GaoX, TaoY, LamasV, et al.Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents. Nature, 2018; 553:217–221. DOI: 10.1038/nature25164.
2.
El RefaeyM, XuL, GaoY, et al.In vivo genome editing restores dystrophin expression and cardiac function in dystrophic mice. Circ Res, 2017; 121:923–929. DOI: 10.1161/circresaha.117.310996.
3.
GajT, OjalaDS, EkmanFK, et al.In vivo genome editing improves motor function and extends survival in a mouse model of ALS. Sci Adv, 2017; 3:eaar3952. DOI: 10.1126/sciadv.aar3952.
4.
KimK, ParkSW, KimJH, et al.Genome surgery using Cas9 ribonucleoproteins for the treatment of age-related macular degeneration. Genome Res, 2017; 27:419–426. DOI: 10.1101/gr.219089.116.
5.
StaahlBT, BenekareddyM, Coulon-BainierC, et al.Efficient genome editing in the mouse brain by local delivery of engineered Cas9 ribonucleoprotein complexes. Nat Biotechnol, 2017; 35:431–434. DOI: 10.1038/nbt.3806.
6.
WuW, LuZ, LiF, et al.Efficient in vivo gene editing using ribonucleoproteins in skin stem cells of recessive dystrophic epidermolysis bullosa mouse model. Proc Natl Acad Sci U S A, 2017; 114:1660–1665. DOI: 10.1073/pnas.1614775114.
7.
YinH, SongCQ, SureshS, et al.Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing. Nat Biotechnol, 2017; 35:1179–1187. DOI: 10.1038/nbt.4005.
8.
AskewC, RochatC, PanB, et al.Tmc gene therapy restores auditory function in deaf mice. Sci Transl Med, 2015; 7:295ra10–8. DOI: 10.1126/scitranslmed.aab1996.
9.
LentzJJ, JodelkaFM, HinrichAJ, et al.Rescue of hearing and vestibular function by antisense oligonucleotides in a mouse model of human deafness. Nat Med, 2013; 19:345–350. DOI: 10.1038/nm.3106).
10.
CharlesworthCT, DeshpandePS, DeverDP, et al.Identification of pre-existing adaptive immunity to Cas9 proteins in humans. biorXiv, 2018Jan5 [Epub ahead of print]; DOI: https://doi.org/10.1101/243345.
