What’s new and what’s next for gene therapy in Pompe disease?-Reference-Cited by-同舟云学术

What’s new and what’s next for gene therapy in Pompe disease?

Published:2022-04-27 Issue:9 Volume:22 Page:1117-1135
ISSN:1471-2598
Container-title:Expert Opinion on Biological Therapy
language:en
Short-container-title:Expert Opinion on Biological Therapy

Author:

Roger Angela L.¹,Sethi Ronit¹,Huston Meredith L.¹,Scarrow Evelyn¹,Bao-Dai Joy¹,Lai Elias¹,Biswas Debolina D.¹,El Haddad Léa¹,Strickland Laura M.¹,Kishnani Priya S.²,ElMallah Mai K.¹

Affiliation:

1. Division of Pulmonary Medicine, Department of Pediatrics, Duke University Medical Center Box 2644, Durham, North Carolina, 27710, USA

2. Division of Medical Genetics, Department of Pediatrics, Duke University, Durham, North Carolina USA

Funder

Pfizer-NCBiotech Distinguished Postdoctoral Fellowship in Gene Therapy

Publisher

Informa UK Limited

Subject

Clinical Biochemistry,Drug Discovery,Pharmacology

Link

https://www.tandfonline.com/doi/pdf/10.1080/14712598.2022.2067476

Reference195 articles.

1. α-Glucosidase deficiency in generalized glycogen-storage disease (Pompe's disease)

2. Pompe's disease

3. Pompe disease: Design, methodology, and early findings from the Pompe Registry

4. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

5. Neural deficits contribute to respiratory insufficiency in Pompe disease

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1. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12

2. Monitoring and Management of Respiratory Function in Pompe Disease: Current Perspectives;Therapeutics and Clinical Risk Management;2023-09

3. AAV-mediated delivery of secreted acid α-glucosidase with enhanced uptake corrects neuromuscular pathology in Pompe mice;JCI Insight;2023-08-22

4. An enhancer-based gene-therapy strategy for spatiotemporal control of cargoes during tissue repair;Cell Stem Cell;2023-01