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In vivo gene editing in dystrophic mouse muscle and muscle stem cells.
Tabebordbar, Mohammadsharif; Zhu, Kexian; Cheng, Jason K W; Chew, Wei Leong; Widrick, Jeffrey J; Yan, Winston X; Maesner, Claire; Wu, Elizabeth Y; Xiao, Ru; Ran, F Ann; Cong, Le; Zhang, Feng; Vandenberghe, Luk H; Church, George M; Wagers, Amy J.
Afiliación
  • Tabebordbar M; Department of Stem Cell and Regenerative Biology, Harvard University and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  • Zhu K; Biological and Biomedical Sciences Program, Harvard Medical School, Boston, MA 02115, USA.
  • Cheng JKW; Department of Stem Cell and Regenerative Biology, Harvard University and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  • Chew WL; Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.
  • Widrick JJ; Department of Stem Cell and Regenerative Biology, Harvard University and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  • Yan WX; Biological and Biomedical Sciences Program, Harvard Medical School, Boston, MA 02115, USA.
  • Maesner C; Department of Genetics, Harvard Medical School, Boston, MA 02115, USA.
  • Wu EY; Division of Genetics and Program in Genomics, Boston Children's Hospital, Harvard Medical School, Boston, MA 02115, USA.
  • Xiao R; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  • Ran FA; McGovern Institute for Brain Research, Department of Brain and Cognitive Science, and Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
  • Cong L; Department of Stem Cell and Regenerative Biology, Harvard University and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  • Zhang F; Department of Stem Cell and Regenerative Biology, Harvard University and Harvard Stem Cell Institute, Cambridge, MA 02138, USA.
  • Vandenberghe LH; Grousbeck Gene Therapy Center, Schepens Eye Research Institute and Massachusetts Eye and Ear Infirmary, 20 Staniford Street, Boston, MA 02114, USA.
  • Church GM; Broad Institute of MIT and Harvard, Cambridge, MA 02142, USA.
  • Wagers AJ; McGovern Institute for Brain Research, Department of Brain and Cognitive Science, and Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Science ; 351(6271): 407-411, 2016 Jan 22.
Article en En | MEDLINE | ID: mdl-26721686
Frame-disrupting mutations in the DMD gene, encoding dystrophin, compromise myofiber integrity and drive muscle deterioration in Duchenne muscular dystrophy (DMD). Removing one or more exons from the mutated transcript can produce an in-frame mRNA and a truncated, but still functional, protein. In this study, we developed and tested a direct gene-editing approach to induce exon deletion and recover dystrophin expression in the mdx mouse model of DMD. Delivery by adeno-associated virus (AAV) of clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 endonucleases coupled with paired guide RNAs flanking the mutated Dmd exon23 resulted in excision of intervening DNA and restored the Dmd reading frame in myofibers, cardiomyocytes, and muscle stem cells after local or systemic delivery. AAV-Dmd CRISPR treatment partially recovered muscle functional deficiencies and generated a pool of endogenously corrected myogenic precursors in mdx mouse muscle.
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transducción Genética / Terapia Genética / Distrofia Muscular de Duchenne / Células Satélite del Músculo Esquelético Límite: Animals Idioma: En Revista: Science Año: 2016 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos
Texto completo
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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Asunto principal: Transducción Genética / Terapia Genética / Distrofia Muscular de Duchenne / Células Satélite del Músculo Esquelético Límite: Animals Idioma: En Revista: Science Año: 2016 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos