参考文献/References:
［1］ ISHINO Y,SHINAGAWA H,MAKINO K,AMEMURA M,NAKATA A.Nucleotide sequence of the iap gene,responsible for alkaline phosphatase isozyme conversion in Escherichia coli,and identification of the gene product［J］.J Bacteriol,1987,169（12）:5429-5433.
［2］ JANSEN R,EMBDEN J D,GAASTRA W,SCHOULS L M.Identification of genes that are associated with DNA repeats in prokaryotes［J］.Mol Microbiol,2002,43（6）:1565-1575.
［3］ BOLOTIN A,QUINQUIS B,SOROKIN A,EHRLICH S D.Clustered regularly interspaced short palindrome repeats （CRISPRs） have spacers of extrachromosomal origin［J］.Microbiology,2005,151（Pt 8）:2551-2561.
［4］ MOJICA F J,DIEZ-VILLASENOR C,GARCIA-MARTINEZ J,SORIA E.Intervening sequences of regularly spaced prokaryotic repeats derive from foreign genetic elements［J］.J Mol Evol,2005,60（2）:174-182.
［5］ POURCEL C,SALVIGNOL G,VERGNAUD G.CRISPR elements in Yersinia pestis acquire new repeats by preferential uptake of bacteriophage DNA,and provide additional tools for evolutionary studies［J］.Microbiology,2005,151（Pt 3）:653-663.
［6］ BARRANGOU R,FREMAUX C,DEVEAU H,RICHARDS M,BOYAVAL P,MOINEAU S.CRISPR provides acquired resistance against viruses in prokaryotes［J］.Science,2007,315（5819）:1709-1712.
［7］ MAKAROVA K S,WOLF Y I,ALKHNBASHI O S,COSTA F,SHAH S A,SAUNDERS S J,et al.An updated evolutionary classification of CRISPR-Cas systems［J］.Nat Rev Microbiol,2015,13（11）:722-736.
［8］ WILKINSON R,WIEDENHEFT B.A CRISPR method for genome engineering［J］.F1000Prime Reports,2014,6:3.
［9］ PLATT R J,CHEN S,ZHOU Y，YIM M J,SWIECH L,KEMPTON H R,et al.CRISPR-Cas9 knockin mice for genome editing and cancer modeling［J］.Cell,2014,159（2）:440-455.
［10］ MIZUNO S,DINH T T,KATO K，MIZUNO-IIJIMA S,TANIMOTO Y,DAITOKU Y,et al.Simple generation of albino C57BL/6J mice with G291T mutation in the tyrosinase gene by the CRISPR/Cas9 system［J］.Mamm Genome,2014,25（7-8）:327-334.
［11］ HAREL I,BENAYOUN B A,MACHADO B,SINGH P P,HU C K,PECH M F,et al.A platform for rapid exploration of aging and diseases in a naturally short-lived vertebrate［J］.Cell,2015,160（5）:1013-1026.
［12］ BENAVENTE C A,DYER M A.Genetics and epigenetics of human retinoblastoma［J］.Annu Rev Pathol,2015,10:547-562.
［13］ NAERT T,COLPAERT R,VAN NIEUWENHUYSEN T，DIMITRAKOPOULOU D,LEOEN J,HAUSTRAETE J,et al.CRISPR/Cas9 mediated knockout of rb1 and rbl1 leads to rapid and penetrant retinoblastoma development in Xenopus tropicalis［J］.Sci Rep,2016,6:35264.
［14］ REDDY M A,FRANCIS P J,BERRY V,BHATTACHARYA S S,MOORE A T.Molecular genetic basis of inherited cataract and associated phenotypes［J］.Surv Ophthalmol,2004,49（3）:300-315.
［15］ GONG X,CHENG C,XIA C H.Connexins in lens development and cataractogenesis［J］.J Membr Biol,2007,218（1-3）:9-12.
［16］ YUAN L,SUI T,CHEN M，DENG J,HUANG Y,ZENG J,et al.CRISPR/Cas9-mediated GJA8 knockout in rabbits recapitulates human congenital cataracts［J］.Sci Rep,2016,6:22024.
［17］ LITT M,KRAMER P,LAMORTICELLA D M,MURPHEY W,LOVRIEN E W,WELEBER R G.Autosomal dominant congenital cataract associated with a missense mutation in the human alpha crystallin gene CRYAA［J］.Hum Mol Genet,1998,7（3）:471-474.
［18］ JIAO X,KHAN S Y,IRUM B，KHAN A O,WANG Q,KABIR F,et al.Missense mutations in CRYAB are liable for recessive congenital cataracts［J］.PLoS One,2015,10（9）:e0137973.
［19］ YUAN L,YAO H,XU Y，CHEN M,DENG J,SONG Y,et al.CRISPR/Cas9-mediated mutation of alphaA-crystallin gene induces congenital cataracts in rabbits［J］.Invest Ophthalmol Vis Sci,2017,58（6）:BIO34-BIO41.
［20］ LONG C Z,MCANALLY J R,SHELTON J M,MIREAULT A A,BASSEL-DUBY R,OLSON E N.Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA［J］.Science,2014,345（6201）:1184-1188.
［21］ GUAN Y,MA Y,LI Q，SUN Z，MA L，WU L,et al.CRISPR/Cas9-mediated somatic correction of a novel coagulator factor IX gene mutation ameliorates hemophilia in mouse［J］.EMBO Mol Med,2016,8（5）:477-488.
［22］ YIN H,XUE W,CHEN S，BOGORAD R L，BENEDETTI E，GROMPE M,et al.Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype［J］.Nat Biotechnol,2014,32（6）:551-553.
［23］ YANG S,CHANG R,YANG H，ZHAO T，HONG Y，KONG H E,et al.CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington’s disease［J］.J Clin Invest,2017,127（7）:2719-2724.
［24］ HAMEL C.Retinitis pigmentosa［J］.Orphanet J Rare Dis,2006,1:40.
［25］ WU W H,TSAI Y T,JUSTUS S，LEE T T,ZHANG L,LIN C S,et al.CRISPR repair reveals causative mutation in a preclinical model of retinitis pigmentosa［J］.Mol Ther,2016,24（8）:1388-1394.
［26］ SUNG C H,CHUANG J Z.The cell biology of vision［J］.J Cell Biol,2010,190（6）:953-963.
［27］ BAKONDI B,LV W,LU B，JONES M K，TSAI Y，KIM K J,et al.In vivo CRISPR/Cas9 gene editing corrects retinal dystrophy in the s334ter-3 rat model of autosomal dominant retinitis pigmentosa［J］.Mol Ther,2016,24（3）:556-563.
［28］ GIANNELLI SG,LUONI M,CASTOLDI V，MASSIMINO L,CABASSI T,ANGELONI D,et al.Cas9/sgRNA selective targeting of the P23H Rhodopsin mutant allele for treating retinitis pigmentosa by intravitreal AAV9.PHP.B-based delivery［J］.Hum Mol Genet,2018,27（5）:761-779.
［29］ BASSUK A G,ZHENG A,LI Y,TSANG S H,MAHAJAN V B.Precision medicine:genetic repair of retinitis pigmentosa in patient-derived stem cells［J］.Sci Rep,2016,6:19969.
［30］ DEN HOLLANDER A I,KOENEKOOP R K,YZER S，LOPEZ I,ARENDS M L,VOESENEK K E,et al.Mutations in the CEP290 （NPHP6） gene are a frequent cause of Leber congenital amaurosis［J］.Am J Hum Genet,2006,79（3）:556-561.
［31］ RUAN G X,BARRY E,YU D,LUKASON M，CHENG S H，SCARIA A.CRISPR/Cas9-Mediated genome editing as a therapeutic approach for leber congenital amaurosis 10［J］.Mol Ther,2017,25（2）:331-341.
［32］ WIGGS J L,PASQUALE L R.Genetics of glaucoma［J］.Hum Mol Genet,2017,26（R1）:R21-R27.
［33］ JAIN A,ZODE G,KASETTI RB，RAN F A,YAN W,SHARMA T P,et al.CRISPR-Cas9-based treatment of myocilin-associated glaucoma［J］.Proc Natl Acad Sci USA,2017,114（42）:11199-11204.
［34］ ZHAO L,LI K,BAO S，ZHOU Y,LIANG Y,ZHAO G,et al.A 1-bp deletion in the gammaC-crystallin leads to dominant cataracts in mice［J］.Mamm Genome,2010,21（7-8）:361-369.
［35］ WU Y,LIANG D,WANG Y，BAI M,TANG W,BAO S,et al.Correction of a genetic disease in mouse via use of CRISPR-Cas9［J］.Cell Stem Cell,2013,13（6）:659-662.
［36］ JAMAL M,ULLAH A,AHSAN M,TYAGI R,HABIB Z,REHMAN K.Improving CRISPR-Cas9 On-Target Specificity［J］.Curr Issues Mol Biol,2018,26:65-80.