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AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder.
Argyriou, Catherine; Polosa, Anna; Song, Ji Yun; Omri, Samy; Steele, Bradford; Cécyre, Bruno; McDougald, Devin S; Di Pietro, Erminia; Bouchard, Jean-François; Bennett, Jean; Hacia, Joseph G; Lachapelle, Pierre; Braverman, Nancy E.
Afiliação
  • Argyriou C; Department of Human Genetics, McGill University, Montreal, QC, Canada.
  • Polosa A; Research Institute of the McGill University Health Centre, Montreal, QC, Canada.
  • Song JY; Department of Ophthalmology & Visual Sciences, McGill University, Montreal, QC, Canada.
  • Omri S; Research Institute of the McGill University Health Centre, Montreal, QC, Canada.
  • Steele B; Center for Advanced Retinal and Ocular Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Cécyre B; Research Institute of the McGill University Health Centre, Montreal, QC, Canada.
  • McDougald DS; Department of Biochemistry and Molecular Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
  • Di Pietro E; School of Optometry, Université de Montréal, Montreal, QC, Canada.
  • Bouchard JF; Center for Advanced Retinal and Ocular Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Bennett J; Research Institute of the McGill University Health Centre, Montreal, QC, Canada.
  • Hacia JG; School of Optometry, Université de Montréal, Montreal, QC, Canada.
  • Lachapelle P; Center for Advanced Retinal and Ocular Therapeutics, F.M. Kirby Center for Molecular Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA.
  • Braverman NE; Department of Biochemistry and Molecular Medicine, Keck School of Medicine, University of Southern California, Los Angeles, CA, USA.
Mol Ther Methods Clin Dev ; 23: 225-240, 2021 Dec 10.
Article em En | MEDLINE | ID: mdl-34703844

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Canadá

Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2021 Tipo de documento: Article País de afiliação: Canadá