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Journal Abstract Search

776 related items for PubMed ID: 28195574

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  • 3. The gRNA Vector Level Determines the Outcome of Systemic AAV CRISPR Therapy for Duchenne Muscular Dystrophy.
    Wasala NB, Million ED, Watkins TB, Wasala LP, Han J, Yue Y, Lu B, Chen SJ, Hakim CH, Duan D.
    Hum Gene Ther; 2022 May; 33(9-10):518-528. PubMed ID: 35350865
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  • 5. Correction of Three Prominent Mutations in Mouse and Human Models of Duchenne Muscular Dystrophy by Single-Cut Genome Editing.
    Min YL, Chemello F, Li H, Rodriguez-Caycedo C, Sanchez-Ortiz E, Mireault AA, McAnally JR, Shelton JM, Zhang Y, Bassel-Duby R, Olson EN.
    Mol Ther; 2020 Sep 02; 28(9):2044-2055. PubMed ID: 32892813
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  • 6. Dystrophin Gene-Editing Stability Is Dependent on Dystrophin Levels in Skeletal but Not Cardiac Muscles.
    Bengtsson NE, Tasfaout H, Hauschka SD, Chamberlain JS.
    Mol Ther; 2021 Mar 03; 29(3):1070-1085. PubMed ID: 33160075
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  • 7. Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.
    Pickar-Oliver A, Gough V, Bohning JD, Liu S, Robinson-Hamm JN, Daniels H, Majoros WH, Devlin G, Asokan A, Gersbach CA.
    Mol Ther; 2021 Nov 03; 29(11):3243-3257. PubMed ID: 34509668
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  • 8. In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
    Nelson CE, Hakim CH, Ousterout DG, Thakore PI, Moreb EA, Castellanos Rivera RM, Madhavan S, Pan X, Ran FA, Yan WX, Asokan A, Zhang F, Duan D, Gersbach CA.
    Science; 2016 Jan 22; 351(6271):403-7. PubMed ID: 26721684
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  • 9. CRISPR-Cas9 Correction of Duchenne Muscular Dystrophy in Mice by a Self-Complementary AAV Delivery System.
    Zhang Y, Bassel-Duby R, Olson EN.
    Methods Mol Biol; 2023 Jan 22; 2587():411-425. PubMed ID: 36401041
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  • 10. Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.
    Zhang Y, Li H, Min YL, Sanchez-Ortiz E, Huang J, Mireault AA, Shelton JM, Kim J, Mammen PPA, Bassel-Duby R, Olson EN.
    Sci Adv; 2020 Feb 22; 6(8):eaay6812. PubMed ID: 32128412
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  • 12. Life-Long AAV-Mediated CRISPR Genome Editing in Dystrophic Heart Improves Cardiomyopathy without Causing Serious Lesions in mdx Mice.
    Xu L, Lau YS, Gao Y, Li H, Han R.
    Mol Ther; 2019 Aug 07; 27(8):1407-1414. PubMed ID: 31129119
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  • 15. In vivo gene editing in dystrophic mouse muscle and muscle stem cells.
    Tabebordbar M, Zhu K, Cheng JKW, Chew WL, Widrick JJ, Yan WX, Maesner C, Wu EY, Xiao R, Ran FA, Cong L, Zhang F, Vandenberghe LH, Church GM, Wagers AJ.
    Science; 2016 Jan 22; 351(6271):407-411. PubMed ID: 26721686
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  • 17. In vivo genome editing in mouse restores dystrophin expression in Duchenne muscular dystrophy patient muscle fibers.
    Chen M, Shi H, Gou S, Wang X, Li L, Jin Q, Wu H, Zhang H, Li Y, Wang L, Li H, Lin J, Guo W, Jiang Z, Yang X, Xu A, Zhu Y, Zhang C, Lai L, Li X.
    Genome Med; 2021 Apr 12; 13(1):57. PubMed ID: 33845891
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  • 18. CRISPR-mediated Genome Editing Restores Dystrophin Expression and Function in mdx Mice.
    Xu L, Park KH, Zhao L, Xu J, El Refaey M, Gao Y, Zhu H, Ma J, Han R.
    Mol Ther; 2016 Mar 12; 24(3):564-9. PubMed ID: 26449883
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  • 19. Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
    Wong TWY, Cohn RD.
    Curr Gene Ther; 2017 Mar 12; 17(4):301-308. PubMed ID: 29173172
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  • 20. AAV-Mediated Somatic Gene Editing for Cardiac and Skeletal Muscle in a Large Animal Model.
    Ziegler T, Bozoglu T, Kupatt C.
    Methods Mol Biol; 2022 Mar 12; 2573():63-74. PubMed ID: 36040587
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