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Publications by Barry J. Byrne
Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease
Annals of Neurology
Neurology
472. Evaluation of Re-Administration of a Recombinant Adeno-Associated Vector Expressing Acid-Alpha-Glucosidase (rAAV9-DES-hGAA) in Pompe Disease: Preclinical to Clinical Planning
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Glycosylation-Independent Lysosomal Targeting of Acid Α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice
Journal of Biological Chemistry
Biochemistry
Cell Biology
Molecular Biology
AAV9-TAZ Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome
Molecular Therapy - Methods and Clinical Development
Molecular Medicine
Genetics
Molecular Biology
Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells
Molecular Therapy - Methods and Clinical Development
Molecular Medicine
Genetics
Molecular Biology
Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material
Human Gene Therapy
Molecular Medicine
Genetics
Molecular Biology
Dynamic Respiratory Muscle Function in Late-Onset Pompe Disease
Scientific Reports
Multidisciplinary
Hypoglossal Neuropathology and Respiratory Activity in Pompe Mice
Frontiers in Physiology
Physiology
Skeletal Muscle Magnetic Resonance Biomarkers Correlate With Function and Sentinel Events in Duchenne Muscular Dystrophy
PLoS ONE
Multidisciplinary
Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys With Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study
PLoS ONE
Multidisciplinary
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