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Full-length human dystrophin on human artificial chromosome compensates for mouse dystrophin deficiency in a Duchenne muscular dystrophy mouse model.
Hiramuki, Yosuke; Abe, Satoshi; Uno, Narumi; Kazuki, Kanako; Takata, Shuta; Miyamoto, Hitomaru; Takayama, Haruka; Morimoto, Kayoko; Takehara, Shoko; Osaki, Mitsuhiko; Tanihata, Jun; Takeda, Shin'ichi; Tomizuka, Kazuma; Oshimura, Mitsuo; Kazuki, Yasuhiro.
Afiliação
  • Hiramuki Y; Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Abe S; Trans Chromosomics Inc., 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Uno N; Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Kazuki K; Department of Chromosome Biomedical Engineering, School of Life Science, Faculty of Medicine, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Takata S; Laboratory of Bioengineering, Faculty of Life Sciences, Tokyo University of Pharmacy and Life Sciences, 1432-1 Horinouchi, Hachioji, Tokyo, 192-0392, Japan.
  • Miyamoto H; Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Takayama H; Department of Chromosome Biomedical Engineering, School of Life Science, Faculty of Medicine, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Morimoto K; Department of Chromosome Biomedical Engineering, Integrated Medical Sciences, Graduate School of Medical Sciences, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Takehara S; Department of Biomedical Science, Institute of Regenerative Medicine and Biofunction, Graduate School of Medical Sciences, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Osaki M; Trans Chromosomics Inc., 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Tanihata J; Trans Chromosomics Inc., 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Takeda S; Trans Chromosomics Inc., 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Tomizuka K; Chromosome Engineering Research Center, Tottori University, 86 Nishi-cho, Yonago, Tottori, 683-8503, Japan.
  • Oshimura M; Division of Experimental Pathology, Department of Functional Morphology, Faculty of Medicine, Tottori University, Yonago, Tottori, 683­8503, Japan.
  • Kazuki Y; Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (NCNP), Kodaira, Tokyo, 187-8502, Japan.
Sci Rep ; 13(1): 4360, 2023 03 16.
Article em En | MEDLINE | ID: mdl-36928364

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne / Cromossomos Artificiais Humanos Idioma: En Ano de publicação: 2023 Tipo de documento: Article

Texto completo: 1 Base de dados: MEDLINE Assunto principal: Distrofina / Distrofia Muscular de Duchenne / Cromossomos Artificiais Humanos Idioma: En Ano de publicação: 2023 Tipo de documento: Article