메뉴 건너뛰기




Volumn 10, Issue 1, 2015, Pages

CRISPR/Cas9: a powerful genetic engineering tool for establishing large animal models of neurodegenerative diseases

Author keywords

Animal model; CRISPR Cas9; Neurodegenerative diseases; Non human primates

Indexed keywords

TRANSCRIPTION ACTIVATOR LIKE EFFECTOR NUCLEASE; BACTERIAL PROTEIN; CRISPR ASSOCIATED PROTEIN; ENDONUCLEASE; GUIDE RNA; HYBRID PROTEIN;

EID: 84938537178     PISSN: None     EISSN: 17501326     Source Type: Journal    
DOI: 10.1186/s13024-015-0031-x     Document Type: Article
Times cited : (93)

References (86)
  • 1
    • 84868149287 scopus 로고    scopus 로고
    • Neddylation dysfunction in Alzheimer's disease
    • 1:CAS:528:DC%2BC3sXnt1Oruw%3D%3D 3484225 22805479
    • Chen Y, Neve RL, Liu H. Neddylation dysfunction in Alzheimer's disease. J Cell Mol Med. 2012;16(11):2583-91.
    • (2012) J Cell Mol Med , vol.16 , Issue.11 , pp. 2583-2591
    • Chen, Y.1    Neve, R.L.2    Liu, H.3
  • 2
    • 22244442489 scopus 로고    scopus 로고
    • The biochemistry of Parkinson's disease
    • 1:CAS:528:DC%2BD2MXpsVensrk%3D 15952880
    • Cookson MR. The biochemistry of Parkinson's disease. Annu Rev Biochem. 2005;74:29-52.
    • (2005) Annu Rev Biochem , vol.74 , pp. 29-52
    • Cookson, M.R.1
  • 5
    • 84886652249 scopus 로고    scopus 로고
    • Animal models of Parkinson's disease: vertebrate genetics
    • Lee Y, Dawson VL, Dawson TM. Animal models of Parkinson's disease: vertebrate genetics. Cold Spring Harb Perspect Med. 2012;2(10): doi: 10.1101/cshperspect.a009324.
    • (2012) Cold Spring Harb Perspect Med , vol.2 , Issue.10
    • Lee, Y.1    Dawson, V.L.2    Dawson, T.M.3
  • 6
    • 0027490598 scopus 로고
    • Molecular genetics of Huntington's disease
    • 1:STN:280:DyaK2c%2FivFKntQ%3D%3D 8215974
    • Gusella JF, MacDonald ME, Ambrose CM, Duyao MP. Molecular genetics of Huntington's disease. Arch Neurol. 1993;50(11):1157-63.
    • (1993) Arch Neurol , vol.50 , Issue.11 , pp. 1157-1163
    • Gusella, J.F.1    MacDonald, M.E.2    Ambrose, C.M.3    Duyao, M.P.4
  • 7
    • 0027176364 scopus 로고
    • The relationship between trinucleotide (CAG) repeat length and clinical features of Huntington's disease
    • 1:CAS:528:DyaK3sXlsFOmsL4%3D 8401589
    • Andrew SE, Goldberg YP, Kremer B, Telenius H, Theilmann J, Adam S, et al. The relationship between trinucleotide (CAG) repeat length and clinical features of Huntington's disease. Nat Genet. 1993;4(4):398-403.
    • (1993) Nat Genet , vol.4 , Issue.4 , pp. 398-403
    • Andrew, S.E.1    Goldberg, Y.P.2    Kremer, B.3    Telenius, H.4    Theilmann, J.5    Adam, S.6
  • 9
    • 79952233317 scopus 로고    scopus 로고
    • Genetic rodent models of amyotrophic lateral sclerosis
    • Van Den Bosch L. Genetic rodent models of amyotrophic lateral sclerosis. J Biomed Biotechnol. 2011;2011:348765.
    • (2011) J Biomed Biotechnol , vol.2011 , pp. 348765
    • Van Den Bosch, L.1
  • 10
    • 84897843931 scopus 로고    scopus 로고
    • Rodent models of chemotherapy-induced peripheral neuropathy
    • 24615440
    • Hoke A, Ray M. Rodent models of chemotherapy-induced peripheral neuropathy. ILAR J. 2014;54(3):273-81.
    • (2014) ILAR J , vol.54 , Issue.3 , pp. 273-281
    • Hoke, A.1    Ray, M.2
  • 11
    • 84901837074 scopus 로고    scopus 로고
    • Zebrafish models of human motor neuron diseases: Advantages and limitations
    • 1:CAS:528:DC%2BC2cXmvVWrtbw%3D
    • Babin PJ, Goizet C, Raldua D. Zebrafish models of human motor neuron diseases: Advantages and limitations. Progr Neurobiol. 2014;118:36-58.
    • (2014) Progr Neurobiol , vol.118 , pp. 36-58
    • Babin, P.J.1    Goizet, C.2    Raldua, D.3
  • 12
    • 0036637271 scopus 로고    scopus 로고
    • Animal models of neurological deficits: how relevant is the rat?
    • Angela Cenci M, Whishaw IQ, Schallert T. Animal models of neurological deficits: how relevant is the rat? Nature. 2002;3:6.
    • (2002) Nature , vol.3 , pp. 6
    • Angela Cenci, M.1    Whishaw, I.Q.2    Schallert, T.3
  • 13
    • 84938537486 scopus 로고    scopus 로고
    • Large Animal Models of Huntington's Disease
    • 24048953
    • Li XJ, Li S. Large Animal Models of Huntington's Disease. Curr Top Behav Neurosci. 2015;22:149-60.
    • (2015) Curr Top Behav Neurosci. , vol.22 , pp. 149-160
    • Li, X.J.1    Li, S.2
  • 14
    • 0023181439 scopus 로고
    • Molecular genetics and Huntington's disease. The South African situation
    • 1:STN:280:DyaL2s3jsVKquw%3D%3D 2954228
    • Hayden MR, Goldblatt J, Wallis G, Winship IM, Beighton P. Molecular genetics and Huntington's disease. The South African situation. S Afr Med J. 1987;71(11):683-6.
    • (1987) S Afr Med J , vol.71 , Issue.11 , pp. 683-686
    • Hayden, M.R.1    Goldblatt, J.2    Wallis, G.3    Winship, I.M.4    Beighton, P.5
  • 15
    • 79551655290 scopus 로고    scopus 로고
    • Huntington's disease: can mice lead the way to treatment?
    • 1:CAS:528:DC%2BC3MXhvFertrw%3D 21315254
    • Crook ZR, Housman D. Huntington's disease: can mice lead the way to treatment? Neuron. 2011;69(3):423-35.
    • (2011) Neuron , vol.69 , Issue.3 , pp. 423-435
    • Crook, Z.R.1    Housman, D.2
  • 16
    • 84862663712 scopus 로고    scopus 로고
    • Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis
    • 1:CAS:528:DC%2BC38XptVKgtbs%3D 3383626 22726834
    • Kordasiewicz HB, Stanek LM, Wancewicz EV, Mazur C, McAlonis MM, Pytel KA, et al. Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis. Neuron. 2012;74(6):1031-44.
    • (2012) Neuron , vol.74 , Issue.6 , pp. 1031-1044
    • Kordasiewicz, H.B.1    Stanek, L.M.2    Wancewicz, E.V.3    Mazur, C.4    McAlonis, M.M.5    Pytel, K.A.6
  • 17
    • 84938501911 scopus 로고    scopus 로고
    • Characterization of progressive motor deficits in mice transgenic for the human Huntington's disease mutation
    • Mangiarini L, Sathasivam K, Seller M, Cozens B, Harper A, Hetherington C, et al. Characterization of progressive motor deficits in mice transgenic for the human Huntington's disease mutation. Cell. 1996;87:14.
    • (1996) Cell , vol.87 , pp. 14
    • Mangiarini, L.1    Sathasivam, K.2    Seller, M.3    Cozens, B.4    Harper, A.5    Hetherington, C.6
  • 18
    • 0033560924 scopus 로고    scopus 로고
    • Characterization of progressive motor deficits in mice transgenic for the human Huntington's disease mutation
    • 1:CAS:528:DyaK1MXitlGmsro%3D 10191337
    • Carter RJ, Lione LA, Humby T, Mangiarini L, Mahal A, Bates GP, et al. Characterization of progressive motor deficits in mice transgenic for the human Huntington's disease mutation. J Neurosci. 1999;19(8):3248-57.
    • (1999) J Neurosci , vol.19 , Issue.8 , pp. 3248-3257
    • Carter, R.J.1    Lione, L.A.2    Humby, T.3    Mangiarini, L.4    Mahal, A.5    Bates, G.P.6
  • 19
    • 10744227174 scopus 로고    scopus 로고
    • Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease
    • 1:CAS:528:DC%2BD3sXkvVSmtr0%3D 12812983
    • Slow EJ, van Raamsdonk J, Rogers D, Coleman SH, Graham RK, Deng Y, et al. Selective striatal neuronal loss in a YAC128 mouse model of Huntington disease. Hum Mol Genet. 2003;12(13):1555-67.
    • (2003) Hum Mol Genet , vol.12 , Issue.13 , pp. 1555-1567
    • Slow, E.J.1    Van Raamsdonk, J.2    Rogers, D.3    Coleman, S.H.4    Graham, R.K.5    Deng, Y.6
  • 20
    • 46749157501 scopus 로고    scopus 로고
    • Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice
    • 1:CAS:528:DC%2BD1cXns1ygu7Y%3D 2630800 18550760
    • Gray M, Shirasaki DI, Cepeda C, Andre VM, Wilburn B, Lu XH, et al. Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci. 2008;28(24):6182-95.
    • (2008) J Neurosci , vol.28 , Issue.24 , pp. 6182-6195
    • Gray, M.1    Shirasaki, D.I.2    Cepeda, C.3    Andre, V.M.4    Wilburn, B.5    Lu, X.H.6
  • 21
    • 0032938295 scopus 로고    scopus 로고
    • Length-dependent gametic CAG repeat instability in the Huntington's disease knock-in mouse
    • 1:CAS:528:DyaK1MXoslCmsA%3D%3D 9887339
    • Wheeler VC, Auerbach W, White JK, Srinidhi J, Auerbach A, Ryan A, et al. Length-dependent gametic CAG repeat instability in the Huntington's disease knock-in mouse. Hum Mol Genet. 1999;8(1):115-22.
    • (1999) Hum Mol Genet , vol.8 , Issue.1 , pp. 115-122
    • Wheeler, V.C.1    Auerbach, W.2    White, J.K.3    Srinidhi, J.4    Auerbach, A.5    Ryan, A.6
  • 22
    • 0034163497 scopus 로고    scopus 로고
    • Long glutamine tracts cause nuclear localization of a novel form of huntingtin in medium spiny striatal neurons in HdhQ92 and HdhQ111 knock-in mice
    • 1:CAS:528:DC%2BD3cXhvVGntLc%3D 10699173
    • Wheeler VC, White JK, Gutekunst CA, Vrbanac V, Weaver M, Li XJ, et al. Long glutamine tracts cause nuclear localization of a novel form of huntingtin in medium spiny striatal neurons in HdhQ92 and HdhQ111 knock-in mice. Hum Mol Genet. 2000;9(4):503-13.
    • (2000) Hum Mol Genet , vol.9 , Issue.4 , pp. 503-513
    • Wheeler, V.C.1    White, J.K.2    Gutekunst, C.A.3    Vrbanac, V.4    Weaver, M.5    Li, X.J.6
  • 23
    • 0033054555 scopus 로고    scopus 로고
    • Intranuclear inclusions and neuritic aggregates in transgenic mice expressing a mutant N-terminal fragment of huntingtin
    • 1:CAS:528:DyaK1MXhslKnsb0%3D 9949199
    • Schilling G, Becher MW, Sharp AH, Jinnah HA, Duan K, Kotzuk JA, et al. Intranuclear inclusions and neuritic aggregates in transgenic mice expressing a mutant N-terminal fragment of huntingtin. Hum Mol Genet. 1999;8(3):397-407.
    • (1999) Hum Mol Genet , vol.8 , Issue.3 , pp. 397-407
    • Schilling, G.1    Becher, M.W.2    Sharp, A.H.3    Jinnah, H.A.4    Duan, K.5    Kotzuk, J.A.6
  • 24
    • 0033136692 scopus 로고    scopus 로고
    • A YAC mouse model for Huntington's disease with full-length mutant huntingtin, cytoplasmic toxicity, and selective striatal neurodegeneration
    • 1:CAS:528:DyaK1MXjvVSjurw%3D 10402204
    • Hodgson JG, Agopyan N, Gutekunst C-A, Leavitt BR, LePiane F, Singaraja R, et al. A YAC mouse model for Huntington's disease with full-length mutant huntingtin, cytoplasmic toxicity, and selective striatal neurodegeneration. Neuron. 1999;23(1):181-92.
    • (1999) Neuron , vol.23 , Issue.1 , pp. 181-192
    • Hodgson, J.G.1    Agopyan, N.2    Gutekunst, C.-A.3    Leavitt, B.R.4    LePiane, F.5    Singaraja, R.6
  • 25
    • 77957732707 scopus 로고    scopus 로고
    • Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs
    • 1:CAS:528:DC%2BC3cXht1eqtLzF 2947404 20660116
    • Yang D, Wang CE, Zhao B, Li W, Ouyang Z, Liu Z, et al. Expression of Huntington's disease protein results in apoptotic neurons in the brains of cloned transgenic pigs. Hum Mol Genet. 2010;19(20):3983-94.
    • (2010) Hum Mol Genet , vol.19 , Issue.20 , pp. 3983-3994
    • Yang, D.1    Wang, C.E.2    Zhao, B.3    Li, W.4    Ouyang, Z.5    Liu, Z.6
  • 27
    • 0035543870 scopus 로고    scopus 로고
    • Production of transgenic miniature pigs by pronuclear microinjection
    • 1:CAS:528:DC%2BD38XmtlSksQ%3D%3D 11817545
    • Uchida M, Shimatsu Y, Onoe K, Matsuyama N, Niki R, Ikeda JE, et al. Production of transgenic miniature pigs by pronuclear microinjection. Transgenic Res. 2001;10(6):577-82.
    • (2001) Transgenic Res , vol.10 , Issue.6 , pp. 577-582
    • Uchida, M.1    Shimatsu, Y.2    Onoe, K.3    Matsuyama, N.4    Niki, R.5    Ikeda, J.E.6
  • 30
    • 84923225433 scopus 로고    scopus 로고
    • Update on Huntington's disease: advances in care and emerging therapeutic options
    • 10.1016/j.parkreldis.2014 25572500
    • Zielonka D, Mielcarek M, Landwehrmeyer GB. Update on Huntington's disease: advances in care and emerging therapeutic options. Parkinsonism Relat Disord. 2015;21(3):169-78. doi: 10.1016/j.parkreldis.2014.
    • (2015) Parkinsonism Relat Disord , vol.21 , Issue.3 , pp. 169-178
    • Zielonka, D.1    Mielcarek, M.2    Landwehrmeyer, G.B.3
  • 31
    • 84868538087 scopus 로고    scopus 로고
    • Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice
    • 1:CAS:528:DC%2BC38Xhslyiu7fP 3494930 23054839
    • Garriga-Canut M, Agustin-Pavon C, Herrmann F, Sanchez A, Dierssen M, Fillat C, et al. Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6/2 mice. Proc Natl Acad Sci U S A. 2012;109(45):E3136-45.
    • (2012) Proc Natl Acad Sci U S A , vol.109 , Issue.45 , pp. E3136-E3145
    • Garriga-Canut, M.1    Agustin-Pavon, C.2    Herrmann, F.3    Sanchez, A.4    Dierssen, M.5    Fillat, C.6
  • 32
    • 0023869802 scopus 로고
    • Amyotrophic lateral sclerosis. Recent advances in pathogenesis and therapeutic trials
    • 1:STN:280:DyaL1c7jtlCnsQ%3D%3D 3277599
    • Mitsumoto H, Hanson MR, Chad DA. Amyotrophic lateral sclerosis. Recent advances in pathogenesis and therapeutic trials. Arch Neurol. 1988;45(2):189-202.
    • (1988) Arch Neurol , vol.45 , Issue.2 , pp. 189-202
    • Mitsumoto, H.1    Hanson, M.R.2    Chad, D.A.3
  • 33
    • 0030825472 scopus 로고    scopus 로고
    • Therapeutic advances in amyotrophic lateral sclerosis
    • 1:CAS:528:DyaK2sXktFCrur4%3D 9226998
    • Louvel E, Hugon J, Doble A. Therapeutic advances in amyotrophic lateral sclerosis. Trends Pharmacol Sci. 1997;18(6):196-203.
    • (1997) Trends Pharmacol Sci , vol.18 , Issue.6 , pp. 196-203
    • Louvel, E.1    Hugon, J.2    Doble, A.3
  • 34
    • 84879389114 scopus 로고    scopus 로고
    • Amyotrophic lateral sclerosis: update on etiological treatment
    • 23773350
    • Ayach L, Curti C, Montana M, Pisano P, Vanelle P. Amyotrophic lateral sclerosis: update on etiological treatment. Therapie. 2013;68(2):93-106.
    • (2013) Therapie , vol.68 , Issue.2 , pp. 93-106
    • Ayach, L.1    Curti, C.2    Montana, M.3    Pisano, P.4    Vanelle, P.5
  • 35
    • 80051551180 scopus 로고    scopus 로고
    • SOD1 and TDP-43 animal models of amyotrophic lateral sclerosis: recent advances in understanding disease toward the development of clinical treatments
    • 1:CAS:528:DC%2BC3MXhtVSitbnP 21706386
    • Joyce PI, Fratta P, Fisher EMC, Acevedo-Arozena A. SOD1 and TDP-43 animal models of amyotrophic lateral sclerosis: recent advances in understanding disease toward the development of clinical treatments. Mamm Genome. 2011;22(7-8):420-48.
    • (2011) Mamm Genome , vol.22 , Issue.7-8 , pp. 420-448
    • Joyce, P.I.1    Fratta, P.2    Fisher, E.M.C.3    Acevedo-Arozena, A.4
  • 36
    • 77953902544 scopus 로고    scopus 로고
    • Understanding the molecular mechanisms of Friedreich Ataxia to develop therapeutic approaches
    • Schmucker S, Puccio H. Understanding the molecular mechanisms of Friedreich Ataxia to develop therapeutic approaches. Hum Mol Genet. 2010;19(R1):R103-10
    • (2010) Hum Mol Genet , vol.19 , Issue.R1 , pp. R103-R110
    • Schmucker, S.1    Puccio, H.2
  • 37
    • 33749632259 scopus 로고    scopus 로고
    • Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis
    • 1:CAS:528:DC%2BD28XhtVCiurrL 17023659
    • Neumann M, Sampathu DM, Kwong LK, Truax AC, Micsenyi MC, Chou TT, et al. Ubiquitinated TDP-43 in frontotemporal lobar degeneration and amyotrophic lateral sclerosis. Science. 2006;314(5796):130-3.
    • (2006) Science , vol.314 , Issue.5796 , pp. 130-133
    • Neumann, M.1    Sampathu, D.M.2    Kwong, L.K.3    Truax, A.C.4    Micsenyi, M.C.5    Chou, T.T.6
  • 38
    • 84896730248 scopus 로고    scopus 로고
    • Human stem cell models of neurodegeneration: a novel approach to study mechanisms of disease development
    • 1:CAS:528:DC%2BC3sXhvFCisb%2FN 24306942
    • Hargus G, Ehrlich M, Hallmann AL, Kuhlmann T. Human stem cell models of neurodegeneration: a novel approach to study mechanisms of disease development. Acta Neuropathol. 2014;127(2):151-73.
    • (2014) Acta Neuropathol , vol.127 , Issue.2 , pp. 151-173
    • Hargus, G.1    Ehrlich, M.2    Hallmann, A.L.3    Kuhlmann, T.4
  • 39
    • 84898033899 scopus 로고    scopus 로고
    • Species-dependent neuropathology in transgenic SOD1 pigs
    • 1:CAS:528:DC%2BC2cXjvV2hsro%3D 3975503 24577199
    • Yang H, Wang G, Sun H, Shu R, Liu T, Wang CE, et al. Species-dependent neuropathology in transgenic SOD1 pigs. Cell Res. 2014;24(4):464-81.
    • (2014) Cell Res , vol.24 , Issue.4 , pp. 464-481
    • Yang, H.1    Wang, G.2    Sun, H.3    Shu, R.4    Liu, T.5    Wang, C.E.6
  • 41
    • 0035516124 scopus 로고    scopus 로고
    • From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS
    • 1:CAS:528:DC%2BD38Xmt1WitLs%3D 11715057
    • Cleveland DW, Rothstein JD. From Charcot to Lou Gehrig: deciphering selective motor neuron death in ALS. Nat Rev Neurosci. 2001;2(11):806-19.
    • (2001) Nat Rev Neurosci , vol.2 , Issue.11 , pp. 806-819
    • Cleveland, D.W.1    Rothstein, J.D.2
  • 42
    • 84857055792 scopus 로고    scopus 로고
    • Mitochondria in motor nerve terminals: function in health and in mutant superoxide dismutase 1 mouse models of familial ALS
    • 1:CAS:528:DC%2BC3MXhs1SgsLnI 3237816 22089637
    • Barrett EF, Barrett JN, David G. Mitochondria in motor nerve terminals: function in health and in mutant superoxide dismutase 1 mouse models of familial ALS. J Bioenerg Biomembr. 2011;43(6):581-6.
    • (2011) J Bioenerg Biomembr , vol.43 , Issue.6 , pp. 581-586
    • Barrett, E.F.1    Barrett, J.N.2    David, G.3
  • 43
    • 84865968137 scopus 로고    scopus 로고
    • Object recognition as a measure of memory in 1-2 years old transgenic minipigs carrying the APPsw mutation for Alzheimer's disease
    • 1:CAS:528:DC%2BC38XhslWkt7bN 22661126
    • Sondergaard LV, Ladewig J, Dagnaes-Hansen F, Herskin MS, Holm IE. Object recognition as a measure of memory in 1-2 years old transgenic minipigs carrying the APPsw mutation for Alzheimer's disease. Transgenic Res. 2012;21(6):1341-8.
    • (2012) Transgenic Res , vol.21 , Issue.6 , pp. 1341-1348
    • Sondergaard, L.V.1    Ladewig, J.2    Dagnaes-Hansen, F.3    Herskin, M.S.4    Holm, I.E.5
  • 44
    • 0035847075 scopus 로고    scopus 로고
    • Transgenic monkeys produced by retroviral gene transfer into mature oocytes
    • 1:CAS:528:DC%2BD3MXktlKmtA%3D%3D 11209082
    • Chan AWS, Chong KY, Martinovich C, Simerly C, Schatten G. Transgenic monkeys produced by retroviral gene transfer into mature oocytes. Science. 2001;291(5502):309-12.
    • (2001) Science , vol.291 , Issue.5502 , pp. 309-312
    • Chan, A.W.S.1    Chong, K.Y.2    Martinovich, C.3    Simerly, C.4    Schatten, G.5
  • 45
    • 0035350713 scopus 로고    scopus 로고
    • Transgenic monkey raises hope for primate models of human diseases
    • 1:STN:280:DC%2BD3M3ns1Sgtw%3D%3D 11359460
    • Nasir J. Transgenic monkey raises hope for primate models of human diseases. Clin Genet. 2001;59(5):304-5.
    • (2001) Clin Genet , vol.59 , Issue.5 , pp. 304-305
    • Nasir, J.1
  • 46
    • 0035835718 scopus 로고    scopus 로고
    • What next after the first transgenic monkey?
    • Senior K. What next after the first transgenic monkey? Lancet. 2001;357(9254):450
    • (2001) Lancet , vol.357 , Issue.9254 , pp. 450
    • Senior, K.1
  • 47
    • 45149105779 scopus 로고    scopus 로고
    • Towards a transgenic model of Huntington's disease in a non-human primate
    • 1:CAS:528:DC%2BD1cXntVCqurw%3D 2652570 18488016
    • Yang SH, Cheng PH, Banta H, Piotrowska-Nitsche K, Yang JJ, Cheng EC, et al. Towards a transgenic model of Huntington's disease in a non-human primate. Nature. 2008;453(7197):921-4.
    • (2008) Nature , vol.453 , Issue.7197 , pp. 921-924
    • Yang, S.H.1    Cheng, P.H.2    Banta, H.3    Piotrowska-Nitsche, K.4    Yang, J.J.5    Cheng, E.C.6
  • 48
    • 84926483293 scopus 로고    scopus 로고
    • Early Parkinson's disease symptoms in α-synuclein transgenic monkeys
    • Niu Y, Guo X, Chen Y, Wang CE, Gao J, Yang W, et al. Early Parkinson's disease symptoms in α-synuclein transgenic monkeys. Hum Mol Genet. 2014;24(8):2308-17.
    • (2014) Hum Mol Genet , vol.24 , Issue.8 , pp. 2308-2317
    • Niu, Y.1    Guo, X.2    Chen, Y.3    Wang, C.E.4    Gao, J.5    Yang, W.6
  • 50
    • 0030744876 scopus 로고    scopus 로고
    • Mutation in the α-synuclein gene identified in families with Parkinson's disease
    • Polymeropoulos MH, Lavedan C, Leroy E, Ide SE, Dehejia A, Dutra A, et al. Mutation in the α-synuclein gene identified in families with Parkinson's disease. science. 1997;276(5321):2045-47.
    • (1997) science , vol.276 , Issue.5321 , pp. 2045-2047
    • Polymeropoulos, M.H.1    Lavedan, C.2    Leroy, E.3    Ide, S.E.4    Dehejia, A.5    Dutra, A.6
  • 51
    • 41149163183 scopus 로고    scopus 로고
    • Parkinson's disease: clinical features and diagnosis
    • 1:STN:280:DC%2BD1c7oslamtA%3D%3D 18344392
    • Jankovic J. Parkinson's disease: clinical features and diagnosis. J Neurol Neurosurg Psychiatr. 2008;79(4):368-76.
    • (2008) J Neurol Neurosurg Psychiatr , vol.79 , Issue.4 , pp. 368-376
    • Jankovic, J.1
  • 52
    • 68149182585 scopus 로고    scopus 로고
    • Beyond the rat models of human neurodegenerative disorders
    • 19263215
    • Bugos O, Bhide M, Zilka N. Beyond the rat models of human neurodegenerative disorders. Cell Mol Neurobiol. 2009;29(6-7):859-69.
    • (2009) Cell Mol Neurobiol , vol.29 , Issue.6-7 , pp. 859-869
    • Bugos, O.1    Bhide, M.2    Zilka, N.3
  • 53
    • 0030272769 scopus 로고    scopus 로고
    • The unilateral 6-hydroxydopamine lesion model in behavioral brain research. Analysis of functional deficits, recovery and treatments
    • 1:CAS:528:DyaK2sXks12hsQ%3D%3D
    • Schwarting R, Huston J. The unilateral 6-hydroxydopamine lesion model in behavioral brain research. Analysis of functional deficits, recovery and treatments. Progr Neurobiol. 1996;50(2):275-331.
    • (1996) Progr Neurobiol , vol.50 , Issue.2 , pp. 275-331
    • Schwarting, R.1    Huston, J.2
  • 54
    • 77956217986 scopus 로고    scopus 로고
    • Parkinson's disease: a model dilemma
    • 1:CAS:528:DC%2BC3cXhtVKlurjM 20739935
    • Beal MF. Parkinson's disease: a model dilemma. Nature. 2010;466(7310):S8-S10.
    • (2010) Nature , vol.466 , Issue.7310 , pp. S8-S10
    • Beal, M.F.1
  • 55
    • 0141741347 scopus 로고    scopus 로고
    • Parkinson's disease: mechanisms and models
    • 1:CAS:528:DC%2BD3sXnsFait7o%3D 12971891
    • Dauer W, Przedborski S. Parkinson's disease: mechanisms and models. Neuron. 2003;39(6):889-909.
    • (2003) Neuron , vol.39 , Issue.6 , pp. 889-909
    • Dauer, W.1    Przedborski, S.2
  • 56
    • 84930260508 scopus 로고    scopus 로고
    • Mutant alpha-synuclein causes Age-dependent neuropathology in monkey brain
    • 1:CAS:528:DC%2BC2MXpvFKlt7g%3D 26019347
    • Yang W, Wang G, Wang C-E, Guo X, Yin P, Gao J, et al. Mutant alpha-synuclein causes Age-dependent neuropathology in monkey brain. J Neurosci. 2015;35(21):8345-58.
    • (2015) J Neurosci , vol.35 , Issue.21 , pp. 8345-8358
    • Yang, W.1    Wang, G.2    Wang, C.-E.3    Guo, X.4    Yin, P.5    Gao, J.6
  • 57
    • 84874624936 scopus 로고    scopus 로고
    • Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease
    • 1:CAS:528:DC%2BC3sXhsFGhsL8%3D 23360966
    • Cho SW, Kim S, Kim JM, Kim JS. Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease. Nat Biotechnol. 2013;31(3):230-2.
    • (2013) Nat Biotechnol , vol.31 , Issue.3 , pp. 230-232
    • Cho, S.W.1    Kim, S.2    Kim, J.M.3    Kim, J.S.4
  • 58
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • 1:CAS:528:DC%2BC3sXit1ygtb8%3D 3795411 23287718
    • Cong L, Ran FA, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex genome engineering using CRISPR/Cas systems. Science. 2013;339(6121):819-23.
    • (2013) Science , vol.339 , Issue.6121 , pp. 819-823
    • Cong, L.1    Ran, F.A.2    Cox, D.3    Lin, S.4    Barretto, R.5    Habib, N.6
  • 59
    • 84884160273 scopus 로고    scopus 로고
    • CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
    • 1:CAS:528:DC%2BC3sXht1SjsL7I 23907171
    • Mali P, Aach J, Stranges PB, Esvelt KM, Moosburner M, Kosuri S, et al. CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering. Nat Biotechnol. 2013;31(9):833-8.
    • (2013) Nat Biotechnol , vol.31 , Issue.9 , pp. 833-838
    • Mali, P.1    Aach, J.2    Stranges, P.B.3    Esvelt, K.M.4    Moosburner, M.5    Kosuri, S.6
  • 60
    • 84884856342 scopus 로고    scopus 로고
    • Cas9 as a versatile tool for engineering biology
    • 1:CAS:528:DC%2BC3sXhsFaks7rO 4051438 24076990
    • Mali P, Esvelt KM, Church GM. Cas9 as a versatile tool for engineering biology. Nat Methods. 2013;10(10):957-63.
    • (2013) Nat Methods , vol.10 , Issue.10 , pp. 957-963
    • Mali, P.1    Esvelt, K.M.2    Church, G.M.3
  • 61
    • 85042815594 scopus 로고    scopus 로고
    • Targeted genome modification of crop plants using a CRISPR-Cas system
    • 1:CAS:528:DC%2BC3sXht1Cgsb%2FI
    • Shan Q, Wang Y, Li J, Zhang Y, Chen K, Liang Z, et al. Targeted genome modification of crop plants using a CRISPR-Cas system. Nat Biotech. 2013;31(8):686-8.
    • (2013) Nat Biotech , vol.31 , Issue.8 , pp. 686-688
    • Shan, Q.1    Wang, Y.2    Li, J.3    Zhang, Y.4    Chen, K.5    Liang, Z.6
  • 62
    • 84897594300 scopus 로고    scopus 로고
    • One-step generation of knockout pigs by zygote injection of CRISPR/Cas system
    • 1:CAS:528:DC%2BC2cXhsl2gsLk%3D 3945887 24481528
    • Hai T, Teng F, Guo R, Li W, Zhou Q. One-step generation of knockout pigs by zygote injection of CRISPR/Cas system. Cell Res. 2014;24(3):372-5.
    • (2014) Cell Res , vol.24 , Issue.3 , pp. 372-375
    • Hai, T.1    Teng, F.2    Guo, R.3    Li, W.4    Zhou, Q.5
  • 63
    • 84925519674 scopus 로고    scopus 로고
    • Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer
    • 1:CAS:528:DC%2BC2cXhs12ntbzJ 25274063
    • Zhou X, Xin J, Fan N, Zou Q, Huang J, Ouyang Z, et al. Generation of CRISPR/Cas9-mediated gene-targeted pigs via somatic cell nuclear transfer. Cell Mol Life Sci. 2015;72(6):1175-84.
    • (2015) Cell Mol Life Sci , vol.72 , Issue.6 , pp. 1175-1184
    • Zhou, X.1    Xin, J.2    Fan, N.3    Zou, Q.4    Huang, J.5    Ouyang, Z.6
  • 64
    • 84894081986 scopus 로고    scopus 로고
    • Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos
    • 1:CAS:528:DC%2BC2cXhs1ajtLY%3D 24486104
    • Niu Y, Shen B, Cui Y, Chen Y, Wang J, Wang L, et al. Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos. Cell. 2014;156(4):836-43.
    • (2014) Cell , vol.156 , Issue.4 , pp. 836-843
    • Niu, Y.1    Shen, B.2    Cui, Y.3    Chen, Y.4    Wang, J.5    Wang, L.6
  • 65
    • 84936765320 scopus 로고    scopus 로고
    • Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9
    • 25859012
    • Chen Y, Zheng Y, Kang Y, Yang W, Niu Y, Guo X, et al. Functional disruption of the dystrophin gene in rhesus monkey using CRISPR/Cas9. Hum Mol Genet. 2015;24(13):3764-74.
    • (2015) Hum Mol Genet , vol.24 , Issue.13 , pp. 3764-3774
    • Chen, Y.1    Zheng, Y.2    Kang, Y.3    Yang, W.4    Niu, Y.5    Guo, X.6
  • 66
    • 84922225042 scopus 로고    scopus 로고
    • Germline acquisition of Cas9/RNA-mediated gene modifications in monkeys
    • 1:CAS:528:DC%2BC2cXitFyhu7vM 25533168
    • Chen Y, Cui Y, Shen B, Niu Y, Zhao X, Wang L, et al. Germline acquisition of Cas9/RNA-mediated gene modifications in monkeys. Cell Res. 2015;25(2):262-5.
    • (2015) Cell Res , vol.25 , Issue.2 , pp. 262-265
    • Chen, Y.1    Cui, Y.2    Shen, B.3    Niu, Y.4    Zhao, X.5    Wang, L.6
  • 67
    • 84922258547 scopus 로고    scopus 로고
    • One-step generation of p53 gene biallelic mutant Cynomolgus monkey via the CRISPR/Cas system
    • 1:CAS:528:DC%2BC2cXhvF2mtbzM 25430965
    • Wan H, Feng C, Teng F, Yang S, Hu B, Niu Y, et al. One-step generation of p53 gene biallelic mutant Cynomolgus monkey via the CRISPR/Cas system. Cell Res. 2015;25(2):258-61.
    • (2015) Cell Res , vol.25 , Issue.2 , pp. 258-261
    • Wan, H.1    Feng, C.2    Teng, F.3    Yang, S.4    Hu, B.5    Niu, Y.6
  • 68
    • 72149110399 scopus 로고    scopus 로고
    • Breaking the code of DNA binding specificity of TAL-type III effectors
    • 1:CAS:528:DC%2BD1MXhsFensbnL 19933107
    • Boch J, Scholze H, Schornack S, Landgraf A, Hahn S, Kay S, et al. Breaking the code of DNA binding specificity of TAL-type III effectors. Science. 2009;326(5959):1509-12.
    • (2009) Science , vol.326 , Issue.5959 , pp. 1509-1512
    • Boch, J.1    Scholze, H.2    Schornack, S.3    Landgraf, A.4    Hahn, S.5    Kay, S.6
  • 69
    • 78951479577 scopus 로고    scopus 로고
    • Targeting DNA double-strand breaks with TAL effector nucleases
    • 1:CAS:528:DC%2BC3cXhsFOnt7jP 2942870 20660643
    • Christian M, Cermak T, Doyle EL, Schmidt C, Zhang F, Hummel A, et al. Targeting DNA double-strand breaks with TAL effector nucleases. Genetics. 2010;186(2):757-61.
    • (2010) Genetics , vol.186 , Issue.2 , pp. 757-761
    • Christian, M.1    Cermak, T.2    Doyle, E.L.3    Schmidt, C.4    Zhang, F.5    Hummel, A.6
  • 70
    • 84879410743 scopus 로고    scopus 로고
    • TALEN or Cas9 - rapid, efficient and specific choices for genome modifications
    • 1:CAS:528:DC%2BC3sXhvFamsrfI 23790627
    • Wei C, Liu J, Yu Z, Zhang B, Gao G, Jiao R. TALEN or Cas9 - rapid, efficient and specific choices for genome modifications. J Genet Genomics. 2013;40(6):281-9.
    • (2013) J Genet Genomics , vol.40 , Issue.6 , pp. 281-289
    • Wei, C.1    Liu, J.2    Yu, Z.3    Zhang, B.4    Gao, G.5    Jiao, R.6
  • 72
    • 84906541312 scopus 로고    scopus 로고
    • CRISPR-Cas systems: new players in gene regulation and bacterial physiology
    • 3983513 24772391
    • Sampson TR, Weiss DS. CRISPR-Cas systems: new players in gene regulation and bacterial physiology. Front Cell Infect Microbiol. 2014;4:37.
    • (2014) Front Cell Infect Microbiol , vol.4 , pp. 37
    • Sampson, T.R.1    Weiss, D.S.2
  • 73
    • 84900314611 scopus 로고    scopus 로고
    • CRISPR-Cas systems for editing, regulating and targeting genomes
    • 1:CAS:528:DC%2BC2cXjtlyrsLo%3D 4022601 24584096
    • Sander JD, Joung JK. CRISPR-Cas systems for editing, regulating and targeting genomes. Nat Biotechnol. 2014;32(4):347-55.
    • (2014) Nat Biotechnol , vol.32 , Issue.4 , pp. 347-355
    • Sander, J.D.1    Joung, J.K.2
  • 74
    • 84902096048 scopus 로고    scopus 로고
    • Development and applications of CRISPR-Cas9 for genome engineering
    • 1:CAS:528:DC%2BC2cXpslagt7s%3D 4343198 24906146
    • Hsu PD, Lander ES, Zhang F. Development and applications of CRISPR-Cas9 for genome engineering. Cell. 2014;157(6):1262-78.
    • (2014) Cell , vol.157 , Issue.6 , pp. 1262-1278
    • Hsu, P.D.1    Lander, E.S.2    Zhang, F.3
  • 75
    • 84905732049 scopus 로고    scopus 로고
    • Somatic mosaicism and allele complexity induced by CRISPR/Cas9 RNA injections in mouse zygotes
    • 1:CAS:528:DC%2BC2cXhtFCiu7fM 4166609 24984260
    • Yen ST, Zhang M, Deng JM, Usman SJ, Smith CN, Parker-Thornburg J, et al. Somatic mosaicism and allele complexity induced by CRISPR/Cas9 RNA injections in mouse zygotes. Dev Biol. 2014;393(1):3-9.
    • (2014) Dev Biol , vol.393 , Issue.1 , pp. 3-9
    • Yen, S.T.1    Zhang, M.2    Deng, J.M.3    Usman, S.J.4    Smith, C.N.5    Parker-Thornburg, J.6
  • 76
    • 84907200149 scopus 로고    scopus 로고
    • Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA
    • 1:CAS:528:DC%2BC2cXhsVGhtb7F 4398027 25123483
    • Long CZ, McAnally JR, Shelton JM, Mireault AA, Bassel-Duby R, Olson EN. Prevention of muscular dystrophy in mice by CRISPR/Cas9-mediated editing of germline DNA. Science. 2014;345(6201):1184-8.
    • (2014) Science , vol.345 , Issue.6201 , pp. 1184-1188
    • Long, C.Z.1    McAnally, J.R.2    Shelton, J.M.3    Mireault, A.A.4    Bassel-Duby, R.5    Olson, E.N.6
  • 77
    • 84884925278 scopus 로고    scopus 로고
    • Highly improved gene targeting by germline-specific Cas9 expression in Drosophila
    • 1:CAS:528:DC%2BC2cXhtlGjtLk%3D 3813859 24002648
    • Kondo S, Ueda R. Highly improved gene targeting by germline-specific Cas9 expression in Drosophila. Genetics. 2013;195(3):715-21.
    • (2013) Genetics , vol.195 , Issue.3 , pp. 715-721
    • Kondo, S.1    Ueda, R.2
  • 78
    • 0024524606 scopus 로고
    • Muscle hypertrophy in Duchenne muscular dystrophy. A pathological and morphometric study
    • 1:STN:280:DyaL1M7isV2qtw%3D%3D 2915226
    • Cros D, Harnden P, Pellissier JF, Serratrice G. Muscle hypertrophy in Duchenne muscular dystrophy. A pathological and morphometric study. J Neurol. 1989;236(1):43-7.
    • (1989) J Neurol , vol.236 , Issue.1 , pp. 43-47
    • Cros, D.1    Harnden, P.2    Pellissier, J.F.3    Serratrice, G.4
  • 79
    • 84938516868 scopus 로고    scopus 로고
    • A novel mechanism for the expression of dystrophin in a Duchenne muscular dystrophy patient
    • Tsao CY, Bartolo C, Luquette MH, Mendell JR, Prior TW. A novel mechanism for the expression of dystrophin in a Duchenne muscular dystrophy patient. Neurology. 1996;46(2):12002-2.
    • (1996) Neurology , vol.46 , Issue.2 , pp. 12002-12012
    • Tsao, C.Y.1    Bartolo, C.2    Luquette, M.H.3    Mendell, J.R.4    Prior, T.W.5
  • 80
    • 84929166074 scopus 로고    scopus 로고
    • Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining
    • 1:CAS:528:DC%2BC2MXkvFejur0%3D 25798939
    • Maruyama T, Dougan SK, Truttmann MC, Bilate AM, Ingram JR, Ploegh HL. Increasing the efficiency of precise genome editing with CRISPR-Cas9 by inhibition of nonhomologous end joining. Nat Biotechnol. 2015;33(5):538-42.
    • (2015) Nat Biotechnol , vol.33 , Issue.5 , pp. 538-542
    • Maruyama, T.1    Dougan, S.K.2    Truttmann, M.C.3    Bilate, A.M.4    Ingram, J.R.5    Ploegh, H.L.6
  • 81
    • 84891704542 scopus 로고    scopus 로고
    • Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases
    • 1:CAS:528:DC%2BC2cXosVCitA%3D%3D 3875853 24253447
    • Sung YH, Kim JM, Kim HT, Lee J, Jeon J, Jin Y, et al. Highly efficient gene knockout in mice and zebrafish with RNA-guided endonucleases. Genome Res. 2014;24(1):125-31.
    • (2014) Genome Res , vol.24 , Issue.1 , pp. 125-131
    • Sung, Y.H.1    Kim, J.M.2    Kim, H.T.3    Lee, J.4    Jeon, J.5    Jin, Y.6
  • 82
    • 84884288934 scopus 로고    scopus 로고
    • Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity
    • 1:CAS:528:DC%2BC3sXhtlGrur3M 3856256 23992846
    • Ran FA, Hsu PD, Lin CY, Gootenberg JS, Konermann S, Trevino AE, et al. Double nicking by RNA-guided CRISPR Cas9 for enhanced genome editing specificity. Cell. 2013;154(6):1380-9.
    • (2013) Cell , vol.154 , Issue.6 , pp. 1380-1389
    • Ran, F.A.1    Hsu, P.D.2    Lin, C.Y.3    Gootenberg, J.S.4    Konermann, S.5    Trevino, A.E.6
  • 83
    • 84929147435 scopus 로고    scopus 로고
    • Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells
    • 1:CAS:528:DC%2BC2MXltFWks7g%3D 25803306
    • Chu VT, Weber T, Wefers B, Wurst W, Sander S, Rajewsky K, et al. Increasing the efficiency of homology-directed repair for CRISPR-Cas9-induced precise gene editing in mammalian cells. Nat Biotechnol. 2015;33(5):543-8.
    • (2015) Nat Biotechnol , vol.33 , Issue.5 , pp. 543-548
    • Chu, V.T.1    Weber, T.2    Wefers, B.3    Wurst, W.4    Sander, S.5    Rajewsky, K.6
  • 84
    • 84938497932 scopus 로고    scopus 로고
    • CRISPR/Cas9-mediated gene knock-down in post-mitotic neurons
    • 4139396 25140704
    • Straub C, Granger AJ, Saulnier JL, Sabatini BL. CRISPR/Cas9-mediated gene knock-down in post-mitotic neurons. PLoS One. 2014;9(8), e105584.
    • (2014) PLoS One , vol.9 , Issue.8
    • Straub, C.1    Granger, A.J.2    Saulnier, J.L.3    Sabatini, B.L.4
  • 85
    • 84907963330 scopus 로고    scopus 로고
    • Efficient Complete Deletion of Synaptic Proteins using CRISPR
    • 1:CAS:528:DC%2BC2cXhsVKntrvE 4195490 25155957
    • Incontro S et al. Efficient Complete Deletion of Synaptic Proteins using CRISPR. Neuron. 2014;83:1051-7.
    • (2014) Neuron , vol.83 , pp. 1051-1057
    • Incontro, S.1
  • 86
    • 84926061715 scopus 로고    scopus 로고
    • In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9
    • 1:CAS:528:DC%2BC2cXhslOisrvK 4492112 25326897
    • Swiech L et al. In vivo interrogation of gene function in the mammalian brain using CRISPR-Cas9. Nat Biotechnol. 2015;33:102-6.
    • (2015) Nat Biotechnol , vol.33 , pp. 102-106
    • Swiech, L.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.