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Treatment of type 1 myotonic dystrophy by engineering site-specific RNA endonucleases that target (CUG)(n) repeats.
Zhang, Wenjing; Wang, Yang; Dong, Shuyun; Choudhury, Rajarshi; Jin, Yongfeng; Wang, Zefeng.
Afiliação
  • Zhang W; Institute of Biochemistry, College of Life Sciences, Zhejiang University, Hangzhou, China; Department of Pharmacology and Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
  • Wang Y; Department of Pharmacology and Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
  • Dong S; Department of Pharmacology and Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA; Department of Pharmaceutics and Pharmaceutical Chemistry, University of Utah, Salt Lake City, Utah, USA.
  • Choudhury R; Department of Pharmacology and Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA.
  • Jin Y; Institute of Biochemistry, College of Life Sciences, Zhejiang University, Hangzhou, China.
  • Wang Z; Department of Pharmacology and Lineberger Comprehensive Cancer Center, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, USA; Macro Science Solutions LLP, Chapel Hill, North Carolina, USA. Electronic address: zefeng@med.unc.edu.
Mol Ther ; 22(2): 312-320, 2014 Feb.
Article em En | MEDLINE | ID: mdl-24196578

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Repetições de Trinucleotídeos / Endorribonucleases / Distrofia Miotônica Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2014 Tipo de documento: Article País de afiliação: Estados Unidos

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Repetições de Trinucleotídeos / Endorribonucleases / Distrofia Miotônica Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2014 Tipo de documento: Article País de afiliação: Estados Unidos
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