Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III-Reference-Cited by-同舟云学术

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Challenges of Gene Therapy for the Treatment of Glycogen Storage Diseases Type I and Type III

Published:2019-10-01 Issue:10 Volume:30 Page:1263-1273
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Jauze Louisa¹²³⁴,Monteillet Laure²³⁴,Mithieux Gilles²³⁴,Rajas Fabienne²³⁴,Ronzitti Giuseppe¹

Affiliation:

1. INTEGRARE, Genethon, Inserm, Université d'Evry, Université Paris-Saclay, Evry, France

2. Institut National de la Santé et de la Recherche Médicale, U1213, Lyon, France

3. Université de Lyon, Lyon, France

4. Université Lyon I, Villeurbanne, France.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2019.102

Reference96 articles.

1. Gut-Brain Glucose Signaling in Energy Homeostasis

2. Glycogen metabolism and glycogen storage disorders

3. Hepatic glycogen storage disorders

4. Skeletal muscle disorders of glycogenolysis and glycolysis

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2. Impaired Glucose Metabolism, Primary Cilium Defects, and Kidney Cystogenesis in Glycogen Storage Disease Type Ia;Journal of the American Society of Nephrology;2024-08-14

3. A functional mini-GDE transgene corrects impairment in models of glycogen storage disease type III;Journal of Clinical Investigation;2024-01-16

4. Liver Systems in Safety Pharmacology;Drug Discovery and Evaluation: Safety and Pharmacokinetic Assays;2024

5. Efficient and reproducible generation of human induced pluripotent stem cell-derived expandable liver organoids for disease modeling;Scientific Reports;2023-12-22

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