Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease-Reference-Cited by-同舟云学术

Intravenous Injection of an AAV-PHP.B Vector Encoding Human Acid α-Glucosidase Rescues Both Muscle and CNS Defects in Murine Pompe Disease

Published:2019-03 Issue: Volume:12 Page:233-245
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Lim Jeong-A,Yi Haiqing,Gao Fengqin,Raben Nina,Kishnani Priya S.,Sun Baodong

Funder

Alice and Y.T. Chen Pediatric Genetics and Genomics Center at Duke University

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference87 articles.

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3. Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease;Pauly;Hum. Gene Ther.,2001

4. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003

5. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J. Pediatr.,2006

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