Bezafibrate Enhances AAV Vector-Mediated Genome Editing in Glycogen Storage Disease Type Ia.

Kang, Hye-Ri; Waskowicz, Lauren; Seifts, Andrea M; Landau, Dustin J; Young, Sarah P; Koeberl, Dwight D

Kang, Hye-Ri; Waskowicz, Lauren; Seifts, Andrea M; Landau, Dustin J; Young, Sarah P; Koeberl, Dwight D.

Afiliación

Kang HR; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Waskowicz L; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Seifts AM; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Landau DJ; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Young SP; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Koeberl DD; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Mol Ther Methods Clin Dev ; 13: 265-273, 2019 Jun 14.

Article en En | MEDLINE | ID: mdl-30859111

Palabras clave

adeno-associated virus; autophagy; bezafibrate; gene therapy; genome editing; glycogen storage disease type Ia; pan-agonist of peroxisome proliferator-activated receptors; zinc-finger nuclease

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Texto completo: 1 Colección: 01-internacional Base de datos: MEDLINE Idioma: En Revista: Mol Ther Methods Clin Dev Año: 2019 Tipo del documento: Article País de afiliación: Estados Unidos Pais de publicación: Estados Unidos

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