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4. 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]
5. 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; 351(6271):407-411. PubMed ID: 26721686 [TBL] [Abstract][Full Text] [Related]
6. Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy. Long C; Amoasii L; Mireault AA; McAnally JR; Li H; Sanchez-Ortiz E; Bhattacharyya S; Shelton JM; Bassel-Duby R; Olson EN Science; 2016 Jan; 351(6271):400-3. PubMed ID: 26721683 [TBL] [Abstract][Full Text] [Related]
7. Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient. Takeshima Y; Wada H; Yagi M; Ishikawa Y; Ishikawa Y; Minami R; Nakamura H; Matsuo M Brain Dev; 2001 Dec; 23(8):788-90. PubMed ID: 11720794 [No Abstract] [Full Text] [Related]
8. In Vivo Modeling of Skeletal Muscle Diseases Using the CRISPR/Cas9 System in Rats. Nakamura K; Tanaka T; Yamanouchi K Methods Mol Biol; 2023; 2640():277-285. PubMed ID: 36995602 [TBL] [Abstract][Full Text] [Related]
10. CTLA4Ig delivered by high-capacity adenoviral vector induces stable expression of dystrophin in mdx mouse muscle. Jiang Z; Schiedner G; Gilchrist SC; Kochanek S; Clemens PR Gene Ther; 2004 Oct; 11(19):1453-61. PubMed ID: 15269713 [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. Retroviral vectors for gene therapy of Duchenne muscular dystrophy. Fassati A; Bresolin N Neurol Sci; 2000; 21(5 Suppl):S925-7. PubMed ID: 11382191 [TBL] [Abstract][Full Text] [Related]
13. Exon Skipping Therapy Using Phosphorodiamidate Morpholino Oligomers in the mdx52 Mouse Model of Duchenne Muscular Dystrophy. Miyatake S; Mizobe Y; Takizawa H; Hara Y; Yokota T; Takeda S; Aoki Y Methods Mol Biol; 2018; 1687():123-141. PubMed ID: 29067660 [TBL] [Abstract][Full Text] [Related]
14. The Use of Antisense Oligonucleotides for the Treatment of Duchenne Muscular Dystrophy. Relizani K; Goyenvalle A Methods Mol Biol; 2018; 1687():171-183. PubMed ID: 29067663 [TBL] [Abstract][Full Text] [Related]
15. Toward the correction of muscular dystrophy by gene editing. Olson EN Proc Natl Acad Sci U S A; 2021 Jun; 118(22):. PubMed ID: 34074727 [TBL] [Abstract][Full Text] [Related]
16. Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy. Echigoya Y; Lim KRQ; Trieu N; Bao B; Miskew Nichols B; Vila MC; Novak JS; Hara Y; Lee J; Touznik A; Mamchaoui K; Aoki Y; Takeda S; Nagaraju K; Mouly V; Maruyama R; Duddy W; Yokota T Mol Ther; 2017 Nov; 25(11):2561-2572. PubMed ID: 28865998 [TBL] [Abstract][Full Text] [Related]
17. [Development of novel therapeutic approaches for Duchenne muscular dystrophy]. Takeshima Y No To Hattatsu; 2014 Mar; 46(2):109-14. PubMed ID: 24738186 [No Abstract] [Full Text] [Related]
18. Cardiac Myoediting Attenuates Cardiac Abnormalities in Human and Mouse Models of Duchenne Muscular Dystrophy. Atmanli A; Chai AC; Cui M; Wang Z; Nishiyama T; Bassel-Duby R; Olson EN Circ Res; 2021 Sep; 129(6):602-616. PubMed ID: 34372664 [TBL] [Abstract][Full Text] [Related]
19. [Frontline studies on Duchenne muscular dystrophy treatment]. Matsuo M No To Hattatsu; 2009 Mar; 41(2):92-5. PubMed ID: 19517771 [TBL] [Abstract][Full Text] [Related]
20. Neuromuscular disease: Genome editing shows promise in an in vivo model of Duchenne muscular dystrophy. Wood H Nat Rev Neurol; 2016 Feb; 12(2):63. PubMed ID: 26782331 [No Abstract] [Full Text] [Related] [Next] [New Search]