Professor of Pediatrics, University Fla College of Medicine
Pediatric Cardiology
Dr. Barry J. Byrne, MD
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Barry J. Byrne, MD practices Pediatric Cardiology. He attended University of Illinois College of Medicine. He over the years received 2 awards: "Regional Top Doctor" and "Fellow (FACC)". Barry J. Byrne, MD is also published. He has 86 publications published. The lastest publication: "Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.'
Publications
- Respiratory motor function in individuals with centronuclear myopathies.
- Diaphragm Pacing as a Rehabilitative Tool for Ventilator-Dependent Pompe Disease: Case Series.
- Altered activation of the diaphragm in late-onset Pompe Disease.
- Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice.
- Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.
- Respiratory motor function in centronuclear myopathy.
- Perspectives on best practices for gene therapy programs.
- Ampakines Stimulate Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease.
- Altered activation of the tibialis anterior in individuals with pompe Disease: Implications for motor unit dysfunction.
- Co-Packaging of Multiple AAV Vectors in a Single Production Step.
- Charting a Clear Path: The ASGCT Standardized Pathways Conference.
- New mechanistic and therapeutic targets for pediatric Heart failure: report from a national Heart, lung, and blood institute working group.
- Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in Pompe mice.
- Adeno-associated virus-mediated gene therapy for metabolic myopathy.
- The respiratory neuromuscular system in Pompe Disease.
- Preclinical Toxicology and Biodistribution Studies of Recombinant Adeno-associated Virus 1 (rAVV1) human acid α-glucosidase (GAA).
- Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease.
- Skeletal Muscles of Ambulant Children with Duchenne Muscular Dystrophy: Validation of Multicenter Study of Evaluation with MR Imaging and MR Spectroscopy.
- B-Cell Depletion and Immunomodulation before Initiation of Enzyme Replacement Therapy Blocks the Immune Response to Acid Alpha-Glucosidase in Infantile-Onset Pompe Dis...
- Phase I/II trial of AAV1-GAA gene therapy to the diaphragm for chronic respiratory failure in Pompe Disease: initial safety and ventilatory outcomes.
- Gene Therapy for Rare Diseases: Summary of a National Institutes of Health Workshop, September 13, 2012.
- Translating the genomics revolution: the need for an international gene therapy consortium for monogenic Diseases.
- Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young-adults with Barth syndrome.
- The Barth Syndrome Registry: distinguishing Disease characteristics and growth data from a longitudinal study.
- A degradable, bioactive, gelatinized alginate hydrogel to improve stem cell/growth factor delivery and facilitate healing after myocardial infarction.
- MRI/MRS evaluation of a female carrier of Duchenne muscular dystrophy.
- Gene Therapy Approaches for Lysosomal Storage Disease: Next Generation Treatment.
- Retrograde gene delivery to hypoglossal motoneurons using AAV9.
- Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy.
- Structural insight into the unique properties of Adeno-Associated Virus Serotype 9.
- Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9.
- Mapping the T helper cell response to acid α-glucosidase in Pompe mice.
- Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice.
- Gene Therapy for Leber Congenital Amaurosis Caused by RPE65 Mutations: Safety and Efficacy in 15 Children and Adults Followed Up to 3 Years.
- Impaired cardiac reserve and severely diminished skeletal muscle O? utilization mediate exercise intolerance in Barth syndrome.
- AAV Vectors for Cardiac Gene Transfer: Experimental Tools and Clinical Opportunities.
- Characterization of a Transgenic Short Hairpin RNA-Induced Murine Model of Tafazzin Deficiency.
- Pompe Disease: Design, methodology, and early findings from the Pompe Registry.
- A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo.
- AAV6-mediated gene silencing fALS short.
- Glycogen storage Disease type ia in canines: a model for human metabolic and genetic liver Disease.
- Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
- Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material.
- Research priorities in hypertrophic cardiomyopathy: report of a Working Group of the National Heart, Lung, and Blood Institute.
- PRX-08066, a novel 5-hydroxytryptamine receptor 2B antagonist, reduces monocrotaline-induced pulmonary arterial Hypertension and right ventricular hypertrophy in rats.
- Adeno-associated virus-mediated correction of a canine model of glycogen storage Disease type Ia.
- Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established Disease.
- The effect of DNA-dependent protein kinase on adeno-associated virus replication.
- Innovative vector design: cross-packaged, self-complementary and now trans-splicing AAV vectors.
- Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy.
- Human RPE65 gene therapy for Leber Congenital amaurosis: persistence of early visual improvements and safety at 1 year.
- Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins.
- Vision 1 year after gene therapy for Leber's Congenital amaurosis.
- Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies.
- Neural deficits contribute to respiratory insufficiency in Pompe Disease.
- Psychosocial Functioning in Youth with Barth Syndrome.
- Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity.
- Treatment of leber Congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I tr...
- Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.
- Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees.
- Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5.
- Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D.
- Physiological correction of Pompe Disease by systemic delivery of adeno-associated virus serotype 1 vectors.
- Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and disseminatio...
- Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults.
- Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo.
- Cardiac and clinical phenotype in Barth syndrome.
- Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system.
- Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector.
- Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors.
- Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage dise...
- Noninvasive monitoring and tracking of muscle stem cell transplants.
- Non-invasive analysis of myoblast transplants in rodent cardiac muscle.
- A new method for recombinant adeno-associated virus vector delivery to murine diaphragm.
- Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults.
- Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice.
- Cell therapy in the Heart: cell production, transplantation, and applications.
- Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors.
- Improved method of recombinant AAV2 delivery for systemic targeted gene therapy.
- Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization.
- Correction of the enzymatic and functional deficits in a model of Pompe Disease using adeno-associated virus vectors.
- Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine Heart.
- Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with duchenne muscular dystrophy: a multicenter cross sectional study.
- Targeted approaches to induce immune tolerance for Pompe Disease therapy.
- Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent.
Schools
University Of Illinois College Of Medicine Chicago
Johns Hopkins Hospital
Procedures Preformed
- Cardiac Imaging
- Chest CT (incl. Heart and Lungs)
Conditions Treated
- Cardiomegaly
- Congenital Heart Disease
- Congestive Heart Failure
- Hypertension
Doctors Specialties
- Pediatric Cardiology
Accepted Insurances
Awards
- Regional Top Doctor
- Fellow (FACC)
Education
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Johns Hopkins University -
University of Illinois College of Medicine
Hospital
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UF Health Shands Hospital
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