 10.1038/mt.2008.20 Porteus, 2006, Mammalian gene targeting with designed zinc finger nucleases, Mol Ther, 13, 438, 10.1016/j.ymthe.2005.08.003 Maeder, 2008, Rapid “open-source” engineering of customized zinc-finger nucleases for highly efficient gene modification, Mol Cell, 31, 294, 10.1016/j.molcel.2008.06.016 Potts, 2006, Human SMC5/6 complex promotes sister chromatid homologous recombination by recruiting the SMC1/3 cohesin complex to double-strand breaks, EMBO J, 25, 3377, 10.1038/sj.emboj.7601218 Todaro, 1963, Quantitative studies of the growth of mouse embryo cells in culture and their development into established lines, J Cell Biol, 17, 299, 10.1083/jcb.17.2.299 Qiu, 1996, Implantation of autologous skin fibroblast genetically modified to secrete clotting factor IX partially corrects the hemorrhagic tendencies in two hemophilia B patients, Chin Med J, 109, 832 Roth, 2001, Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A, N Engl J Med, 344, 1735, 10.1056/NEJM200106073442301 Palmer, 1989, Production of human factor IX in animals by genetically modified skin fibroblasts: potential therapy for hemophilia B, Blood, 73, 438, 10.1182/blood.V73.2.438.438 Miller, 2007, An improved zinc-finger nuclease architecture for highly specific genome editing, Nat Biotechnol, 25, 778, 10.1038/nbt1319 Szczepek, 2007, Structure-based redesign of the dimerization interface reduces the toxicity of zinc-finger nucleases, Nat Biotechnol, 25, 786, 10.1038/nbt1317 Bibikova, 2002, Targeted chromosomal cleavage and mutagenesis in Drosophila using zinc-finger nucleases, Genetics, 161, 1169, 10.1093/genetics/161.3.1169 Lloyd, 2005, Targeted mutagenesis using zinc-finger nucleases in Arabidopsis, Proc Natl Acad Sci USA, 102, 2232, 10.1073/pnas.0409339102 Santiago, 2008, Targeted gene knockout in mammalian cells by using engineered zinc-finger nucleases, Proc Natl Acad Sci USA, 105, 5809, 10.1073/pnas.0800940105 Moehle, 2007, Targeted gene addition into a specified location in the human genome using designed zinc finger nucleases, Proc Natl Acad Sci USA, 104, 3055, 10.1073/pnas.0611478104 Miller, 2006, Gene targeting in vivo by adeno-associated virus vectors, Nat Biotechnol, 24, 1022, 10.1038/nbt1231 Hanna, 2007, Treatment of sickle cell anemia mouse model with iPS cells generated from autologous skin, Science, 318, 1920, 10.1126/science.1152092