Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy.

Shoti, Jakob; Qing, Keyun; Keeler, Geoffrey D; Duan, Dongsheng; Byrne, Barry J; Srivastava, Arun

Shoti, Jakob; Qing, Keyun; Keeler, Geoffrey D; Duan, Dongsheng; Byrne, Barry J; Srivastava, Arun.

Afiliação

Shoti J; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
Qing K; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
Keeler GD; Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
Duan D; Departments of Microbiology and Immunology, Neurology, Biomedical Sciences, and Chemical and Biomedical Engineering, University of Missouri, Columbia, MO, USA.
Byrne BJ; Child Health Research Institute, Department of Pediatrics, University of Florida College of Medicine, Gainesville, FL, USA.
Srivastava A; Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL, USA.

Mol Ther Methods Clin Dev ; 31: 101147, 2023 Dec 14.

Article em En | MEDLINE | ID: mdl-38046199

Palavras-chave

AAV vectors; gene expression; gene therapy; gene transfer; muscle diseases, capsid-modifications, genome-modifications

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Texto completo: 1 Coleções: 01-internacional Base de dados: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Ano de publicação: 2023 Tipo de documento: Article País de afiliação: Estados Unidos

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