Adeno-Associated Virus–Mediated Gene Therapy for Metabolic Myopathy

Phase I/II Trial of Adeno-Associated Virus–Mediated Alpha-Glucosidase Gene Therapy to the Diaphragm for Chronic Respiratory Failure in Pompe Disease: Initial Safety and Ventilatory Outcomes

(2013) Barbara K. Smith et al.   HUMAN GENE THERAPY

Site-specific Mtm1 mutagenesis by an AAV-Cre vector reveals that myotubularin is essential in adult muscle

(2013) Romain Joubert et al.   HUMAN MOLECULAR GENETICS

Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease

(2013) Darin J Falk et al.   MOLECULAR THERAPY

Severity of cardiomyopathy associated with adenine nucleotide translocator-1 deficiency correlates with mtDNA haplogroup

(2013) Kevin A. Strauss et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Advancing Translational Research Through the NHLBI Gene Therapy Resource Program (GTRP)

(2013) Cheryl L. McDonald et al.   Human Gene Therapy Clinical Development

Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

(2012) Barry J. Byrne et al.   HUMAN GENE THERAPY

Cell and Gene Therapy for Genetic Diseases: Inherited Disorders Affecting the Lung and Those Mimicking Sudden Infant Death Syndrome

(2012) Allison M. Keeler et al.   HUMAN GENE THERAPY

Center for Fetal Monkey Gene Transfer for Heart, Lung, and Blood Diseases: An NHLBI Resource for the Gene Therapy Community

(2012) Alice F. Tarantal et al.   HUMAN GENE THERAPY

Long-term Correction of Very Long-chain Acyl-CoA Dehydrogenase Deficiency in Mice Using AAV9 Gene Therapy

(2012) Allison M Keeler et al.   MOLECULAR THERAPY

The AAV Vector Toolkit: Poised at the Clinical Crossroads

(2012) Aravind Asokan et al.   MOLECULAR THERAPY

Muscle function in A canine model of X-linked myotubular myopathy

(2012) Robert W. Grange et al.   MUSCLE & NERVE

Retrograde Gene Delivery to Hypoglossal Motoneurons Using Adeno-Associated Virus Serotype 9

(2012) Mai K. ElMallah et al.   Human Gene Therapy Methods

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

(2011) S R Bacman et al.   GENE THERAPY

Pompe disease gene therapy

(2011) B. J. Byrne et al.   HUMAN MOLECULAR GENETICS

Rapid progressive course of later-onset Pompe disease in Chinese patients

(2011) Chih-Chao Yang et al.   MOLECULAR GENETICS AND METABOLISM

Pompe disease: Design, methodology, and early findings from the Pompe Registry

(2011) Barry J. Byrne et al.   MOLECULAR GENETICS AND METABOLISM

Hypoglossal Neuropathology and Respiratory Activity in Pompe Mice

(2011) Kun-Ze Lee et al.   Frontiers in Physiology

Translational Research for Cardiovascular Diseases at the National Heart, Lung, and Blood Institute

(2010) Michael S. Lauer et al.   CIRCULATION

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

(2010) S R Bacman et al.   GENE THERAPY

The next step in gene delivery: Molecular engineering of adeno-associated virus serotypes

(2010) Jinhui Wang et al.   JOURNAL OF MOLECULAR AND CELLULAR CARDIOLOGY

Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease

(2010) Cathryn S Mah et al.   MOLECULAR THERAPY

MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers

(2010) A. H. Beggs et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins

(2009) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Long-Term Luciferase Expression Monitored by Bioluminescence Imaging After Adeno-Associated Virus-Mediated Fetal Gene Delivery in Rhesus Monkeys (Macaca mulatta)

(2009) Alice F. Tarantal et al.   HUMAN GENE THERAPY

Biochemical Correction of Very Long–chain Acyl-CoA Dehydrogenase Deficiency Following Adeno-associated Virus Gene Therapy

(2009) J. Lawrence Merritt et al.   MOLECULAR THERAPY

Neural deficits contribute to respiratory insufficiency in Pompe disease

(2009) L. R. DeRuisseau et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy

(2009) M. L. Brantly et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Intramuscular Administration of AAV1-Lipoprotein Lipase S447X Lowers Triglycerides in Lipoprotein Lipase–Deficient Patients

(2008) Erik S. Stroes et al.   ARTERIOSCLEROSIS THROMBOSIS AND VASCULAR BIOLOGY

Adeno-Associated Virus (AAV) Serotype 9 Provides Global Cardiac Gene Transfer Superior to AAV1, AAV6, AAV7, and AAV8 in the Mouse and Rat

(2008) Lawrence T. Bish et al.   HUMAN GENE THERAPY

Modulation of glycogen synthesis by RNA interference: towards a new therapeutic approach for glycogenosis type II

(2008) Gaelle Douillard-Guilloux et al.   HUMAN MOLECULAR GENETICS

AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis

(2008) Anna Buj-Bello et al.   HUMAN MOLECULAR GENETICS

Recombinant adeno-associated virus-mediated gene delivery of long chain acyl coenzyme A dehydrogenase (LCAD) into LCAD-deficient mice

(2008) Stuart G. Beattie et al.   JOURNAL OF GENE MEDICINE

Correction of Multiple Striated Muscles in Murine Pompe Disease Through Adeno-associated Virus–mediated Gene Therapy

(2008) Baodong Sun et al.   MOLECULAR THERAPY

Self-complementary AAV Vectors; Advances and Applications

(2008) Douglas M McCarty   MOLECULAR THERAPY

Analysis of AAV Serotypes 1–9 Mediated Gene Expression and Tropism in Mice After Systemic Injection

(2008) Carmela Zincarelli et al.   MOLECULAR THERAPY

Adenovirus and adeno-associated virus-mediated delivery of human myophosphorylase cDNA and LacZ cDNA to muscle in the ovine model of McArdle’s disease: Expression and re-expression of glycogen phosphorylase

(2008) J.McC. Howell et al.   NEUROMUSCULAR DISORDERS

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

(2008) L. Zhong et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA