메뉴 건너뛰기




Volumn 4, Issue 2, 2017, Pages 139-145

Creation of a Novel Humanized Dystrophic Mouse Model of Duchenne Muscular Dystrophy and Application of a CRISPR/Cas9 Gene Editing Therapy

Author keywords

animal models; CRISPR; Duchenne muscular dystrophy; gene editing; mice

Indexed keywords

DYSTROPHIN; DMD PROTEIN, HUMAN;

EID: 85032567494     PISSN: 22143599     EISSN: 22143602     Source Type: Journal    
DOI: 10.3233/JND-170218     Document Type: Article
Times cited : (67)

References (17)
  • 2
    • 0023718118 scopus 로고
    • An explanation for the phenotypic differences between patients bearing partial deletions of theDMDlocus
    • Monaco AP, Bertelson CJ, Liechti-Gallati S, Moser H, Kunkel LM. An explanation for the phenotypic differences between patients bearing partial deletions of theDMDlocus. Genomics. 1988;2(1):90-5.
    • (1988) Genomics. , vol.2 , Issue.1 , pp. 90-95
    • Monaco, A.P.1    Bertelson, C.J.2    Liechti-Gallati, S.3    Moser, H.4    Kunkel, L.M.5
  • 3
    • 34748906143 scopus 로고    scopus 로고
    • Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications
    • Aartsma-Rus A, van Ommen G-JB. Antisense-mediated exon skipping: A versatile tool with therapeutic and research applications. RNA. 2007;13(10):1609-24. doi: 10. 1261/rna. 653607
    • (2007) RNA , vol.13 , Issue.10 , pp. 1609-1624
    • Aartsma-Rus, A.1    Van Ommen, G.-J.B.2
  • 5
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using crispr/cas systems
    • Cong L, Ran AF, Cox D, Lin S, Barretto R, Habib N, et al. Multiplex Genome Engineering Using CRISPR/Cas Systems. Science. 2013;339:819-23. doi: 10. 1126/science. 1231143
    • (2013) Science. , vol.339 , pp. 819-823
    • Cong, L.1    Ran, A.F.2    Cox, D.3    Lin, S.4    Barretto, R.5    Habib, N.6
  • 6
    • 84865070369 scopus 로고    scopus 로고
    • A programmable dual-RNA-guided DNA endonuclease in adaptive bacterial immunity
    • Jinek M, Chylinski K, Fonfara I, Hauer M, Doudna JA, Charpentier E. A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity. Science. 2012;337:816-22. doi: 10. 1126/science. 1225829
    • (2012) Science. , vol.337 , pp. 816-822
    • Jinek, M.1    Chylinski, K.2    Fonfara, I.3    Hauer, M.4    Doudna, J.A.5    Charpentier, E.6
  • 7
    • 41949122156 scopus 로고    scopus 로고
    • Generation and characterization of transgenic mice with the full-length human DMD gene
    • 't Hoen PAC, de Meijer EJ, Boer JM, Vossen RHAM, Turk R, Maatman RGHJ, et al. Generation and characterization of transgenic mice with the full-length human DMD gene. J Biol Chem. 2008;283(9):5899-907. doi: 10. 1074/jbc. M709410200
    • (2008) J Biol Chem. , vol.283 , Issue.9 , pp. 5899-5907
    • T'Hoen, P.A.C.1    De Meijer, E.J.2    Boer, J.M.3    Vossen, R.H.A.M.4    Turk, R.5    Maatman, R.G.H.J.6
  • 8
    • 72849135317 scopus 로고    scopus 로고
    • Latent TGF-binding protein 4 modifies muscular dystrophy in mice
    • Heydemann A, Ceco E, Lim JE, Hadhazy M, Ryder P, Moran JL, et al. Latent TGF-binding protein 4 modifies muscular dystrophy in mice. JCI. 2009;119(12):3703-12. doi: 10. 1172/JCI39845
    • (2009) JCI , vol.119 , Issue.12 , pp. 3703-3712
    • Heydemann, A.1    Ceco, E.2    Lim, J.E.3    Hadhazy, M.4    Ryder, P.5    Moran, J.L.6
  • 10
    • 84899083880 scopus 로고    scopus 로고
    • Annexin A6 modifies muscular dystrophy by mediating sarcolemmal repair
    • Swaggart KA, Demonbreun AR, Vo AH, Swanson KE, Kim EY, Fahrenbach JP, et al. Annexin A6 modifies muscular dystrophy by mediating sarcolemmal repair. PNAS. 2014;111(16):6004-9. doi: 10. 1073/pnas. 1324242111
    • (2014) PNAS , vol.111 , Issue.16 , pp. 6004-6009
    • Swaggart, K.A.1    Demonbreun, A.R.2    Vo, A.H.3    Swanson, K.E.4    Kim, E.Y.5    Fahrenbach, J.P.6
  • 11
    • 84962787321 scopus 로고    scopus 로고
    • A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-DerivedMuscle Cells
    • Young CS, Hicks MR, Ermolova NV, Nakano H, Jan M, Younesi S, et al. A Single CRISPR-Cas9 Deletion Strategy that Targets the Majority of DMD Patients Restores Dystrophin Function in hiPSC-DerivedMuscle Cells. Cell Stem Cell. 2016;18(4):533-40. doi: 10. 1016/j. stem. 2016. 01. 021
    • (2016) Cell Stem Cell. , vol.18 , Issue.4 , pp. 533-540
    • Young, C.S.1    Hicks, M.R.2    Ermolova, N.V.3    Nakano, H.4    Jan, M.5    Younesi, S.6
  • 12
    • 80055107411 scopus 로고    scopus 로고
    • DNA transfection of mammalian skeletal muscles using in vivo electroporation
    • DiFranco M, Quinonez M, Capote J, Vergara J. DNA transfection of mammalian skeletal muscles using in vivo electroporation. JoVE. 2009;(32):1-7. doi: 10. 3791/1520
    • (2009) JoVE. , Issue.32 , pp. 1-7
    • DiFranco, M.1    Quinonez, M.2    Capote, J.3    Vergara, J.4
  • 13
    • 84922735816 scopus 로고    scopus 로고
    • In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system
    • Maddalo D, Manchado E, Concepcion CP, Bonetti C, Vidigal JA, Han Y-C, et al. In vivo engineering of oncogenic chromosomal rearrangements with the CRISPR/Cas9 system. Nature. 2014;516(7531):423-7. doi: 10. 1038/nature13902
    • (2014) Nature. , vol.516 , Issue.7531 , pp. 423-427
    • Maddalo, D.1    Manchado, E.2    Concepcion, C.P.3    Bonetti, C.4    Vidigal, J.A.5    Han, Y.-C.6
  • 14
    • 0028910144 scopus 로고
    • Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin
    • Thanh LT, Nguyen TM, Helliwell TR, Morris GE. Characterization of revertant muscle fibers in Duchenne muscular dystrophy, using exon-specific monoclonal antibodies against dystrophin. Am J Hum Genet. 1995;56(3): 725-31.
    • (1995) Am J Hum Genet. , vol.56 , Issue.3 , pp. 725-731
    • Thanh, L.T.1    Nguyen, T.M.2    Helliwell, T.R.3    Morris, G.E.4
  • 15
    • 84873729095 scopus 로고    scopus 로고
    • Multiplex genome engineering using CRISPR/Cas systems
    • 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. doi: 10. 1126/science. 1231143
    • (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
  • 16
    • 0027365063 scopus 로고
    • Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy
    • Man N, Morris GE. Use of epitope libraries to identify exon-specific monoclonal antibodies for characterization of altered dystrophins in muscular dystrophy. Am J Hum Genet. 1993;52:1057-66.
    • (1993) Am J Hum Genet. , vol.52 , pp. 1057-1066
    • Man, N.1    Morris, G.E.2
  • 17
    • 84871061937 scopus 로고    scopus 로고
    • Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy
    • Kendall GC, Mokhonova EI, Moran M, Sejbuk NE, Wang DW, SilvaO, et al. Dantrolene enhances antisense-mediated exon skipping in human and mouse models of Duchenne muscular dystrophy. Sci Transl Med. 2012;4:164ra160. doi: 10. 1126/scitranslmed. 3005054
    • (2012) Sci Transl Med. , vol.4 , pp. 164ra160
    • Kendall, G.C.1    Mokhonova, E.I.2    Moran, M.3    Sejbuk, N.E.4    Wang, D.W.5    Silva, O.6


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.