Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease-Reference-Cited by-同舟云学术

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Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease

Published:2015-06-30 Issue:2 Volume:78 Page:222-234
ISSN:0364-5134
Container-title:Annals of Neurology
language:en
Short-container-title:Ann Neurol.

Author:

Todd Adrian G.¹,McElroy Jessica A.¹,Grange Robert W.²,Fuller David D.³,Walter Glenn A.⁴,Byrne Barry J.¹,Falk Darin J.¹

Affiliation:

1. Department of Pediatrics; University of Florida; Gainesville FL

2. Department of Human Nutrition; Foods and Exercise, Virginia Tech University; Blacksburg VA

3. Department of Physical Therapy; University of Florida; Gainesville FL

4. Department of Physiology and Functional Genomics; University of Florida; Gainesville FL

Funder

National Institutes of Health

Muscular Dystrophy Association

Publisher

Wiley

Subject

Neurology (clinical),Neurology

Link

http://onlinelibrary.wiley.com/wol1/doi/10.1002/ana.24433/fullpdf

Reference49 articles.

1. Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN;Foust;Nat Biotechnol,2010

2. Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease);Raben;Curr Mol Med,2002

3. Pompe's disease;der Ploeg;Lancet,2008

4. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease;Falk;Mol Ther,2013

5. Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors;Mah;Mol Ther,2007

Cited by 48 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. A Comprehensive Update on Late-Onset Pompe Disease;Biomolecules;2023-08-22

2. Current avenues of gene therapy in Pompe disease;Current Opinion in Neurology;2023-07-19

3. Nerve ultrasound for the diagnosis and follow-up of peripheral neuropathies;Current Opinion in Neurology;2023-06-23

4. Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa^−/− mice;Journal of Inherited Metabolic Disease;2023-05-29

5. What’s new and what’s next for gene therapy in Pompe disease?;Expert Opinion on Biological Therapy;2022-04-27

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