Research Topics
Genomes and GenesSpecies | Barry ByrneSummaryAffiliation: University of Florida Country: USA Publications
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Publications
Gene therapy approaches for lysosomal storage disease: next-generation treatmentBarry 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...- Pompe disease gene therapyBarry 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... 
Pompe disease: design, methodology, and early findings from the Pompe RegistryBarry 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...- Structure of adeno-associated virus serotype 8, a gene therapy vectorHyun 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... - Structural studies of adeno-associated virus serotype 8 capsid transitions associated with endosomal traffickingHyun 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... 
Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndromeR 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...- Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8Michael Douglas Lane
Department of Biochemistry and Molecular Biology, Center for Structural Biology, McKnight Brain Institute, University of Florida, Gainesville, FL 32610, USA
Acta Crystallograph 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... - Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vectorMatthias 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... - Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heartCatalin 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... - Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivoChristina 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.... - Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adultsMark L Brantly
Department of Medicine, University of Florida, Gainesville, FL 32611, USA
Hum Gene Ther 17:1177-86. 2006.... - Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectorsCathryn Mah
Powell Gene Therapy Center, University of Florida, Gainesville, Florida, USA
Mol Ther 15:501-7. 2007.... - 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 sitesTerence 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... - Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2DChristina 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... - Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularizationMaria 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... - Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5Steven Polyak
Division of Gastroenterology, Department of Medicine, University of Florida, Gainesville, FL 32610, USA
Dig Dis Sci 53:1261-70. 2008.... - Update of the Pompe disease mutation database with 107 sequence variants and a format for severity ratingMarian 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... - Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzeesTerence 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... - Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectorsThomas J Fraites
Powell Gene Therapy Center, University of Florida College of Medicine, Gainesville, Florida 32610, USA
Mol Ther 5:571-8. 2002.... - 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... - Cardiac and clinical phenotype in Barth syndromeCarolyn 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... - Improved method of recombinant AAV2 delivery for systemic targeted gene therapyCathryn 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... - Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A miceCathryn 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... - Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adultsTerence R Flotte
Department of Pediatrics, University of Florida, Gainesville, USA
Hum Gene Ther 15:93-128. 2004.... - A new method for recombinant adeno-associated virus vector delivery to murine diaphragmCathryn 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... - Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe diseaseMary 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... - Herpesvirus-based infectious titering of recombinant adeno-associated viral vectorsImran 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... - 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 IIKerry 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... - Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vectorMark 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..However, rAAV2-fEPO vector administration may result in pure RBC aplasia or pathologic erythrocytosis, and injection site removal does not consistently abolish the biological response... - Successful production of pseudotyped rAAV vectors using a modified baculovirus expression systemErik 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... - Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in miceMao 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... - Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectorsSergei 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... - Cell therapy in the heart: cell production, transplantation, and applicationsKevin S Cahill
Department of Pediatrics, University of Florida School of Medicine, Gainesville, USA
Methods Mol Biol 219:73-81. 2003 - Pompe disease diagnosis and management guidelinePriya S Kishnani
Duke University Medical Center, NC, USA
Genet Med 8:267-88. 2006 - Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kineticsArtur 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... - AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytesLourdes 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)... - Non-invasive analysis of myoblast transplants in rodent cardiac muscleKevin S Cahill
Powell Gene Therapy Center, University of Florida, Gainesville 32610, USA
Int J Cardiovasc Imaging 20:593-8. 2004..Additional techniques are in development to non-invasively determine stem cell engraftment rates, viability and differentiation... - Virus-based gene delivery systemsCathryn 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...
Research Grants
- GLYCOGEN STORAGE DISEASE TREATMENT: HEPATIC GENE THERAPYBarry 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. ..
- NGVL TOXICOLOGY LABORATORYBarry 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. ..