Gene therapy for glycogen storage diseases.

Kishnani, Priya S; Sun, Baodong; Koeberl, Dwight D

Kishnani, Priya S; Sun, Baodong; Koeberl, Dwight D.

Affiliation

Kishnani PS; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.
Sun B; Department of Molecular Genetics and Microbiology, Duke University, Durham, NC 27710, USA.
Koeberl DD; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC 27710, USA.

Hum Mol Genet ; 28(R1): R31-R41, 2019 10 01.

Article in En | MEDLINE | ID: mdl-31227835

Subject(s)

Genetic Therapy; Glycogen Storage Disease/genetics; Glycogen Storage Disease/therapy; Animals; Biomarkers; Clinical Trials as Topic; Combined Modality Therapy; Gene Editing; Gene Expression Regulation; Genetic Predisposition to Disease; Genetic Therapy/adverse effects; Genetic Therapy/methods; Genetic Vectors/genetics; Glycogen Storage Disease/metabolism; Humans; Immunomodulation; Liver/metabolism; Organ Specificity; Standard of Care; Transduction, Genetic; Transgenes; Treatment Outcome

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Glycogen Storage Disease Limits: Animals / Humans Language: En Journal: Hum Mol Genet Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2019 Type: Article Affiliation country: United States

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