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Exons 45-55 Skipping Using Mutation-Tailored Cocktails of Antisense Morpholinos in the DMD Gene.
Echigoya, Yusuke; Lim, Kenji Rowel Q; Melo, Dyanna; Bao, Bo; Trieu, Nhu; Mizobe, Yoshitaka; Maruyama, Rika; Mamchaoui, Kamel; Tanihata, Jun; Aoki, Yoshitsugu; Takeda, Shin'ichi; Mouly, Vincent; Duddy, William; Yokota, Toshifumi.
Afiliação
  • Echigoya Y; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada; Laboratory of Biomedical Science, Department of Veterinary Medicine, College of Bioresource Sciences, Nihon University, Fujisawa, Kanagawa 252-0880, Japan.
  • Lim KRQ; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
  • Melo D; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
  • Bao B; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
  • Trieu N; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
  • Mizobe Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo 187-8502, Japan.
  • Maruyama R; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada.
  • Mamchaoui K; UPMC-Sorbonne Universités-University Paris 6, UPMC/INSERM UMRS974, CNRS FRE 3617, Myology Centre for Research, Paris Cedex 13 75651, France.
  • Tanihata J; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo 187-8502, Japan; Department of Cell Physiology, The Jikei University School of Medicine, Minato, Tokyo 105-8461, Japan.
  • Aoki Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo 187-8502, Japan.
  • Takeda S; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Kodaira, Tokyo 187-8502, Japan.
  • Mouly V; UPMC-Sorbonne Universités-University Paris 6, UPMC/INSERM UMRS974, CNRS FRE 3617, Myology Centre for Research, Paris Cedex 13 75651, France.
  • Duddy W; Northern Ireland Centre for Stratified Medicine, Altnagelvin Hospital Campus, Ulster University, Londonderry BT47 6SB, UK.
  • Yokota T; Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada; Muscular Dystrophy Canada Research Chair, Edmonton, AB T6G 2H7, Canada. Electronic address: toshifum@ualberta.ca.
Mol Ther ; 27(11): 2005-2017, 2019 11 06.
Article em En | MEDLINE | ID: mdl-31416775
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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Regulação da Expressão Gênica / Éxons / Distrofina / Processamento Alternativo / Distrofia Muscular de Duchenne / Morfolinos / Mutação Tipo de estudo: Diagnostic_studies / Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Japão
Texto completo
Imprimir
XML
PubMed Links
Buscar no Google

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Assunto principal: Regulação da Expressão Gênica / Éxons / Distrofina / Processamento Alternativo / Distrofia Muscular de Duchenne / Morfolinos / Mutação Tipo de estudo: Diagnostic_studies / Prognostic_studies Limite: Animals / Humans Idioma: En Revista: Mol Ther Assunto da revista: BIOLOGIA MOLECULAR / TERAPEUTICA Ano de publicação: 2019 Tipo de documento: Article País de afiliação: Japão