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The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1.
Batra, Ranjan; Nelles, David A; Roth, Daniela M; Krach, Florian; Nutter, Curtis A; Tadokoro, Takahiro; Thomas, James D; Sznajder, Lukasz J; Blue, Steven M; Gutierrez, Haydee L; Liu, Patrick; Aigner, Stefan; Platoshyn, Oleksandr; Miyanohara, Atsushi; Marsala, Martin; Swanson, Maurice S; Yeo, Gene W.
Afiliação
  • Batra R; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, USA.
  • Nelles DA; Stem Cell Program, University of California San Diego, La Jolla, CA, USA.
  • Roth DM; Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA.
  • Krach F; Locanabio, La Jolla, CA, USA.
  • Nutter CA; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, USA.
  • Tadokoro T; Stem Cell Program, University of California San Diego, La Jolla, CA, USA.
  • Thomas JD; Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA.
  • Sznajder LJ; Locanabio, La Jolla, CA, USA.
  • Blue SM; Locanabio, La Jolla, CA, USA.
  • Gutierrez HL; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, USA.
  • Liu P; Stem Cell Program, University of California San Diego, La Jolla, CA, USA.
  • Aigner S; Institute for Genomic Medicine, University of California San Diego, La Jolla, CA, USA.
  • Platoshyn O; Department of Molecular Genetics and Microbiology, Center for NeuroGenetics and the Genetics Institute, University of Florida, College of Medicine, Gainesville, FL, USA.
  • Miyanohara A; Department of Anesthesiology, School of Medicine, University of California San Diego, La Jolla, CA, USA.
  • Marsala M; Department of Molecular Genetics and Microbiology, Center for NeuroGenetics and the Genetics Institute, University of Florida, College of Medicine, Gainesville, FL, USA.
  • Swanson MS; Department of Molecular Genetics and Microbiology, Center for NeuroGenetics and the Genetics Institute, University of Florida, College of Medicine, Gainesville, FL, USA.
  • Yeo GW; Department of Cellular and Molecular Medicine, University of California San Diego, La Jolla, CA, USA.
Nat Biomed Eng ; 5(2): 157-168, 2021 02.
Article em En | MEDLINE | ID: mdl-32929188
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: RNA / Sistemas CRISPR-Cas / Edição de Genes / Proteína 9 Associada à CRISPR / Distrofia Miotônica Limite: Animals Idioma: En Revista: Nat Biomed Eng Ano de publicação: 2021 Tipo de documento: Article
Texto completo
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PubMed Links
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: RNA / Sistemas CRISPR-Cas / Edição de Genes / Proteína 9 Associada à CRISPR / Distrofia Miotônica Limite: Animals Idioma: En Revista: Nat Biomed Eng Ano de publicação: 2021 Tipo de documento: Article