AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice-Reference-Cited by-同舟云学术

AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice

Published:2019-12 Issue: Volume:15 Page:194-203
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Doyle Brendan M.,Turner Sara M.F.,Sunshine Michael D.,Doerfler Phillip A.,Poirier Amy E.,Vaught Lauren A.,Jorgensen Marda L.,Falk Darin J.^ORCID,Byrne Barry J.,Fuller David D.

Funder

NIH

University of Florida Graduate School

NIAMS

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference52 articles.

1. The respiratory neuromuscular system in Pompe disease;Fuller;Respir. Physiol. Neurobiol.,2013

2. Neural deficits contribute to respiratory insufficiency in Pompe disease;DeRuisseau;Proc. Natl. Acad. Sci. USA,2009

3. Pompe disease gene therapy;Byrne;Hum. Mol. Genet.,2011

4. Oropharyngeal dysphagia in infants and children with infantile Pompe disease;Jones;Dysphagia,2010

5. Obstructive sleep apnea syndrome in acid maltase deficiency;Margolis;Chest,1994

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