Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning-Reference-Cited by-同舟云学术

Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning

Published:2015-09 Issue:3 Volume:26 Page:185-193
ISSN:2324-8637
Container-title:Human Gene Therapy Clinical Development
language:en
Short-container-title:Human Gene Therapy Clinical Development

Author:

Corti Manuela¹,Cleaver Brian¹,Clément Nathalie¹,Conlon Thomas J.¹,Faris Kaitlyn J.¹,Wang Gensheng²,Benson Janet²,Tarantal Alice F.³,Fuller Davis⁴,Herzog Roland W.¹,Byrne Barry J.¹

Affiliation:

1. Department of Pediatrics and Powell Gene Therapy Center, University of Florida, College of Medicine, Gainesville, Florida.

2. Applied Toxicology and Gene Therapy Pharm/Tox Program, Lovelace Respiratory Research Institute, Albuquerque, New Mexico.

3. Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases, California National Primate Research Center, Davis, California; Department of Pediatrics, University of California, Davis, Davis, California; Department of Cell Biology and Human Anatomy, University of California, Davis, Davis, California.

4. Department of Physical Therapy, University of Florida, Gainesville, Florida.

Publisher

Mary Ann Liebert Inc

Subject

Genetics (clinical)

Link

http://www.liebertpub.com/doi/pdf/10.1089/humc.2015.068

Reference37 articles.

1. Respiratory Insufficiency in Adult-Type Acid Maltase Deficiency

2. Clinical features of late-onset Pompe disease: A prospective cohort study

3. Peripheral nerve and neuromuscular junction pathology in Pompe disease

4. Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction

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