Barry J. Byrne - Publications

Affiliations: 
University of Florida, Gainesville, Gainesville, FL, United States 
Area:
Medicine and Surgery, Cell Biology

99 high-probability publications. We are testing a new system for linking publications to authors. You can help! If you notice any inaccuracies, please sign in and mark papers as correct or incorrect matches. If you identify any major omissions or other inaccuracies in the publication list, please let us know.

Year Citation  Score
2019 Cade WT, Laforest R, Bohnert KL, Reeds DN, Bittel AJ, de Las Fuentes L, Bashir A, Woodard PK, Pacak CA, Byrne BJ, Gropler RJ, Peterson LR. Myocardial glucose and fatty acid metabolism is altered and associated with lower cardiac function in young adults with Barth syndrome. Journal of Nuclear Cardiology : Official Publication of the American Society of Nuclear Cardiology. PMID 31705425 DOI: 10.1007/s12350-019-01933-3  0.92
2019 Doyle BM, Turner SMF, Sunshine MD, Doerfler PA, Poirier AE, Vaught LA, Jorgensen ML, Falk DJ, Byrne BJ, Fuller DD. AAV Gene Therapy Utilizing Glycosylation-Independent Lysosomal Targeting Tagged GAA in the Hypoglossal Motor System of Pompe Mice. Molecular Therapy. Methods & Clinical Development. 15: 194-203. PMID 31660421 DOI: 10.1016/j.omtm.2019.08.009  0.44
2019 Byrne BJ, Fuller DD, Smith BK, Clement N, Coleman K, Cleaver B, Vaught L, Falk DJ, McCall A, Corti M. Pompe disease gene therapy: neural manifestations require consideration of CNS directed therapy. Annals of Translational Medicine. 7: 290. PMID 31392202 DOI: 10.21037/atm.2019.05.56  0.44
2019 Suzuki-Hatano S, Sriramvenugopal M, Ramanathan M, Soustek M, Byrne BJ, Cade WT, Kang PB, Pacak CA. Increased mtDNA Abundance and Improved Function in Human Barth Syndrome Patient Fibroblasts Following AAV- Gene Delivery. International Journal of Molecular Sciences. 20. PMID 31336787 DOI: 10.3390/ijms20143416  0.92
2019 Cade WT, Bohnert KL, Peterson LR, Patterson BW, Bittel AJ, Okunade AL, de Las Fuentes L, Steger-May K, Bashir A, Schweitzer GG, Chacko SK, Wanders RJ, Pacak CA, Byrne BJ, Reeds DN. Blunted Fat Oxidation upon Submaximal Exercise is Partially Compensated by Enhanced Glucose Metabolism in Children, Adolescents and Young Adults with Barth Syndrome. Journal of Inherited Metabolic Disease. PMID 30924938 DOI: 10.1002/jimd.12094  0.92
2019 Suzuki-Hatano S, Saha M, Soustek MS, Kang PB, Byrne BJ, Cade WT, Pacak CA. AAV9- Gene Replacement Ameliorates Cardiac TMT Proteomic Profiles in a Mouse Model of Barth Syndrome. Molecular Therapy. Methods & Clinical Development. 13: 167-179. PMID 30788385 DOI: 10.1016/j.omtm.2019.01.007  0.92
2018 Suzuki-Hatano S, Saha M, Rizzo SA, Witko RL, Gosiker BJ, Ramanathan M, Soustek MS, Jones MD, Kang PB, Byrne BJ, Cade WT, Pacak CA. AAV-Mediated TAZ Gene Replacement Restores Mitochondrial and Cardioskeletal Function in Barth Syndrome. Human Gene Therapy. PMID 30070157 DOI: 10.1089/hum.2018.020  0.92
2018 Cade WT, Bohnert KL, Reeds DN, Peterson LR, Bittel AJ, Bashir A, Byrne BJ, Taylor CL. Peak oxygen uptake (VO2peak) across childhood, adolescence and young adulthood in Barth syndrome: Data from cross-sectional and longitudinal studies. Plos One. 13: e0197776. PMID 29795646 DOI: 10.1371/journal.pone.0197776  0.44
2018 Bittel AJ, Bohnert KL, Reeds DN, Peterson LR, de Las Fuentes L, Corti M, Taylor CL, Byrne BJ, Todd Cade W. Reduced Muscle Strength in Barth Syndrome May Be Improved by Resistance Exercise Training: A Pilot Study. Jimd Reports. PMID 29654548 DOI: 10.1007/8904_2018_102  0.36
2017 Corti M, Liberati C, Smith BK, Lawson LA, Tuna IS, Conlon TJ, Erger KE, Islam S, Herzog R, Fuller DD, Collins S, Byrne BJ. Safety of Intradiaphragmatic Delivery of Adeno-Associated Virus-Mediated Alpha-Glucosidase (rAAV1-CMV-hGAA) Gene Therapy in children affected by Pompe Disease. Human Gene Therapy. Clinical Development. PMID 29160099 DOI: 10.1089/humc.2017.146  0.52
2017 Keeler AM, Liu D, Zieger M, Xiong L, Salemi J, Bellve K, Byrne BJ, Fuller DD, ZhuGe R, ElMallah MK. Airway smooth muscle dysfunction in Pompe (Gaa-/-) mice. American Journal of Physiology. Lung Cellular and Molecular Physiology. ajplung.00568.2016. PMID 28336814 DOI: 10.1152/ajplung.00568.2016  0.44
2017 Bashir A, Bohnert KL, Reeds DN, Peterson LR, Bittel AJ, de Las Fuentes L, Pacak CA, Byrne BJ, Cade WT. Impaired cardiac and skeletal muscle bioenergetics in children, adolescents, and young adults with Barth syndrome. Physiological Reports. 5. PMID 28196853 DOI: 10.14814/phy2.13130  0.92
2016 Conlon TJ, Mah CS, Pacak CA, Rucker Henninger MB, Erger KE, Jorgensen ML, Lee CC, Tarantal AF, Byrne BJ. Transfer of Therapeutic Genes into Fetal Rhesus Monkeys using Recombinant Adeno-Associated Type I Viral Vectors. Human Gene Therapy. Clinical Development. PMID 27855487 DOI: 10.1089/humc.2016.119  0.92
2016 Turner SM, Falk DJ, Byrne BJ, Fuller DD. Transcriptome assessment of the Pompe (Gaa-/-) mouse spinal cord indicates widespread neuropathology. Physiological Genomics. physiolgenomics.0007. PMID 27614205 DOI: 10.1152/physiolgenomics.00075.2016  0.44
2016 Cade WT, Reeds DN, Peterson LR, Bohnert KL, Tinius RA, Benni PB, Byrne BJ, Taylor CL. Endurance Exercise Training in Young Adults with Barth Syndrome: A Pilot Study. Jimd Reports. PMID 27295193 DOI: 10.1007/8904_2016_553  0.44
2016 Turner SM, Hoyt AK, ElMallah MK, Falk DJ, Byrne BJ, Fuller DD. Neuropathology in respiratory-related motoneurons in young Pompe (Gaa(-/-)) mice. Respiratory Physiology & Neurobiology. PMID 26921786 DOI: 10.1016/j.resp.2016.02.007  0.44
2016 Smith BK, Fuller DD, Martin AD, Lottenberg L, Islam S, Lawson LA, Onders RP, Byrne BJ. Diaphragm Pacing as a Rehabilitative Tool for Ventilator-Dependent Pompe Disease: Case Series. Physical Therapy. PMID 26893511 DOI: 10.2522/ptj.20150122  0.44
2015 Smith BK, Corti M, Martin AD, Fuller DD, Byrne BJ. Altered Activation of the Diaphragm in Late-Onset Pompe Disease. Respiratory Physiology & Neurobiology. PMID 26612101 DOI: 10.1016/j.resp.2015.11.013  0.44
2015 Corti M, Cleaver B, Clément N, Conlon TJ, Faris KJ, Wang G, Benson J, Tarantal AF, Fuller D, Herzog RW, Byrne BJ. Evaluation of Readministration of a Recombinant Adeno-Associated Virus Vector Expressing Acid Alpha-Glucosidase in Pompe Disease: Preclinical to Clinical Planning. Human Gene Therapy. Clinical Development. 26: 185-93. PMID 26390092 DOI: 10.1089/humc.2015.068  0.52
2015 Lee NC, Muramatsu SI, Chien YH, Liu WS, Wang WH, Cheng CH, Hu MK, Chen PW, Tzen KY, Byrne BJ, Hwu WL. Benefits of neuronal preferential systemic gene therapy for neurotransmitter deficiency. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 26137853 DOI: 10.1038/mt.2015.122  0.52
2015 Falk DJ, Soustek MS, Todd AG, Mah CS, Cloutier DA, Kelley JS, Clement N, Fuller DD, Byrne BJ. Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice. Molecular Therapy. Methods & Clinical Development. 2: 15007. PMID 26029718 DOI: 10.1038/mtm.2015.7  0.52
2015 Todd AG, McElroy JA, Grange RW, Fuller DD, Walter GA, Byrne BJ, Falk DJ. Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease. Annals of Neurology. 78: 222-34. PMID 25925726 DOI: 10.1002/ana.24433  0.52
2015 Soustek MS, Baligand C, Falk DJ, Walter GA, Lewin AS, Byrne BJ. Endurance training ameliorates complex 3 deficiency in a mouse model of Barth syndrome. Journal of Inherited Metabolic Disease. PMID 25860817 DOI: 10.1007/s10545-015-9834-8  0.52
2015 ElMallah MK, Pagliardini S, Turner SM, Cerreta AJ, Falk DJ, Byrne BJ, Greer JJ, Fuller DD. Ampakines Stimulate Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease. American Journal of Respiratory Cell and Molecular Biology. PMID 25569118 DOI: 10.1165/rcmb.2014-0374OC  0.44
2015 Falk DJ, Todd AG, Lee S, Soustek MS, ElMallah MK, Fuller DD, Notterpek L, Byrne BJ. Peripheral nerve and neuromuscular junction pathology in Pompe disease. Human Molecular Genetics. 24: 625-36. PMID 25217571 DOI: 10.1093/hmg/ddu476  0.52
2015 Corti M, Smith BK, Falk DJ, Lawson LA, Fuller DD, Subramony SH, Byrne BJ, Christou EA. Altered activation of the tibialis anterior in individuals with Pompe disease: Implications for motor unit dysfunction. Muscle & Nerve. 51: 877-83. PMID 25186912 DOI: 10.1002/mus.24444  0.52
2014 Burns KM, Byrne BJ, Gelb BD, Kühn B, Leinwand LA, Mital S, Pearson GD, Rodefeld M, Rossano JW, Stauffer BL, Taylor MD, Towbin JA, Redington AN. New mechanistic and therapeutic targets for pediatric heart failure: report from a National Heart, Lung, and Blood Institute working group. Circulation. 130: 79-86. PMID 24982119 DOI: 10.1161/CIRCULATIONAHA.113.007980  0.52
2014 Elmallah MK, Falk DJ, Nayak S, Federico RA, Sandhu MS, Poirier A, Byrne BJ, Fuller DD. Sustained correction of motoneuron histopathology following intramuscular delivery of AAV in pompe mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 22: 702-12. PMID 24336173 DOI: 10.1038/mt.2013.282  0.52
2014 Lee NC, Chien YH, Hu MH, Liu WS, Chen PW, Wang WH, Tzen KY, Byrne BJ, Hwu WL. Treatment of congenital neurotransmitter deficiencies by intracerebral ventricular injection of an adeno-associated virus serotype 9 vector. Human Gene Therapy. 25: 189-98. PMID 24251946 DOI: 10.1089/hum.2013.170  0.52
2013 Mah CS, Soustek MS, Todd AG, McCall A, Smith BK, Corti M, Falk DJ, Byrne BJ. Adeno-associated virus-mediated gene therapy for metabolic myopathy. Human Gene Therapy. 24: 928-36. PMID 24164240 DOI: 10.1089/hum.2013.2514  0.52
2013 Conlon TJ, Erger K, Porvasnik S, Cossette T, Roberts C, Combee L, Islam S, Kelley J, Cloutier D, Clément N, Abernathy CR, Byrne BJ. Preclinical toxicology and biodistribution studies of recombinant adeno-associated virus 1 human acid α-glucosidase. Human Gene Therapy. Clinical Development. 24: 127-33. PMID 24021025 DOI: 10.1089/humc.2013.147  0.52
2013 Fuller DD, ElMallah MK, Smith BK, Corti M, Lawson LA, Falk DJ, Byrne BJ. The respiratory neuromuscular system in Pompe disease. Respiratory Physiology & Neurobiology. 189: 241-9. PMID 23797185 DOI: 10.1016/j.resp.2013.06.007  0.52
2013 Falk DJ, Mah CS, Soustek MS, Lee KZ, Elmallah MK, Cloutier DA, Fuller DD, Byrne BJ. Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1661-7. PMID 23732990 DOI: 10.1038/mt.2013.96  0.52
2013 Forbes SC, Walter GA, Rooney WD, Wang DJ, DeVos S, Pollaro J, Triplett W, Lott DJ, Willcocks RJ, Senesac C, Daniels MJ, Byrne BJ, Russman B, Finkel RS, Meyer JS, et al. Skeletal muscles of ambulant children with Duchenne muscular dystrophy: validation of multicenter study of evaluation with MR imaging and MR spectroscopy. Radiology. 269: 198-207. PMID 23696684 DOI: 10.1148/radiol.13121948  0.52
2013 Smith BK, Collins SW, Conlon TJ, Mah CS, Lawson LA, Martin AD, Fuller DD, Cleaver BD, Clément N, Phillips D, Islam S, Dobjia N, Byrne BJ. 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. Human Gene Therapy. 24: 630-40. PMID 23570273 DOI: 10.1089/hum.2012.250  0.52
2013 O'Reilly M, Kohn DB, Bartlett J, Benson J, Brooks PJ, Byrne BJ, Camozzi C, Cornetta K, Crystal RG, Fong Y, Gargiulo L, Gopal-Srivastava R, High KA, Jacobson SG, Jambou RC, et al. Gene therapy for rare diseases: summary of a National Institutes of Health workshop, September 13, 2012. Human Gene Therapy. 24: 355-62. PMID 23517518 DOI: 10.1089/hum.2013.064  0.52
2013 Maga JA, Zhou J, Kambampati R, Peng S, Wang X, Bohnsack RN, Thomm A, Golata S, Tom P, Dahms NM, Byrne BJ, LeBowitz JH. Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice. The Journal of Biological Chemistry. 288: 1428-38. PMID 23188827 DOI: 10.1074/jbc.M112.438663  0.52
2013 Cade WT, Spencer CT, Reeds DN, Waggoner AD, O'Connor R, Maisenbacher M, Crowley JR, Byrne BJ, Peterson LR. Substrate metabolism during basal and hyperinsulinemic conditions in adolescents and young-adults with Barth syndrome. Journal of Inherited Metabolic Disease. 36: 91-101. PMID 22580961 DOI: 10.1007/s10545-012-9486-x  0.52
2012 Martin EA, Barresi R, Byrne BJ, Tsimerinov EI, Scott BL, Walker AE, Gurudevan SV, Anene F, Elashoff RM, Thomas GD, Victor RG. Tadalafil alleviates muscle ischemia in patients with Becker muscular dystrophy. Science Translational Medicine. 4: 162ra155. PMID 23197572 DOI: 10.1126/scitranslmed.3004327  0.52
2012 Roberts AE, Nixon C, Steward CG, Gauvreau K, Maisenbacher M, Fletcher M, Geva J, Byrne BJ, Spencer CT. The Barth Syndrome Registry: distinguishing disease characteristics and growth data from a longitudinal study. American Journal of Medical Genetics. Part A. 158: 2726-32. PMID 23045169 DOI: 10.1002/ajmg.a.35609  0.52
2012 Forbes SC, Lott DJ, Finkel RS, Senesac C, Byrne BJ, Sweeney HL, Walter GA, Vandenborne K. MRI/MRS evaluation of a female carrier of Duchenne muscular dystrophy. Neuromuscular Disorders : Nmd. 22: S111-21. PMID 22980762 DOI: 10.1016/j.nmd.2012.05.013  0.52
2012 Della Rocca DG, Willenberg BJ, Ferreira LF, Wate PS, Petersen JW, Handberg EM, Zheng T, Steindler DA, Terada N, Batich CD, Byrne BJ, Pepine CJ. A degradable, bioactive, gelatinized alginate hydrogel to improve stem cell/growth factor delivery and facilitate healing after myocardial infarction. Medical Hypotheses. 79: 673-7. PMID 22939314 DOI: 10.1016/j.mehy.2012.08.006  0.52
2012 Byrne BJ, Falk DJ, Clément N, Mah CS. Gene therapy approaches for lysosomal storage disease: next-generation treatment. Human Gene Therapy. 23: 808-15. PMID 22794786 DOI: 10.1089/hum.2012.140  0.52
2012 Lee NC, Falk DJ, Byrne BJ, Conlon TJ, Clement N, Porvasnik S, Jorgensen ML, Potter M, Erger KE, Watson R, Ghivizzani SC, Chiu HC, Chien YH, Hwu WL. An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery. Genetic Vaccines and Therapy. 10: 3. PMID 22709483 DOI: 10.1186/1479-0556-10-3  0.52
2012 ElMallah MK, Falk DJ, Lane MA, Conlon TJ, Lee KZ, Shafi NI, Reier PJ, Byrne BJ, Fuller DD. Retrograde gene delivery to hypoglossal motoneurons using adeno-associated virus serotype 9. Human Gene Therapy Methods. 23: 148-56. PMID 22693957 DOI: 10.1089/hgtb.2012.009  0.52
2012 DiMattia MA, Nam HJ, Van Vliet K, Mitchell M, Bennett A, Gurda BL, McKenna R, Olson NH, Sinkovits RS, Potter M, Byrne BJ, Aslanidi G, Zolotukhin S, Muzyczka N, Baker TS, et al. Structural insight into the unique properties of adeno-associated virus serotype 9. Journal of Virology. 86: 6947-58. PMID 22496238 DOI: 10.1128/JVI.07232-11  0.52
2012 Qiu K, Falk DJ, Reier PJ, Byrne BJ, Fuller DD. Spinal delivery of AAV vector restores enzyme activity and increases ventilation in Pompe mice. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 21-7. PMID 22008916 DOI: 10.1038/mt.2011.214  0.52
2012 Jacobson SG, Cideciyan AV, Ratnakaram R, Heon E, Schwartz SB, Roman AJ, Peden MC, Aleman TS, Boye SL, Sumaroka A, Conlon TJ, Calcedo R, Pang JJ, Erger KE, Olivares MB, ... ... Byrne BJ, et al. Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years. Archives of Ophthalmology (Chicago, Ill. : 1960). 130: 9-24. PMID 21911650 DOI: 10.1001/archophthalmol.2011.298  0.52
2011 Traverse JH, Henry TD, Ellis SG, Pepine CJ, Willerson JT, Zhao DX, Forder JR, Byrne BJ, Hatzopoulos AK, Penn MS, Perin EC, Baran KW, Chambers J, Lambert C, Raveendran G, et al. Effect of intracoronary delivery of autologous bone marrow mononuclear cells 2 to 3 weeks following acute myocardial infarction on left ventricular function: the LateTIME randomized trial. Jama. 306: 2110-9. PMID 22084195 DOI: 10.1001/jama.2011.1670  0.52
2011 Spencer CT, Byrne BJ, Bryant RM, Margossian R, Maisenbacher M, Breitenger P, Benni PB, Redfearn S, Marcus E, Cade WT. Impaired cardiac reserve and severely diminished skeletal muscle o 2 utilization mediate exercise intolerance in barth syndrome American Journal of Physiology - Heart and Circulatory Physiology. 301. PMID 21873497 DOI: 10.1152/ajpheart.00479.2010  0.52
2011 Pacak CA, Byrne BJ. AAV vectors for cardiac gene transfer: experimental tools and clinical opportunities. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 1582-90. PMID 21792180 DOI: 10.1038/mt.2011.124  0.92
2011 Lee KZ, Qiu K, Sandhu MS, Elmallah MK, Falk DJ, Lane MA, Reier PJ, Byrne BJ, Fuller DD. Hypoglossal neuropathology and respiratory activity in pompe mice. Frontiers in Physiology. 2: 31. PMID 21747768 DOI: 10.3389/fphys.2011.00031  0.52
2011 Byrne BJ, Falk DJ, Pacak CA, Nayak S, Herzog RW, Elder ME, Collins SW, Conlon TJ, Clement N, Cleaver BD, Cloutier DA, Porvasnik SL, Islam S, Elmallah MK, Martin A, et al. Pompe disease gene therapy. Human Molecular Genetics. 20: R61-8. PMID 21518733 DOI: 10.1093/hmg/ddr174  0.92
2011 Specht A, Fiske L, Erger K, Cossette T, Verstegen J, Campbell-Thompson M, Struck MB, Lee YM, Chou JY, Byrne BJ, Correia CE, Mah CS, Weinstein DA, Conlon TJ. Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease. Journal of Biomedicine & Biotechnology. 2011: 646257. PMID 21318173 DOI: 10.1155/2011/646257  0.52
2011 Mandel RJ, Lowenstein PR, Byrne BJ. AAV6-mediated gene silencing fALS short. Molecular Therapy : the Journal of the American Society of Gene Therapy. 19: 231-3. PMID 21289637 DOI: 10.1038/mt.2010.306  0.52
2011 Soustek MS, Falk DJ, Mah CS, Toth MJ, Schlame M, Lewin AS, Byrne BJ. Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency. Human Gene Therapy. 22: 865-71. PMID 21091282 DOI: 10.1089/hum.2010.199  0.52
2010 Choi YK, Nash K, Byrne BJ, Muzyczka N, Song S. The effect of DNA-dependent protein kinase on adeno-associated virus replication. Plos One. 5: e15073. PMID 21188139 DOI: 10.1371/journal.pone.0015073  0.52
2010 Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, et al. Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D. Annals of Neurology. 68: 629-38. PMID 21031578 DOI: 10.1002/ana.22251  0.52
2010 Traverse JH, Henry TD, Vaughan DE, Ellis SG, Pepine CJ, Willerson JT, Zhao DX, Simpson LM, Penn MS, Byrne BJ, Perin EC, Gee AP, Hatzopoulos AK, McKenna DH, Forder JR, et al. LateTIME: a phase-II, randomized, double-blinded, placebo-controlled, pilot trial evaluating the safety and effect of administration of bone marrow mononuclear cells 2 to 3 weeks after acute myocardial infarction. Texas Heart Institute Journal / From the Texas Heart Institute of St. Luke's Episcopal Hospital, Texas Children's Hospital. 37: 412-20. PMID 20844613  0.52
2010 Force T, Bonow RO, Houser SR, Solaro RJ, Hershberger RE, Adhikari B, Anderson ME, Boineau R, Byrne BJ, Cappola TP, Kalluri R, LeWinter MM, Maron MS, Molkentin JD, Ommen SR, et al. Research priorities in hypertrophic cardiomyopathy: report of a Working Group of the National Heart, Lung, and Blood Institute. Circulation. 122: 1130-3. PMID 20837938 DOI: 10.1161/CIRCULATIONAHA.110.950089  0.52
2010 Willerson JT, Perin EC, Ellis SG, Pepine CJ, Henry TD, Zhao DX, Lai D, Penn MS, Byrne BJ, Silva G, Gee A, Traverse JH, Hatzopoulos AK, Forder JR, Martin D, et al. Intramyocardial injection of autologous bone marrow mononuclear cells for patients with chronic ischemic heart disease and left ventricular dysfunction (First Mononuclear Cells injected in the US [FOCUS]): Rationale and design. American Heart Journal. 160: 215-23. PMID 20691824 DOI: 10.1016/j.ahj.2010.03.029  0.52
2010 Lock M, McGorray S, Auricchio A, Ayuso E, Beecham EJ, Blouin-Tavel V, Bosch F, Bose M, Byrne BJ, Caton T, Chiorini JA, Chtarto A, Clark KR, Conlon T, Darmon C, et al. Characterization of a recombinant adeno-associated virus type 2 Reference Standard Material. Human Gene Therapy. 21: 1273-85. PMID 20486768 DOI: 10.1089/hum.2009.223  0.52
2010 Porvasnik SL, Germain S, Embury J, Gannon KS, Jacques V, Murray J, Byrne BJ, Shacham S, Al-Mousily F. PRX-08066, a novel 5-hydroxytryptamine receptor 2B antagonist, reduces monocrotaline-induced pulmonary arterial hypertension and right ventricular hypertrophy in rats. The Journal of Pharmacology and Experimental Therapeutics. 334: 364-72. PMID 20430844 DOI: 10.1124/jpet.109.165001  0.52
2010 Weinstein DA, Correia CE, Conlon T, Specht A, Verstegen J, Onclin-Verstegen K, Campbell-Thompson M, Dhaliwal G, Mirian L, Cossette H, Falk DJ, Germain S, Clement N, Porvasnik S, Fiske L, ... ... Byrne BJ, et al. Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia. Human Gene Therapy. 21: 903-10. PMID 20163245 DOI: 10.1089/hum.2009.157  0.52
2010 Mah CS, Falk DJ, Germain SA, Kelley JS, Lewis MA, Cloutier DA, DeRuisseau LR, Conlon TJ, Cresawn KO, Fraites TJ, Campbell-Thompson M, Fuller DD, Byrne BJ. Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease. Molecular Therapy : the Journal of the American Society of Gene Therapy. 18: 502-10. PMID 20104213 DOI: 10.1038/mt.2009.305  0.52
2009 Storch EA, Keeley M, Merlo LJ, St Amant JB, Jacob M, Storch JF, Spencer C, Byrne BJ. Psychosocial Functioning in Youth with Barth Syndrome. Children's Health Care : Journal of the Association For the Care of Children's Health. 38: 137-156. PMID 20808735 DOI: 10.1080/02739610902813344  0.52
2009 Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, et al. Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Annals of Neurology. 66: 290-7. PMID 19798725 DOI: 10.1002/ana.21732  0.52
2009 Brantly ML, Chulay JD, Wang L, Mueller C, Humphries M, Spencer LT, Rouhani F, Conlon TJ, Calcedo R, Betts MR, Spencer C, Byrne BJ, Wilson JM, Flotte TR. Sustained transgene expression despite T lymphocyte responses in a clinical trial of rAAV1-AAT gene therapy. Proceedings of the National Academy of Sciences of the United States of America. 106: 16363-8. PMID 19706466 DOI: 10.1073/pnas.0904514106  0.52
2009 Traverse JH, Henry TD, Vaughan DE, Vaughn DE, Ellis SG, Pepine CJ, Willerson JT, Zhao DX, Piller LB, Penn MS, Byrne BJ, Perin EC, Gee AP, Hatzopoulos AK, McKenna DH, et al. Rationale and design for TIME: A phase II, randomized, double-blind, placebo-controlled pilot trial evaluating the safety and effect of timing of administration of bone marrow mononuclear cells after acute myocardial infarction. American Heart Journal. 158: 356-63. PMID 19699857 DOI: 10.1016/j.ahj.2009.06.009  0.52
2009 Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Roman AJ, Byrne BJ, Jacobson SG. Vision 1 year after gene therapy for Leber's congenital amaurosis. The New England Journal of Medicine. 361: 725-7. PMID 19675341 DOI: 10.1056/NEJMc0903652  0.52
2009 Cideciyan AV, Hauswirth WW, Aleman TS, Kaushal S, Schwartz SB, Boye SL, Windsor EA, Conlon TJ, Sumaroka A, Pang JJ, Roman AJ, Byrne BJ, Jacobson SG. Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year. Human Gene Therapy. 20: 999-1004. PMID 19583479 DOI: 10.1089/hum.2009.086  0.52
2009 Jacobs JP, Quintessenza JA, Burke RP, Bleiweis MS, Byrne BJ, Ceithaml EL, Decampli WM, Giroud JM, Perryman RA, Rosenkranz ER, Wolff G, Posner V, Steverson S, Blanchard WB, Schiebler GL. Analysis of regional congenital cardiac surgical outcomes in Florida using the Society of Thoracic Surgeons Congenital Heart Surgery Database. Cardiology in the Young. 19: 360-9. PMID 19575843 DOI: 10.1017/S1047951109990151  0.52
2009 Mitchell M, Nam HJ, Carter A, McCall A, Rence C, Bennett A, Gurda B, McKenna R, Porter M, Sakai Y, Byrne BJ, Muzyczka N, Aslanidi G, Zolotukhin S, Agbandje-McKenna M. Production, purification and preliminary X-ray crystallographic studies of adeno-associated virus serotype 9. Acta Crystallographica. Section F, Structural Biology and Crystallization Communications. 65: 715-8. PMID 19574648 DOI: 10.1107/S1744309109021460  0.52
2009 Clément N, Knop DR, Byrne BJ. Large-scale adeno-associated viral vector production using a herpesvirus-based system enables manufacturing for clinical studies. Human Gene Therapy. 20: 796-806. PMID 19569968 DOI: 10.1089/hum.2009.094  0.52
2009 DeRuisseau LR, Fuller DD, Qiu K, DeRuisseau KC, Donnelly WH, Mah C, Reier PJ, Byrne BJ. Neural deficits contribute to respiratory insufficiency in Pompe disease. Proceedings of the National Academy of Sciences of the United States of America. 106: 9419-24. PMID 19474295 DOI: 10.1073/pnas.0902534106  0.52
2008 Pacak CA, Conlon T, Mah CS, Byrne BJ. Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle. Genetic Vaccines and Therapy. 6: 14. PMID 18854054 DOI: 10.1186/1479-0556-6-14  0.52
2008 Pacak CA, Sakai Y, Thattaliyath BD, Mah CS, Byrne BJ. Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice. Genetic Vaccines and Therapy. 6: 13. PMID 18811960 DOI: 10.1186/1479-0556-6-13  0.52
2008 Cideciyan AV, Aleman TS, Boye SL, Schwartz SB, Kaushal S, Roman AJ, Pang JJ, Sumaroka A, Windsor EA, Wilson JM, Flotte TR, Fishman GA, Heon E, Stone EM, Byrne BJ, et al. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics. Proceedings of the National Academy of Sciences of the United States of America. 105: 15112-7. PMID 18809924 DOI: 10.1073/pnas.0807027105  0.52
2008 Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. 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 trial. Human Gene Therapy. 19: 979-90. PMID 18774912 DOI: 10.1089/hum.2008.107  0.52
2008 Flotte TR, Goetzmann J, Caridi J, Paolillo J, Conlon TJ, Potter M, Mueller C, Byrne BJ. Apparently nonspecific enzyme elevations after portal vein delivery of recombinant adeno-associated virus serotype 2 vector in hepatitis C virus-infected chimpanzees. Human Gene Therapy. 19: 681-9. PMID 18588426 DOI: 10.1089/hum.2007.174  0.52
2008 Andino LM, Takeda M, Kasahara H, Jakymiw A, Byrne BJ, Lewin AS. AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytes. The Journal of Gene Medicine. 10: 132-42. PMID 18064719 DOI: 10.1002/jgm.1131  0.52
2008 Polyak S, Mah C, Porvasnik S, Herlihy JD, Campbell-Thompson M, Byrne BJ, Valentine JF. Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5. Digestive Diseases and Sciences. 53: 1261-70. PMID 17934813 DOI: 10.1007/s10620-007-9991-1  0.52
2007 Pacak CA, Walter GA, Gaidosh G, Bryant N, Lewis MA, Germain S, Mah CS, Campbell KP, Byrne BJ. Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 1775-81. PMID 17653106 DOI: 10.1038/sj.mt.6300246  0.52
2007 Flotte TR, Conlon TJ, Poirier A, Campbell-Thompson M, Byrne BJ. 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. Human Gene Therapy. 18: 245-56. PMID 17376008 DOI: 10.1089/hum.2006.113  0.52
2007 Mah C, Pacak CA, Cresawn KO, Deruisseau LR, Germain S, Lewis MA, Cloutier DA, Fuller DD, Byrne BJ. Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 15: 501-7. PMID 17245350 DOI: 10.1038/sj.mt.6300100  0.52
2006 Brantly ML, Spencer LT, Humphries M, Conlon TJ, Spencer CT, Poirier A, Garlington W, Baker D, Song S, Berns KI, Muzyczka N, Snyder RO, Byrne BJ, Flotte TR. Phase I trial of intramuscular injection of a recombinant adeno-associated virus serotype 2 alphal-antitrypsin (AAT) vector in AAT-deficient adults. Human Gene Therapy. 17: 1177-86. PMID 17115945 DOI: 10.1089/hum.2006.17.1177  0.52
2006 Pacak CA, Mah CS, Thattaliyath BD, Conlon TJ, Lewis MA, Cloutier DE, Zolotukhin I, Tarantal AF, Byrne BJ. Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo. Circulation Research. 99: e3-9. PMID 16873720 DOI: 10.1161/01.RES.0000237661.18885.f6  0.52
2006 Spencer CT, Bryant RM, Day J, Gonzalez IL, Colan SD, Thompson WR, Berthy J, Redfearn SP, Byrne BJ. Cardiac and clinical phenotype in Barth syndrome. Pediatrics. 118: e337-46. PMID 16847078 DOI: 10.1542/peds.2005-2667  0.52
2006 Kishnani PS, Steiner RD, Bali D, Berger K, Byrne BJ, Case LE, Case L, Crowley JF, Downs S, Howell RR, Kravitz RM, Mackey J, Marsden D, Martins AM, et al. Pompe disease diagnosis and management guideline. Genetics in Medicine : Official Journal of the American College of Medical Genetics. 8: 267-88. PMID 16702877 DOI: 10.1097/01.gim.0000218152.87434.f3  0.52
2005 Kohlbrenner E, Aslanidi G, Nash K, Shklyaev S, Campbell-Thompson M, Byrne BJ, Snyder RO, Muzyczka N, Warrington KH, Zolotukhin S. Successful production of pseudotyped rAAV vectors using a modified baculovirus expression system. Molecular Therapy : the Journal of the American Society of Gene Therapy. 12: 1217-25. PMID 16213797 DOI: 10.1016/j.ymthe.2005.08.018  0.52
2005 Mah C, Cresawn KO, Fraites TJ, Pacak CA, Lewis MA, Zolotukhin I, Byrne BJ. Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors Gene Therapy. 12: 1405-1409. PMID 15920463 DOI: 10.1038/sj.gt.3302550  0.52
2005 Cresawn KO, Fraites TJ, Wasserfall C, Atkinson M, Lewis M, Porvasnik S, Liu C, Mah C, Byrne BJ. Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid α-glucosidase in a model of glycogen storage disease type II Human Gene Therapy. 16: 68-80. PMID 15703490 DOI: 10.1089/hum.2005.16.68  0.52
2004 Cahill KS, Germain S, Byrne BJ, Walter GA. Non-invasive analysis of myoblast transplants in rodent cardiac muscle International Journal of Cardiovascular Imaging. 20: 593-598. PMID 15856647 DOI: 10.1007/s10554-004-3902-8  0.52
2004 Rucker M, Fraites TJ, Porvasnik SL, Lewis MA, Zolotukhin I, Cloutier DA, Byrne BJ. Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease. Development (Cambridge, England). 131: 3007-19. PMID 15169761 DOI: 10.1242/dev.01169  0.52
2004 Mah C, Fraites TJ, Cresawn KO, Zolotukhin I, Lewis MA, Byrne BJ. A new method for recombinant adeno-associated virus vector delivery to murine diaphragm. Molecular Therapy : the Journal of the American Society of Gene Therapy. 9: 458-63. PMID 15006614 DOI: 10.1016/j.ymthe.2004.01.006  0.52
2002 Zolotukhin S, Potter M, Zolotukhin I, Sakai Y, Loiler S, Fraites TJ, Chiodo VA, Phillipsberg T, Muzyczka N, Hauswirth WW, Flotte TR, Byrne BJ, Snyder RO. Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors. Methods (San Diego, Calif.). 28: 158-67. PMID 12413414 DOI: 10.1016/S1046-2023(02)00220-7  0.52
2002 Mah C, Fraites TJ, Zolotukhin I, Song S, Flotte TR, Dobson J, Batich C, Byrne BJ. Improved method of recombinant AAV2 delivery for systemic targeted gene therapy Molecular Therapy. 6: 106-112. PMID 12095310 DOI: 10.1006/mthe.2001.0636  0.52
2002 Fraites TJ, Schleissing MR, Shanely RA, Walter GA, Cloutier DA, Zolotukhin I, Pauly DF, Raben N, Plotz PH, Powers SK, Kessler PD, Byrne BJ. Correction of the enzymatic and functional deficits in a model of pompe disease using adeno-associated virus vectors Molecular Therapy. 5: 571-578. PMID 11991748 DOI: 10.1006/mthe.2002.0580  0.52
2001 Pauly DF, Fraites TJ, Toma C, Bayes HS, Huie ML, Hirschhorn R, Plotz PH, Raben N, Kessler PD, Byrne BJ. Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease. Human Gene Therapy. 12: 527-38. PMID 11268285 DOI: 10.1089/104303401300042447  0.44
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