BIOMARKERS

Molecular Biopsy of Human Tumors

- a resource for Precision Medicine *

482 related articles for article (PubMed ID: 32128412)

  • 1. 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; 6(8):eaay6812. PubMed ID: 32128412
    [TBL] [Abstract][Full Text] [Related]  

  • 2. 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; 29(11):3243-3257. PubMed ID: 34509668
    [TBL] [Abstract][Full Text] [Related]  

  • 3. 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; 2587():411-425. PubMed ID: 36401041
    [TBL] [Abstract][Full Text] [Related]  

  • 4. In Vivo Genome Editing Restores Dystrophin Expression and Cardiac Function in Dystrophic Mice.
    El Refaey M; Xu L; Gao Y; Canan BD; Adesanya TMA; Warner SC; Akagi K; Symer DE; Mohler PJ; Ma J; Janssen PML; Han R
    Circ Res; 2017 Sep; 121(8):923-929. PubMed ID: 28790199
    [TBL] [Abstract][Full Text] [Related]  

  • 5. 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; 27(8):1407-1414. PubMed ID: 31129119
    [TBL] [Abstract][Full Text] [Related]  

  • 6. Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.
    Bengtsson NE; Hall JK; Odom GL; Phelps MP; Andrus CR; Hawkins RD; Hauschka SD; Chamberlain JR; Chamberlain JS
    Nat Commun; 2017 Feb; 8():14454. PubMed ID: 28195574
    [TBL] [Abstract][Full Text] [Related]  

  • 7. CRISPR-Cas9 corrects Duchenne muscular dystrophy exon 44 deletion mutations in mice and human cells.
    Min YL; Li H; Rodriguez-Caycedo C; Mireault AA; Huang J; Shelton JM; McAnally JR; Amoasii L; Mammen PPA; Bassel-Duby R; Olson EN
    Sci Adv; 2019 Mar; 5(3):eaav4324. PubMed ID: 30854433
    [TBL] [Abstract][Full Text] [Related]  

  • 8. 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; 29(3):1070-1085. PubMed ID: 33160075
    [TBL] [Abstract][Full Text] [Related]  

  • 9. 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
    [TBL] [Abstract][Full Text] [Related]  

  • 10. 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; 28(9):2044-2055. PubMed ID: 32892813
    [TBL] [Abstract][Full Text] [Related]  

  • 11. The AAV-mediated and RNA-guided CRISPR/Cas9 system for gene therapy of DMD and BMD.
    Wang JZ; Wu P; Shi ZM; Xu YL; Liu ZJ
    Brain Dev; 2017 Aug; 39(7):547-556. PubMed ID: 28390761
    [TBL] [Abstract][Full Text] [Related]  

  • 12. AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
    Nance ME; Shi R; Hakim CH; Wasala NB; Yue Y; Pan X; Zhang T; Robinson CA; Duan SX; Yao G; Yang NN; Chen SJ; Wagner KR; Gersbach CA; Duan D
    Mol Ther; 2019 Sep; 27(9):1568-1585. PubMed ID: 31327755
    [TBL] [Abstract][Full Text] [Related]  

  • 13. 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; 13(1):57. PubMed ID: 33845891
    [TBL] [Abstract][Full Text] [Related]  

  • 14. Therapeutic Applications of CRISPR/Cas for Duchenne Muscular Dystrophy.
    Wong TWY; Cohn RD
    Curr Gene Ther; 2017; 17(4):301-308. PubMed ID: 29173172
    [TBL] [Abstract][Full Text] [Related]  

  • 15. Molecular correction of Duchenne muscular dystrophy by splice modulation and gene editing.
    Hanson B; Wood MJA; Roberts TC
    RNA Biol; 2021 Jul; 18(7):1048-1062. PubMed ID: 33472516
    [TBL] [Abstract][Full Text] [Related]  

  • 16. Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy.
    Nelson CE; Wu Y; Gemberling MP; Oliver ML; Waller MA; Bohning JD; Robinson-Hamm JN; Bulaklak K; Castellanos Rivera RM; Collier JH; Asokan A; Gersbach CA
    Nat Med; 2019 Mar; 25(3):427-432. PubMed ID: 30778238
    [TBL] [Abstract][Full Text] [Related]  

  • 17. AAV CRISPR editing rescues cardiac and muscle function for 18 months in dystrophic mice.
    Hakim CH; Wasala NB; Nelson CE; Wasala LP; Yue Y; Louderman JA; Lessa TB; Dai A; Zhang K; Jenkins GJ; Nance ME; Pan X; Kodippili K; Yang NN; Chen SJ; Gersbach CA; Duan D
    JCI Insight; 2018 Dec; 3(23):. PubMed ID: 30518686
    [TBL] [Abstract][Full Text] [Related]  

  • 18. In vivo non-invasive monitoring of dystrophin correction in a new Duchenne muscular dystrophy reporter mouse.
    Amoasii L; Li H; Zhang Y; Min YL; Sanchez-Ortiz E; Shelton JM; Long C; Mireault AA; Bhattacharyya S; McAnally JR; Bassel-Duby R; Olson EN
    Nat Commun; 2019 Oct; 10(1):4537. PubMed ID: 31586095
    [TBL] [Abstract][Full Text] [Related]  

  • 19. 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; 2573():63-74. PubMed ID: 36040587
    [TBL] [Abstract][Full Text] [Related]  

  • 20. 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; 351(6271):403-7. PubMed ID: 26721684
    [TBL] [Abstract][Full Text] [Related]  

    [Next]    [New Search]
    of 25.