Barry Byrne

Summary

Affiliation: University of Florida
Country: USA

Publications

  1. pmc An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery
    Ni Chung Lee
    Departments of Pediatrics and Medical Genetics, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan
    Genet Vaccines Ther 10:3. 2012
  2. pmc Gene therapy approaches for lysosomal storage disease: next-generation treatment
    Barry J Byrne
    Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA
    Hum Gene Ther 23:808-15. 2012
  3. pmc Pompe disease gene therapy
    Barry J Byrne
    Department of Pediatrics, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA
    Hum Mol Genet 20:R61-8. 2011
  4. doi request reprint Pompe disease: design, methodology, and early findings from the Pompe Registry
    Barry J Byrne
    Department of Pediatrics, University of Florida, College of Medicine, PO Box 100296, Gainesville, FL 32610, USA
    Mol Genet Metab 103:1-11. 2011
  5. pmc Structure of adeno-associated virus serotype 8, a gene therapy vector
    Hyun Joo Nam
    Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA
    J Virol 81:12260-71. 2007
  6. pmc Structural studies of adeno-associated virus serotype 8 capsid transitions associated with endosomal trafficking
    Hyun Joo Nam
    Department of Biochemistry and Molecular Biology, 1600 SW Archer Road, P O Box 100245, Gainesville, FL 32610 0266, USA
    J Virol 85:11791-9. 2011
  7. ncbi request reprint Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome
    R Rodney Howell
    Department of Pediatrics, Miller School of Medicine, University of Miami, Miami, FL 33101, USA
    Genet Med 8:289-96. 2006
  8. pmc Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8
    Michael Douglas Lane
    Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA
    Acta Crystallogr Sect F Struct Biol Cryst Commun 61:558-61. 2005
  9. ncbi request reprint Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector
    Matthias Kapturczak
    Department of Medicine, University of Florida, Gainesville, Florida 32610, USA
    Mol Ther 5:154-60. 2002
  10. ncbi request reprint Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart
    Catalin Toma
    Department of Medicine, Division of Cardiology, Johns Hopkins School of Medicine, Baltimore, MD, USA
    Circulation 105:93-8. 2002

Research Grants

Collaborators

Detail Information

Publications39

  1. pmc An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery
    Ni Chung Lee
    Departments of Pediatrics and Medical Genetics, National Taiwan University Hospital and National Taiwan University College of Medicine, Taipei, Taiwan
    Genet Vaccines Ther 10:3. 2012
    ..abstract:..
  2. pmc Gene therapy approaches for lysosomal storage disease: next-generation treatment
    Barry J Byrne
    Department of Pediatrics and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA
    Hum Gene Ther 23:808-15. 2012
    ..In this review, we discuss the various viral vector systems that have been developed and some of the strategy designs for the treatment of LSDs...
  3. pmc Pompe disease gene therapy
    Barry J Byrne
    Department of Pediatrics, University of Florida, 1600 SW Archer Road, Gainesville, FL 32610, USA
    Hum Mol Genet 20:R61-8. 2011
    ..We will cover the preliminary laboratory studies and rationale for a clinical trial, which is based on the treatment of the high rate of respiratory failure in the early-onset patients receiving ERT...
  4. doi request reprint Pompe disease: design, methodology, and early findings from the Pompe Registry
    Barry J Byrne
    Department of Pediatrics, University of Florida, College of Medicine, PO Box 100296, Gainesville, FL 32610, USA
    Mol Genet Metab 103:1-11. 2011
    ..As the largest dataset on Pompe disease to date, the Pompe Registry will serve to improve recognition of the disease, enhance understanding of the variable disease course, and offer insights into treated and untreated disease course...
  5. pmc Structure of adeno-associated virus serotype 8, a gene therapy vector
    Hyun Joo Nam
    Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA
    J Virol 81:12260-71. 2007
    ..Thus, this AAV8 structure provides an additional platform for mutagenesis efforts to characterize AAV capsid regions responsible for differential cellular tropism, transduction, and antigenicity for these promising gene therapy vectors...
  6. pmc Structural studies of adeno-associated virus serotype 8 capsid transitions associated with endosomal trafficking
    Hyun Joo Nam
    Department of Biochemistry and Molecular Biology, 1600 SW Archer Road, P O Box 100245, Gainesville, FL 32610 0266, USA
    J Virol 85:11791-9. 2011
    ..This disruption points to capsid destabilization which may (i) release VP1 amino acids for its phospholipase A2 function for endosomal escape and nuclear localization signals for nuclear targeting and (ii) trigger genome uncoating...
  7. ncbi request reprint Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome
    R Rodney Howell
    Department of Pediatrics, Miller School of Medicine, University of Miami, Miami, FL 33101, USA
    Genet Med 8:289-96. 2006
    ..This article presents a unified view on the optimal approach to the accurate diagnosis of Pompe disease and to its recognition as one of the possible and treatable causes of floppy baby syndrome...
  8. pmc Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8
    Michael Douglas Lane
    Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA
    Acta Crystallogr Sect F Struct Biol Cryst Commun 61:558-61. 2005
    ..5, c = 443.5 A. The unit cell contains two viral particles, with ten capsid viral protein monomers per crystallographic asymmetric unit...
  9. ncbi request reprint Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector
    Matthias Kapturczak
    Department of Medicine, University of Florida, Gainesville, Florida 32610, USA
    Mol Ther 5:154-60. 2002
    ..These data are the first report of efficient islet cell transduction with two genes using a single bicistronic rAAV vector and have direct implications for strategies aimed at enhancing islet transplant survival...
  10. ncbi request reprint Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart
    Catalin Toma
    Department of Medicine, Division of Cardiology, Johns Hopkins School of Medicine, Baltimore, MD, USA
    Circulation 105:93-8. 2002
    ..We investigated the potential of human mesenchymal stem cells (hMSCs) from adult bone marrow to undergo myogenic differentiation once transplanted into the adult murine myocardium...
  11. ncbi request reprint Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults
    Mark L Brantly
    Department of Medicine, University of Florida, Gainesville, FL 32611, USA
    Hum Gene Ther 17:1177-86. 2006
    ....
  12. ncbi request reprint Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors
    Cathryn Mah
    Powell Gene Therapy Center, University of Florida, Gainesville, Florida, USA
    Mol Ther 15:501-7. 2007
    ....
  13. ncbi request reprint Preclinical characterization of a recombinant adeno-associated virus type 1-pseudotyped vector demonstrates dose-dependent injection site inflammation and dissemination of vector genomes to distant sites
    Terence R Flotte
    Powell Gene Therapy Center, Department of Pediatrics, University of Florida, Gainesville, FL 32610, USA
    Hum Gene Ther 18:245-56. 2007
    ..The copy number of vector DNA in the blood and semen declined over time throughout the study. These two dose-dependent findings have served to guide to the design of a phase 1 human trial of rAAV1-AAT...
  14. ncbi request reprint Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D
    Christina A Pacak
    Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, Florida 32610, USA
    Mol Ther 15:1775-81. 2007
    ..In summary, AAV/AAV-sgca gene transfer provides long-term muscle protection from LGMD and can be non-invasively evaluated using magnetic resonance imaging...
  15. ncbi request reprint Improved method of recombinant AAV2 delivery for systemic targeted gene therapy
    Cathryn Mah
    Department of Pediatrics, University of Florida, Gainesville, Florida 32610, USA
    Mol Ther 6:106-12. 2002
    ..These studies demonstrate a novel method to deliver rAAV vectors more effectively that could prove to be a successful alternative mode of virus-mediated human gene therapy...
  16. pmc Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5
    Steven Polyak
    Division of Gastroenterology, Department of Medicine, University of Florida, Gainesville, FL 32610, USA
    Dig Dis Sci 53:1261-70. 2008
    ....
  17. doi request reprint Update of the Pompe disease mutation database with 107 sequence variants and a format for severity rating
    Marian Kroos
    Departments of Clinical Genetics and Pediatrics, Erasmus MC, Rotterdam, The Netherlands
    Hum Mutat 29:E13-26. 2008
    ..Further, this article introduces a tool to rate the various mutations by severity, which will improve understanding of the genotype-phenotype correlation and facilitate the diagnosis and prognosis in Pompe disease...
  18. pmc Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees
    Terence R Flotte
    Department of Pediatrics, University of Massachusetts Medical School, Worcester, MA 01655, USA
    Hum Gene Ther 19:681-9. 2008
    ..These data indicate an increased susceptibility to subclinical liver toxicity from portal vein injection of rAAV2 in the presence of HCV infection...
  19. ncbi request reprint Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization
    Maria B Grant
    Program in Stem Cell Biology, University of Florida, Gainesville, Florida, USA
    Nat Med 8:607-12. 2002
    ..We also show that recruitment of endothelial precursors to sites of ischemic injury has a significant role in neovascularization...
  20. ncbi request reprint Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors
    Thomas J Fraites
    Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, Florida 32610, USA
    Mol Ther 5:571-8. 2002
    ....
  21. ncbi request reprint Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease)
    Nina Raben
    Arthritis and Rheumatism Branch, National Institute of Arthritis and Musculoskeletal and Skin Diseases, National Institutes of Health, Bethesda, MD 20892, USA
    Curr Mol Med 2:145-66. 2002
    ..We will also highlight some emerging questions following the introduction of enzyme replacement therapy...
  22. ncbi request reprint Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo
    Christina A Pacak
    Center for Fetal Monkey Gene Transfer for Heart, Lung and Blood Diseases, California National Primate Research Center, Davis, USA
    Circ Res 99:e3-9. 2006
    ....
  23. ncbi request reprint Cardiac and clinical phenotype in Barth syndrome
    Carolyn T Spencer
    Congenital Heart Center, University of Florida College of Medicine, PO Box 100296, Gainesville, FL 32610 0296, USA
    Pediatrics 118:e337-46. 2006
    ..We sought to evaluate extent of cardioskeletal myopathy, potential for arrhythmia, delays in growth, and biochemical correlates of disease severity in patients with this disorder...
  24. ncbi request reprint Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice
    Mao Sen Sun
    Section on Cellular Differentiation, Heritable Disorders Branch, National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, MD 20892, USA
    Hum Mol Genet 11:2155-64. 2002
    ..Our results suggest that human GSD-Ia would be treatable by gene therapy...
  25. ncbi request reprint Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice
    Cathryn Mah
    Department of Pediatrics, Department of Molecular Genetics and Microbiology, and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610 0266, USA
    Hum Gene Ther 14:143-52. 2003
    ..Because the murine form of the gene was used in the mouse model, less than 1 Bethesda unit of inhibitors was noted. This work demonstrates the feasibility of using rAAV vectors for the long-term treatment of hemophilia A...
  26. ncbi request reprint Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults
    Terence R Flotte
    Department of Pediatrics, University of Florida, Gainesville, USA
    Hum Gene Ther 15:93-128. 2004
    ....
  27. ncbi request reprint A new method for recombinant adeno-associated virus vector delivery to murine diaphragm
    Cathryn Mah
    Powell Gene Therapy Center, University of Florida, Gainesville, 32610, USA
    Mol Ther 9:458-63. 2004
    ..This report describes a novel method for efficient delivery of rAAV vectors to the mouse diaphragm and is the first demonstration of gene transfer to the diaphragm using recombinant adeno-associated virus vectors...
  28. ncbi request reprint Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease
    Mary Rucker
    Powell Gene Therapy Center, and Departments of Molecular Genetics and Microbiology and Pediatrics, University of Florida College of Medicine, Gainesville, Florida 32610, USA
    Development 131:3007-19. 2004
    ..This method could be employed with disease models where specific rescue of the diaphragm would allow for increased survival and thus further investigation into the impact of the gene deletion on other striated muscle groups...
  29. ncbi request reprint Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors
    Imran Mohiuddin
    Department of Molecular Genetics and Microbiology, University of Florida College of Medicine, Gainesville, FL 32610 0266, USA
    Mol Ther 11:320-6. 2005
    ..These methods generate reliable infectious titers for AAV vectors of different serotypes, thus enhancing product characterization and reducing risk in future clinical applications...
  30. ncbi request reprint Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II
    Kerry O Cresawn
    Department of Pediatrics, and Powell Gene Therapy Center, University of Florida, Gainesville, FL 32610, USA
    Hum Gene Ther 16:68-80. 2005
    ..These findings highlight the importance of the immune response to rAAV-encoded GAA in correcting GSDII and provide additional understanding of the approach to treatment of GSDII...
  31. ncbi request reprint Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector
    Mark C Walker
    Department of Small Animal Clinical Sciences, College of Veterinary Medicine, University of Florida, Gainesville, FL 32610, USA
    Am J Vet Res 66:450-6. 2005
    ....
  32. pmc Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system
    Erik Kohlbrenner
    Powell Gene Therapy Center, Division of Cellular and Molecular Therapy, Department of Pediatrics, University of Florida, Gainesville, FL 32610, USA
    Mol Ther 12:1217-25. 2005
    ..The redesigned baculovirus system improves our capacity for rAAV manufacturing by making this production platform more applicable to other existing serotypes...
  33. ncbi request reprint AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytes
    Lourdes M Andino
    Department of Molecular Genetics and Microbiology, University of Florida, Gainesville, FL 32610 0266, USA
    J Gene Med 10:132-42. 2008
    ..Several animal models of heart failure have been treated by altering the regulation of the sarcoplamic reticulum ATPase through ablation or down-regulation of its inhibitor peptide, phospholamban (PLN)...
  34. ncbi request reprint Non-invasive analysis of myoblast transplants in rodent cardiac muscle
    Kevin S Cahill
    Powell Gene Therapy Center, University of Florida, Gainesville 32610, USA
    Int J Cardiovasc Imaging 20:593-8. 2004
    ..We evaluated the ability of a commercially available, Food and Drug Administration (FDA) approved contrast agent to allow the monitoring of myoblast transplants in the rodent heart...
  35. pmc Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
    Artur V Cideciyan
    Scheie Eye Institute, Department of Ophthalmology, University of Pennsylvania, Philadelphia, PA 19104, USA
    Proc Natl Acad Sci U S A 105:15112-7. 2008
    ..Cone-sensitivity recovery time was rapid. These results demonstrate dramatic, albeit imperfect, recovery of rod- and cone-photoreceptor-based vision after RPE65 gene therapy...
  36. ncbi request reprint Virus-based gene delivery systems
    Cathryn Mah
    Powell Gene Therapy Center, University of Florida, Gainesville, Florida 32610 0266, USA
    Clin Pharmacokinet 41:901-11. 2002
    ..This review will also illustrate several current improved physical delivery systems for optimal vector administration...
  37. pmc Pompe disease diagnosis and management guideline
    Priya S Kishnani
    Duke University Medical Center, NC, USA
    Genet Med 8:267-88. 2006
  38. ncbi request reprint Cell therapy in the heart: cell production, transplantation, and applications
    Kevin S Cahill
    Department of Pediatrics, University of Florida School of Medicine, Gainesville, USA
    Methods Mol Biol 219:73-81. 2003
  39. ncbi request reprint Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors
    Sergei Zolotukhin
    Powell Gene Therapy Center, 1600 SW Archer Road, College of Medicine, University of Florida, 32610 0266, Gainesville, FL 32610 0266, USA
    Methods 28:158-67. 2002
    ..The purified vector stocks are 99% pure with titers of 1 x 10(12) to 1 x 10(13)vector genomes/ml...

Research Grants5

  1. GLYCOGEN STORAGE DISEASE TREATMENT: HEPATIC GENE THERAPY
    Barry Byrne; Fiscal Year: 2000
    ..These studies will yield important new information in establishing a clinically relevant treatment for these fatal diseases and add new understanding to the basic pathophysiology of GSD. ..
  2. GLYCOGEN STORAGE DISEASE TREATMENT: HEPATIC GENE THERAPY
    Barry Byrne; Fiscal Year: 2001
    ..These studies will yield important new information in establishing a clinically relevant treatment for these fatal diseases and add new understanding to the basic pathophysiology of GSD. ..
  3. GLYCOGEN STORAGE DISEASE TREATMENT: HEPATIC GENE THERAPY
    Barry Byrne; Fiscal Year: 2002
    ..These studies will yield important new information in establishing a clinically relevant treatment for these fatal diseases and add new understanding to the basic pathophysiology of GSD. ..
  4. NGVL TOXICOLOGY LABORATORY
    Barry Byrne; Fiscal Year: 2005
    ..These services will be offered in an open flexible, collaborative, and interactive manner, and in compliance with the Terms of Cooperation of the NGVL. ..