Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy-Reference-Cited by-同舟云学术

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Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy

Published:2013-11 Issue:11 Volume:24 Page:928-936
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Mah Cathryn S.¹,Soustek Meghan S.¹,Todd A. Gary¹,McCall Angela¹,Smith Barbara K.²,Corti Manuela¹,Falk Darin J.¹,Byrne Barry J.¹

Affiliation:

1. Powell Gene Therapy Center, Department of Pediatrics, College of Medicine, University of Florida, Gainesville, FL 32610.

2. Department of Physical Therapy, College of Public Health and Health Professions, University of Florida, Gainesville, FL 32610.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2013.2514

Reference84 articles.

1. Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid- -glucosidase

2. The AAV Vector Toolkit: Poised at the Clinical Crossroads

3. Organ-specific shifts in mtDNA heteroplasmy following systemic delivery of a mitochondria-targeted restriction endonuclease

4. Manipulation of mtDNA heteroplasmy in all striated muscles of newborn mice by AAV9-mediated delivery of a mitochondria-targeted restriction endonuclease

5. Lack of Complex I Activity in Human Cells Carrying a Mutation in MtDNA-encoded ND4 Subunit Is Corrected by theSaccharomyces cerevisiae NADH-Quinone Oxidoreductase (NDI1) Gene

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1. Targeting mitochondria in the regulation of neurodegenerative diseases: A comprehensive review;Journal of Neuroscience Research;2022-07-20

2. Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease;Human Gene Therapy;2016-01

3. A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform;Molecular Therapy - Methods & Clinical Development;2016

4. Targeted approaches to induce immune tolerance for Pompe disease therapy;Molecular Therapy - Methods & Clinical Development;2016

5. Strategies for treating mitochondrial disorders: An update;Molecular Genetics and Metabolism;2014-12

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