Screening data from 19 patients with late-onset Pompe disease for a phase I clinical trial of AAV8 vector-mediated gene therapy.

Hannah, William B; Case, Laura E; Smith, Edward C; Walters, Crista; Bali, Deeksha; Kishnani, Priya S; Koeberl, Dwight D

Hannah, William B; Case, Laura E; Smith, Edward C; Walters, Crista; Bali, Deeksha; Kishnani, Priya S; Koeberl, Dwight D.

Afiliação

Hannah WB; Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.
Case LE; Doctor of Physical Therapy Division, Department of Orthopedics Duke University School of Medicine Durham North Carolina USA.
Smith EC; Division of Neurology, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.
Walters C; Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.
Bali D; Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.
Kishnani PS; Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.
Koeberl DD; Division of Medical Genetics, Department of Pediatrics Duke University School of Medicine Durham North Carolina USA.

JIMD Rep ; 64(5): 393-400, 2023 Sep.

Article em En | MEDLINE | ID: mdl-37701327

Palavras-chave

Pompe disease; adenoassociated virus; gene therapy

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Texto completo: 1 Base de dados: MEDLINE Tipo de estudo: Diagnostic_studies / Prognostic_studies / Screening_studies Idioma: En Ano de publicação: 2023 Tipo de documento: Article

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