Advancements in AAV-mediated Gene Therapy for Pompe Disease-Reference-Cited by-同舟云学术

Advancements in AAV-mediated Gene Therapy for Pompe Disease

Published:2020-01-22 Issue:1 Volume:7 Page:15-31
ISSN:2214-3599
Container-title:Journal of Neuromuscular Diseases
language:
Short-container-title:JND

Author:

Salabarria S.M.¹,Nair J.¹,Clement N.¹,Smith B.K.²,Raben N.³,Fuller D.D.²,Byrne B.J.¹,Corti M.¹

Affiliation:

1. Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, Floria, USA

2. Department of Physical Therapy and Center for Respiratory Research and Rehabilitation, University of Florida, Gainesville, Florida, USA

3. Laboratory of Protein Trafficking and Organelle Biology, Cell and Developmental Biology Center, National Heart, Lung and Blood Institute, NIH, Bethesda, Maryland, USA

Publisher

IOS Press

Subject

Neurology (clinical),Neurology

Reference168 articles.

1. Pompe Disease: From Basic Science to Therapy;Kohler;Neurotherapeutics,2018

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

3. Prosser LA , et al. Using Decision Analysis to Support Newborn Screening Policy Decisions: A Case Study for Pompe Disease. MDM Policy Pract. 2018;3(1).

4. The New York pilot newborn screening program for lysosomal storage diseases: Report of the First 65,000 Infants;Wasserstein;Genet Med,2019

5. The genotype-phenotype correlation in Pompe disease;Kroos;Am J Med Genet C Semin Med Genet,2012

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