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References from Applications of Genome Editing Technology in Animal Disease Modeling and Gene Therapy. Local targets link to admitted publications; unresolved targets remain external evidence.
10.1038/nm.3793
10.1038/nm.3793 · External reference
Genome editing with engineered nucleases in plants
10.1093/pcp/pcu170 · 2014 · External reference
10.1038/nbt.3198
10.1038/nbt.3198 · External reference
10.1038/nbt.3190
10.1038/nbt.3190 · External reference
The anaphase promoting complex impacts repair choice by protecting ubiquitin signalling at DNA damage sites
10.1038/ncomms15751 · 2017 · External reference
Development and applications of CRISPR-Cas9 for genome engineering
10.1016/j.cell.2014.05.010 · 2014 · External reference
10.1038/nbt.2507
10.1038/nbt.2507 · External reference
10.1038/nbt.2170
10.1038/nbt.2170 · External reference
10.1126/science.1225829
10.1126/science.1225829 · External reference
10.1038/nbt.2501
10.1038/nbt.2501 · External reference
10.1016/j.cell.2013.04.025
10.1016/j.cell.2013.04.025 · External reference
Molecular imaging and cancer gene therapy
10.1038/cgt.2016.62 · 2016 · External reference
10.1016/j.biocel.2007.02.019
10.1016/j.biocel.2007.02.019 · External reference
Cancer modeling in the modern era: progress and challenges
10.1016/s0092-8674(02)00621-9 · 2002 · External reference
MLL gene fusions in human leukaemias: in vivo modelling to recapitulate these primary tumourigenic events
10.1007/s12185-007-0001-3 · 2007 · External reference
10.1038/ng.2256
10.1038/ng.2256 · External reference
10.1158/0008-5472.can-14-0529
10.1158/0008-5472.can-14-0529 · External reference
TALEN-mediated apc mutation in Xenopus tropicalis phenocopies familial adenomatous polyposis
10.18632/oncoscience.166 · 2015 · External reference
10.1038/nature13906
10.1038/nature13906 · External reference
10.1038/nature06030
10.1038/nature06030 · External reference
CRISPR-Cas9 knockin mice for genome editing and cancer modeling
10.1016/j.cell.2014.09.014 · 2014 · External reference
10.1038/nature13589
10.1038/nature13589 · External reference
10.1038/nature13902
10.1038/nature13902 · External reference
10.1038/ncomms4728
10.1038/ncomms4728 · External reference
Multiplexed pancreatic genome engineering and cancer induction by transfection-based CRISPR/Cas9 delivery in mice
10.1038/ncomms10770 · 2016 · External reference
10.1038/ncomms8391
10.1038/ncomms8391 · External reference
10.1038/nbt.2951
10.1038/nbt.2951 · External reference
Genome editing in cardiovascular diseases
10.1038/nrcardio.2016.139 · 2016 · External reference
10.1038/nature14580
10.1038/nature14580 · External reference
Klf2ash317 mutant Zebrafish do not recapitulate morpholino-induced vascular and haematopoietic phenotypes
10.1371/journal.pone.0141611 · 2015 · External reference
10.1111/gtc.12154
10.1111/gtc.12154 · External reference
Efficient multiple genome modifications induced by the crRNAs, tracrRNA and Cas9 protein complex in Zebrafish
10.1371/journal.pone.0128319 · 2015 · External reference
Single epicardial cell transcriptome sequencing identifies Caveolin 1 as an essential factor in zebrafish heart regeneration
2016 · External reference
10.7554/elife.09406
10.7554/elife.09406 · External reference
10.1073/pnas.1523918113
10.1073/pnas.1523918113 · External reference
10.1038/cr.2011.70
10.1038/cr.2011.70 · External reference
Generation of heterozygous fibrillin-1 mutant cloned pigs from genome-edited foetal fibroblasts
10.1038/srep24413 · 2016 · External reference
Molecular genetics of Meesmann's corneal dystrophy: ancestral and novel mutations in keratin 12 (K12) and complete sequence of the human KRT12 gene
10.1006/exer.1999.0769 · 2000 · External reference
10.1016/0092-8674(91)90284-6
10.1016/0092-8674(91)90284-6 · External reference
A novel GJA8 mutation (p.V44A) causing autosomal dominant congenital cataract
10.1371/journal.pone.0115406 · 2014 · External reference
Gap junctions or hemichannel-dependent and independent roles of connexins in cataractogenesis and lens development
10.2174/156652410793937750 · 2010 · External reference
Gene therapy in animal models of autosomal dominant retinitis pigmentosa
2012 · External reference
Hereditary optic neuropathies: from the mitochondria to the optic nerve
10.1016/j.ajo.2005.03.017 · 2005 · External reference
Severe congenital ocular coloboma
10.11604/pamj.2014.19.1.5025 · 2014 · External reference
A large novel deletion downstream of PAX6 gene in a Chinese family with ocular coloboma
10.1371/journal.pone.0083073 · 2013 · External reference
10.1016/j.ydbio.2015.02.012
10.1016/j.ydbio.2015.02.012 · External reference
CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts
10.1038/srep22024 · 2016 · External reference
CRISPR-engineered mosaicism rapidly reveals that loss of Kcnj13 function in mice mimics human disease phenotypes
10.1038/srep08366 · 2015 · External reference
PITX2 deficiency and associated human disease: insights from the zebrafish model
10.1093/hmg/ddy074 · 2018 · External reference
Protanopia (red color-blindness) in medaka: a simple system for producing color-blind fish and testing their spectral sensitivity
10.1186/s12863-017-0477-7 · 2017 · External reference
CRISPR/Cas9 mediated knockout of rb1 and rbl1 leads to rapid and penetrant retinoblastoma development in Xenopus tropicalis
10.1038/srep35264 · 2016 · External reference
Generation of obese rat model by transcription activator-like effector nucleases targeting the leptin receptor gene
10.1007/s11427-016-5049-y · 2016 · External reference
A viable hypomorphic Arnt2 mutation causes hyperphagic obesity, diabetes and hepatic steatosis
10.1242/dmm.035451 · 2018 · External reference
Generation of TALEN-mediated FH knockout rat model
10.18632/oncotarget.11429 · 2016 · External reference
10.1073/pnas.1211446109
10.1073/pnas.1211446109 · External reference
10.18632/oncotarget.17154
10.18632/oncotarget.17154 · External reference
Modeling Niemann-pick disease type C1 in zebrafish: a robust platform forin vivoscreening of candidate therapeutic compounds
10.1242/dmm.034165 · 2018 · External reference
Production of Wilson disease model rabbits with homology-directed precision point mutations in the ATP7B gene using the CRISPR/Cas9 system
10.1038/s41598-018-19774-4 · 2018 · External reference
NCKX3 was compensated by calcium transporting genes and bone resorption in a NCKX3 KO mouse model
10.1016/j.mce.2017.06.006 · 2017 · External reference
A zebrafish model of X-linked adrenoleukodystrophy recapitulates key disease features and demonstrates a developmental requirement for abcd1 in oligodendrocyte patterning and myelination
10.1093/hmg/ddx249 · 2017 · External reference
10.3727/096368914x678454
10.3727/096368914x678454 · External reference
10.3390/ijms17010026
10.3390/ijms17010026 · External reference
10.3727/096368912x655082
10.3727/096368912x655082 · External reference
10.1016/j.cell.2018.03.005
10.1016/j.cell.2018.03.005 · External reference
Modeling human epilepsy by TALEN targeting of mouse sodium channelScn8a
10.1002/dvg.22731 · 2014 · External reference
10.1371/journal.pone.0110371
10.1371/journal.pone.0110371 · External reference
A novel rabbit model of Duchenne muscular dystrophy generated by CRISPR/Cas9
10.1242/dmm.032201 · 2018 · External reference
10.1111/joim.12469
10.1111/joim.12469 · External reference
10.1016/j.cell.2017.04.035
10.1016/j.cell.2017.04.035 · External reference
10.1093/hmg/ddv120
10.1093/hmg/ddv120 · External reference
Novel genome-editing tools to model and correct primary immunodeficiencies
10.3389/fimmu.2015.00250 · 2015 · External reference
10.1016/j.stem.2016.06.003
10.1016/j.stem.2016.06.003 · External reference
10.1016/j.biocel.2013.10.010
10.1016/j.biocel.2013.10.010 · External reference
10.1073/pnas.1218721110
10.1073/pnas.1218721110 · External reference
Bioluminescent imaging of vaccinia virus infection in immunocompetent and immunodeficient rats as a model for human smallpox
10.1038/srep11397 · 2015 · External reference
Generation of a miniature pig disease model for human Laron syndrome
10.1038/srep15603 · 2015 · External reference
Generation of outbred Ace2 knockout mice by RNA transfection of TALENs displaying colitis reminiscent pathophysiology and inflammation
10.1007/s11248-014-9855-3 · 2014 · External reference
A viable mouse model for Netherton syndrome based on mosaic inactivation of the Spink5 gene
10.1515/hsz-2016-0194 · 2016 · External reference
10.1038/nature13420
10.1038/nature13420 · External reference
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1
10.1126/scitranslmed.aan0820 · 2017 · External reference
10.1038/nature20134
10.1038/nature20134 · External reference
10.1182/blood-2018-03-838540
10.1182/blood-2018-03-838540 · External reference
CRISPR/Cas9-mediated CCR5 ablation in human hematopoietic stem/progenitor cells confers HIV-1 resistance in vivo
10.1016/j.ymthe.2017.04.027 · 2017 · External reference
10.1016/j.cell.2018.05.013
10.1016/j.cell.2018.05.013 · External reference
10.1038/cr.2016.142
10.1038/cr.2016.142 · External reference
10.1056/nejmoa1300662
10.1056/nejmoa1300662 · External reference
10.1089/hum.2017.032
10.1089/hum.2017.032 · External reference
10.1126/science.358.6359.20
10.1126/science.358.6359.20 · External reference
10.1053/j.gastro.2017.03.014
10.1053/j.gastro.2017.03.014 · External reference
10.1074/jbc.m114.624999
10.1074/jbc.m114.624999 · External reference
Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C>T) mutation in β-thalassemia-derived iPSCs
10.1038/srep12065 · 2015 · External reference
10.1089/scd.2014.0347
10.1089/scd.2014.0347 · External reference
10.1016/j.stem.2015.07.001
10.1016/j.stem.2015.07.001 · External reference
10.3390/jcm4010159
10.3390/jcm4010159 · External reference
10.1038/nrg3763
10.1038/nrg3763 · External reference
10.1146/annurev-bioeng-071813-104938
10.1146/annurev-bioeng-071813-104938 · External reference
10.1161/circresaha.115.304351
10.1161/circresaha.115.304351 · External reference
The paradoxical functions of EGFR during breast cancer progression
2017 · External reference
10.1093/nar/gkx490
10.1093/nar/gkx490 · External reference
10.1016/j.ebiom.2018.02.015
10.1016/j.ebiom.2018.02.015 · External reference
10.1038/nrg2988
10.1038/nrg2988 · External reference
10.1038/nature10177
10.1038/nature10177 · External reference
CRISPR/Cas9-mediated genome editing via postnatal administration of AAV vector cures haemophilia B mice
10.1038/s41598-017-04625-5 · 2017 · External reference
10.1182/blood-2014-12-615492
10.1182/blood-2014-12-615492 · External reference
10.1038/nbt.3469
10.1038/nbt.3469 · External reference
10.1126/science.aau1549
10.1126/science.aau1549 · External reference
10.1016/j.ymthe.2018.03.002
10.1016/j.ymthe.2018.03.002 · External reference
10.1038/nbt.4219
10.1038/nbt.4219 · External reference
Single-dose CRISPR–Cas9 therapy extends lifespan of mice with Hutchinson–Gilford progeria syndrome
10.1038/s41591-019-0343-4 · 2019 · External reference
Development of a CRISPR/Cas9-based therapy for Hutchinson-Gilford progeria syndrome
10.1038/s41591-018-0338-6 · 2019 · External reference
10.1038/nbt.3471
10.1038/nbt.3471 · External reference
10.1021/acsnano.6b04261
10.1021/acsnano.6b04261 · External reference
10.1089/hum.2017.209
10.1089/hum.2017.209 · External reference
10.1038/nm.4313
10.1038/nm.4313 · External reference
10.1007/s11427-017-9032-4
10.1007/s11427-017-9032-4 · External reference
Exposing cancer with CRISPR-Cas9: from genetic identification to clinical therapy
10.21037/tcr.2018.06.16 · 2018 · External reference
10.1038/nbt.3437
10.1038/nbt.3437 · External reference
Increasing the specificity of CRISPR systems with engineered RNA secondary structures
2019 · External reference
10.1038/s41591-018-0137-0
10.1038/s41591-018-0137-0 · External reference
10.1038/nature24268
10.1038/nature24268 · External reference
10.1038/nature26155
10.1038/nature26155 · External reference
10.1038/s41586-018-0500-9
10.1038/s41586-018-0500-9 · External reference
10.1126/science.aav9023
10.1126/science.aav9023 · External reference
10.1038/s41591-018-0204-6
10.1038/s41591-018-0204-6 · External reference
10.1038/nmeth.3993
10.1038/nmeth.3993 · External reference
10.1089/hum.2006.17.253
10.1089/hum.2006.17.253 · External reference
10.1016/j.ymgme.2003.08.016
10.1016/j.ymgme.2003.08.016 · External reference
10.1016/j.cell.2015.09.038
10.1016/j.cell.2015.09.038 · External reference
10.1016/j.ymthe.2018.07.011
10.1016/j.ymthe.2018.07.011 · External reference
10.1007/s11427-017-9033-0
10.1007/s11427-017-9033-0 · External reference
