| Year |
Citation |
Score |
| 2023 |
Shoti J, Qing K, Keeler GD, Duan D, Byrne BJ, Srivastava A. Development of capsid- and genome-modified optimized AAVrh74 vectors for muscle gene therapy. Molecular Therapy. Methods & Clinical Development. 31: 101147. PMID 38046199 DOI: 10.1016/j.omtm.2023.101147 |
0.327 |
|
| 2023 |
Schweitzer GG, Ditzenberger GL, Hughey CC, Finck BN, Martino MR, Pacak CA, Byrne BJ, Cade WT. Elevated liver glycogenolysis mediates higher blood glucose during acute exercise in Barth syndrome. Plos One. 18: e0290832. PMID 37651450 DOI: 10.1371/journal.pone.0290832 |
0.62 |
|
| 2023 |
Corti M, Byrne BJ, Gessler DJ, Thompson G, Norman S, Lammers J, Coleman KE, Liberati C, Elder ME, Escolar ML, Tuna IS, Mesaros C, Kleiner GI, Barbouth DS, Gray-Edwards HL, et al. Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation. Molecular Therapy. Methods & Clinical Development. 30: 303-314. PMID 37601414 DOI: 10.1016/j.omtm.2023.06.001 |
0.309 |
|
| 2023 |
Birch SM, Lawlor MW, Conlon TJ, Guo LJ, Crudele JM, Hawkins EC, Nghiem PP, Ahn M, Meng H, Beatka MJ, Fickau BA, Prieto JC, Styner MA, Struharik MJ, Shanks C, ... ... Byrne BJ, et al. Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy. Science Translational Medicine. 15: eabo1815. PMID 36599002 DOI: 10.1126/scitranslmed.abo1815 |
0.359 |
|
| 2022 |
Singer ML, Rana S, Benevides ES, Barral BE, Byrne BJ, Fuller DD. Chemogenetic activation of hypoglossal motoneurons in a mouse model of Pompe disease. Journal of Neurophysiology. PMID 35976060 DOI: 10.1152/jn.00026.2022 |
0.349 |
|
| 2021 |
Bohnert KL, Ditzenberger G, Bittel AJ, de Las Fuentes L, Corti M, Pacak CA, Taylor C, Byrne BJ, Reeds DN, Cade WT. Resistance exercise training with protein supplementation improves skeletal muscle strength and improves quality of life in late adolescents and young adults with Barth syndrome: A pilot study. Jimd Reports. 62: 74-84. PMID 34765401 DOI: 10.1002/jmd2.12244 |
0.616 |
|
| 2021 |
Doyle BM, Singer ML, Fleury-Curado T, Rana S, Benevides ES, Byrne BJ, Polotsky VY, Fuller DD. Gene delivery to the hypoglossal motor system: preclinical studies and translational potential. Gene Therapy. PMID 33574581 DOI: 10.1038/s41434-021-00225-1 |
0.323 |
|
| 2020 |
Cade WT, Bohnert KL, Bittel AJ, Chacko SJ, Patterson BW, Pacak CA, Byrne BJ, Vernon HJ, Reeds DN. Arginine kinetics are altered in a pilot sample of adolescents and young adults with Barth syndrome. Molecular Genetics and Metabolism Reports. 25: 100675. PMID 33204638 DOI: 10.1016/j.ymgmr.2020.100675 |
0.568 |
|
| 2020 |
Hakim CH, Clément N, Wasala LP, Yang HT, Yue Y, Zhang K, Kodippili K, Adamson-Small L, Pan X, Schneider JS, Yang NN, Chamberlain JS, Byrne BJ, Duan D. Micro-dystrophin AAV Vectors Made by Transient Transfection and Herpesvirus System Are Equally Potent in Treating mdx Mouse Muscle Disease. Molecular Therapy. Methods & Clinical Development. 18: 664-678. PMID 32775499 DOI: 10.1016/J.Omtm.2020.07.004 |
0.454 |
|
| 2020 |
Lins-Austin B, Patel S, Mietzsch M, Brooke D, Bennett A, Venkatakrishnan B, Van Vliet K, Smith AN, Long JR, McKenna R, Potter M, Byrne B, Boye SL, Bothner B, Heilbronn R, et al. Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking. Viruses. 12. PMID 32575696 DOI: 10.3390/V12060668 |
0.323 |
|
| 2020 |
Stockton DW, Kishnani P, van der Ploeg A, Llerena J, Boentert M, Roberts M, Byrne BJ, Araujo R, Maruti SS, Thibault N, Verhulst K, Berger KI. Respiratory function during enzyme replacement therapy in late-onset Pompe disease: longitudinal course, prognostic factors, and the impact of time from diagnosis to treatment start. Journal of Neurology. PMID 32524257 DOI: 10.1007/S00415-020-09936-8 |
0.38 |
|
| 2020 |
Perez BA, Shutterly A, Chan YK, Byrne BJ, Corti M. Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases. Brain Sciences. 10. PMID 32098339 DOI: 10.3390/Brainsci10020119 |
0.408 |
|
| 2020 |
Salabarria SM, Nair J, Clement N, Smith BK, Raben N, Fuller DD, Byrne BJ, Corti M. Advancements in AAV-mediated Gene Therapy for Pompe Disease. Journal of Neuromuscular Diseases. 7: 15-31. PMID 31796685 DOI: 10.3233/Jnd-190426 |
0.446 |
|
| 2019 |
Smith BK, Allen S, Mays S, Martin AD, Byrne BJ. Dynamic respiratory muscle function in late-onset Pompe disease. Scientific Reports. 9: 19006. PMID 31831753 DOI: 10.1038/S41598-019-54314-8 |
0.33 |
|
| 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 |
0.622 |
|
| 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 |
0.482 |
|
| 2019 |
Graham RJ, Muntoni F, Hughes I, Yum SW, Kuntz NL, Yang ML, Byrne BJ, Prasad S, Alvarez R, Genetti CA, Haselkorn T, James ES, LaRusso LB, Noursalehi M, Rico S, et al. Mortality and respiratory support in X-linked myotubular myopathy: a RECENSUS retrospective analysis. Archives of Disease in Childhood. PMID 31484632 DOI: 10.1136/Archdischild-2019-317910 |
0.31 |
|
| 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 |
0.471 |
|
| 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 |
0.658 |
|
| 2019 |
McCall AL, Stankov SG, Cowen G, Cloutier D, Zhang Z, Yang L, Clement N, Falk DJ, Byrne BJ. Reduction of Autophagic Accumulation in Pompe Disease Mouse Model Following Gene Therapy. Current Gene Therapy. PMID 31223086 DOI: 10.2174/1566523219666190621113807 |
0.475 |
|
| 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 |
0.605 |
|
| 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 |
0.698 |
|
| 2019 |
Taylor M, Jefferies J, Byrne B, Lima J, Ambale-Venkatesh B, Ostovaneh MR, Makkar R, Goldstein B, Smith RR, Fudge J, Malliaras K, Fedor B, Rudy J, Pogoda JM, Marbán L, et al. Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial. Neurology. PMID 30674601 DOI: 10.1212/Wnl.0000000000006950 |
0.357 |
|
| 2019 |
Kishnani P, Schoser B, Bratkovic D, Byrne BJ, Clemens PR, Goker-Alpan O, Ming X, Roberts M, Schwenkreis P, Sivakumar K, van der Ploeg AT, Jain V, Sitaraman S, Barth JA, Lagast H, et al. First-in-human study of advanced and targeted acid α-glucosidase (AT-GAA) (ATB200/AT2221) in patients with Pompe disease: preliminary functional assessment results from the ATB200-02 trial Molecular Genetics and Metabolism. 126: S86. DOI: 10.1016/J.Ymgme.2018.12.212 |
0.361 |
|
| 2018 |
Pena LDM, Barohn RJ, Byrne BJ, Desnuelle C, Goker-Alpan O, Ladha S, Laforêt P, Mengel KE, Pestronk A, Pouget J, Schoser B, Straub V, Trivedi J, Van Damme P, Vissing J, et al. Safety, tolerability, pharmacokinetics, pharmacodynamics, and exploratory efficacy of the novel enzyme replacement therapy avalglucosidase alfa (neoGAA) in treatment-naïve and alglucosidase alfa-treated patients with late-onset Pompe disease: A phase 1, open-label, multicenter, multinational, ascending dose study. Neuromuscular Disorders : Nmd. PMID 30770310 DOI: 10.1016/J.Nmd.2018.12.004 |
0.334 |
|
| 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 |
0.727 |
|
| 2018 |
Keeler AM, Zieger M, Todeasa S, Mccall A, Gifford J, Bircsak S, Choudhury SR, Byrne BJ, Sena-Esteves M, ElMallah MK. Systemic delivery of AAVB1-GAA clears glycogen and prolongs survival in a mouse model of Pompe disease. Human Gene Therapy. PMID 29901418 DOI: 10.1089/Hum.2018.016 |
0.543 |
|
| 2018 |
Burg M, Rosebrough C, Drouin LM, Bennett A, Mietzsch M, Chipman P, McKenna R, Sousa D, Potter M, Byrne B, Jude Samulski R, Agbandje-McKenna M. Atomic structure of a rationally engineered gene delivery vector, AAV2.5. Journal of Structural Biology. PMID 29775653 DOI: 10.1016/J.Jsb.2018.05.004 |
0.363 |
|
| 2018 |
Batra A, Harrington A, Lott DJ, Willcocks R, Senesac C, McGehee W, Xu D, Mathur S, Daniels MJ, Rooney WD, Forbes SC, Triplett W, Deol JK, Arpan I, Bendixen R, ... ... Byrne B, et al. Two-Year Longitudinal changes in lower limb strength and its relation to loss in function in a large cohort of patients with Duchenne Muscular Dystrophy. American Journal of Physical Medicine & Rehabilitation. PMID 29734234 DOI: 10.1097/Phm.0000000000000957 |
0.354 |
|
| 2018 |
Barnard AM, Willcocks RJ, Finanger EL, Daniels MJ, Triplett WT, Rooney WD, Lott DJ, Forbes SC, Wang DJ, Senesac CR, Harrington AT, Finkel RS, Russman BS, Byrne BJ, Tennekoon GI, et al. Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy. Plos One. 13: e0194283. PMID 29554116 DOI: 10.1371/Journal.Pone.0194283 |
0.319 |
|
| 2018 |
Byrne BJ. Safety First: Perspective on Patient-Centered Development of AAV Gene Therapy Products. Molecular Therapy : the Journal of the American Society of Gene Therapy. 26: 669-671. PMID 29503193 DOI: 10.1016/J.Ymthe.2018.02.009 |
0.303 |
|
| 2018 |
Johnson FK, Bratkovic D, Byrne B, Clemens PR, Goker-Alpan O, Geberhiwot T, Kishnani P, Ming X, Mozaffar T, Roberts M, Schoser B, Schwenkreis P, Sivakumar K, van der Ploeg A, Sitaraman S, et al. First-in-human preliminary pharmacokinetic data on a novel recombinant acid α-glucosidase, ATB200, co-administered with the pharmacological chaperone, AT2221, in patients with late-onset Pompe disease Molecular Genetics and Metabolism. 123: S71-S72. DOI: 10.1016/J.Ymgme.2017.12.177 |
0.322 |
|
| 2017 |
Chien YH, Lee NC, Tseng SH, Tai CH, Muramatsu SI, Byrne BJ, Hwu WL. Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial. The Lancet. Child & Adolescent Health. 1: 265-273. PMID 30169182 DOI: 10.1016/S2352-4642(17)30125-6 |
0.352 |
|
| 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 |
0.302 |
|
| 2017 |
Baligand C, Todd AG, Lee-McMullen B, Vohra RS, Byrne BJ, Falk DJ, Walter GA. (13)C/(31)P MRS Metabolic Biomarkers of Disease Progression and Response to AAV Delivery of hGAA in a Mouse Model of Pompe Disease. Molecular Therapy. Methods & Clinical Development. 7: 42-49. PMID 29018835 DOI: 10.1016/J.Omtm.2017.09.002 |
0.359 |
|
| 2017 |
Lee NC, Hwu WL, Muramatsu SI, Falk DJ, Byrne BJ, Cheng CH, Shih NC, Chang KL, Tsai LK, Chien YH. A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice. Molecular Neurobiology. PMID 28895054 DOI: 10.1007/S12035-017-0763-4 |
0.464 |
|
| 2017 |
Byrne BJ, Geberhiwot T, Barshop BA, Barohn R, Hughes D, Bratkovic D, Desnuelle C, Laforet P, Mengel E, Roberts M, Haroldsen P, Reilley K, Jayaram K, Yang K, Walsh L, et al. A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease. Orphanet Journal of Rare Diseases. 12: 144. PMID 28838325 DOI: 10.1186/S13023-017-0693-2 |
0.418 |
|
| 2017 |
Wang X, Herzog RW, Byrne BJ, Kumar SRP, Zhou Q, Buchholz CJ, Biswas M. Immune Modulatory Cell Therapy for Hemophilia B Based on CD20-Targeted Lentiviral Gene Transfer to Primary B Cells. Molecular Therapy. Methods & Clinical Development. 5: 76-82. PMID 28480307 DOI: 10.1016/J.Omtm.2017.03.005 |
0.355 |
|
| 2017 |
Kishnani P, Tarnopolsky M, Roberts M, Sivakumar K, Dasouki M, Dimachkie MM, Finanger E, Goker-Alpan O, Guter KA, Mozaffar T, Pervaiz MA, Laforet P, Levine T, Adera M, Lazauskas R, ... ... Byrne B, et al. Duvoglustat HCl Increases Systemic and Tissue Exposure of Active Acid α-Glucosidase in Pompe Patients Co-administered with Alglucosidase α. Molecular Therapy : the Journal of the American Society of Gene Therapy. PMID 28341561 DOI: 10.1016/J.Ymthe.2017.02.017 |
0.394 |
|
| 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 |
0.457 |
|
| 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 |
0.642 |
|
| 2017 |
Adamson-Small L, Potter M, Byrne BJ, Clement N. Sodium chloride enhances rAAV production in a serum-free suspension manufacturing platform using the Herpes Simplex Virus System. Human Gene Therapy Methods. PMID 28117600 DOI: 10.1089/Hgtb.2016.151 |
0.323 |
|
| 2017 |
Stockton DW, Berger KI, Boentert M, Byrne B, Kishnani PS, Llerena JC, Roberts M, Maruti S, Araujo R. Impact of earlier treatment on respiratory function in patients with late-onset Pompe disease: data from the Pompe Registry Molecular Genetics and Metabolism. 120. DOI: 10.1016/J.Ymgme.2016.11.335 |
0.315 |
|
| 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 |
0.735 |
|
| 2016 |
Lee NC, Lee YM, Chen PW, Byrne BJ, Hwu WL. Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene. Human Molecular Genetics. PMID 27658936 DOI: 10.1093/Hmg/Ddw323 |
0.347 |
|
| 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 |
0.313 |
|
| 2016 |
Smith BK, Martin AD, Lawson LA, Vernot V, Marcus J, Islam S, Shafi N, Corti M, Collins SW, Byrne BJ. Inspiratory muscle conditioning exercise and diaphragm gene therapy in Pompe disease: Clinical evidence of respiratory plasticity. Experimental Neurology. PMID 27453480 DOI: 10.1016/J.Expneurol.2016.07.013 |
0.387 |
|
| 2016 |
Tseng YS, Vliet KV, Rao L, McKenna R, Byrne BJ, Asokan A, Agbandje-McKenna M. Generation and characterization of anti-AAV8 and anti-AAV9 monoclonal antibodies. Journal of Virological Methods. PMID 27424005 DOI: 10.1016/J.Jviromet.2016.07.009 |
0.314 |
|
| 2016 |
Adamson-Small L, Potter M, Falk DJ, Cleaver B, Byrne BJ, Clément N. A scalable method for the production of high-titer and high-quality adeno-associated type 9 vectors using the HSV platform. Molecular Therapy. Methods & Clinical Development. 3: 16031. PMID 27222839 DOI: 10.1038/Mtm.2016.31 |
0.361 |
|
| 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 |
0.364 |
|
| 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 |
0.359 |
|
| 2016 |
Willcocks RJ, Rooney WD, Triplett WT, Forbes SC, Lott DJ, Senesac CR, Daniels MJ, Wang DJ, Harrington AT, Tennekoon GI, Russman BS, Finanger EL, Byrne BJ, Finkel RS, Walter GA, et al. Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort. Annals of Neurology. PMID 26891991 DOI: 10.1002/Ana.24599 |
0.302 |
|
| 2016 |
Doerfler PA, Nayak S, Corti M, Morel L, Herzog RW, Byrne BJ. Targeted approaches to induce immune tolerance for Pompe disease therapy. Molecular Therapy. Methods & Clinical Development. 3: 15053. PMID 26858964 DOI: 10.1038/Mtm.2015.53 |
0.41 |
|
| 2016 |
Doerfler PA, Todd AG, Clément N, Falk DJ, Nayak S, Herzog RW, Byrne BJ. Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease. Human Gene Therapy. 27: 43-59. PMID 26603344 DOI: 10.1089/Hum.2015.103 |
0.436 |
|
| 2016 |
Corti M, Cleaver B, Clement N, Conlon T, Faris K, Wang G, Benson J, Tarantal A, Fuller D, Herzog R, Byrne BJ. 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. 24: S186-S187. DOI: 10.1016/S1525-0016(16)33281-6 |
0.493 |
|
| 2016 |
McCall AL, Stankov SG, Falk DJ, Byrne BJ. 382. Correction of Autophagic Dysregulation in Pompe Disease Following Gene Therapy Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33191-4 |
0.479 |
|
| 2016 |
Lee N, Lee Y, Chien Y, Byrne B, Hwu W. 372. U1 snRNA-Mediated Correction of a Splicing Error of the Dopa Decarboxylase Gene Molecular Therapy. 24. DOI: 10.1016/S1525-0016(16)33181-1 |
0.361 |
|
| 2016 |
Bolfer L, Estrada AH, Winter B, Taggart K, Lourenço F, Terada N, Byrne BJ, Conlon TJ, Pacak CA. 180. The Role of the Pyruvate Dehydrogenase Kinase 4 (PDK4) Gene Mutation in the Development of Dilated Cardiomyopathy in Dobermanpinschers and Potential Application of Cardiac Gene Therapy Molecular Therapy. 24: S70-S71. DOI: 10.1016/S1525-0016(16)32989-6 |
0.671 |
|
| 2016 |
Pena L, Baron R, Byrne B, Desnuelle C, Goker-Alpan O, Ladha S, Laforet P, Mengel E, Pestronki A, Pouget J, Schoser B, Straub V, Trivedi J, Damme PV, Vissing J, et al. Phase 1 exploratory efficacy of the novel enzyme replacement therapy neoGAA in treatment-naive and alglucosidase alfa-treated late-onset Pompe disease patients Molecular Genetics and Metabolism. 117. DOI: 10.1016/J.Ymgme.2015.12.396 |
0.328 |
|
| 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. 23: 1572-1581. PMID 28141997 DOI: 10.1038/mt.2015.122 |
0.327 |
|
| 2015 |
Chien YH, van der Ploeg A, Jones S, Byrne B, Vellodi A, Leslie N, Mengel E, Shankar SP, Tanpaiboon P, Stockton DW, Hennermann JB, Devecseri Z, Kempf J, Keutzer J, Kishnani P. Survival and Developmental Milestones Among Pompe Registry Patients with Classic Infantile-Onset Pompe Disease with Different Timing of Initiation of Treatment with Enzyme Replacement Therapy. Journal of Neuromuscular Diseases. 2: S61-S62. PMID 27858651 DOI: 10.1016/J.Ymgme.2013.12.057 |
0.355 |
|
| 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 |
0.351 |
|
| 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 |
0.495 |
|
| 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 |
0.438 |
|
| 2015 |
Su J, Sherman A, Doerfler PA, Byrne BJ, Herzog RW, Daniell H. Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice. Plant Biotechnology Journal. 13: 1023-32. PMID 26053072 DOI: 10.1111/Pbi.12413 |
0.403 |
|
| 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 |
0.474 |
|
| 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 |
0.46 |
|
| 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 |
0.393 |
|
| 2015 |
Doerfler PA, Nayak S, Herzog RW, Morel L, Byrne BJ. BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease. Clinical Immunology (Orlando, Fla.). 158: 140-7. PMID 25842186 DOI: 10.1016/J.Clim.2015.03.022 |
0.346 |
|
| 2015 |
Cheever TR, Berkley D, Braun S, Brown RH, Byrne BJ, Chamberlain JS, Cwik V, Duan D, Federoff HJ, High KA, Kaspar BK, Klinger KW, Larkindale J, Lincecum J, Mavilio F, et al. Perspectives on best practices for gene therapy programs. Human Gene Therapy. 26: 127-33. PMID 25654329 DOI: 10.1089/Hum.2014.147 |
0.324 |
|
| 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 |
0.413 |
|
| 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 |
0.459 |
|
| 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 |
0.372 |
|
| 2015 |
McCall A, Cloutier D, Falk D, Clement N, Byrne B. 615. Characterizing the Endogenous MTM1 Promoter Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)34224-1 |
0.503 |
|
| 2015 |
Falk DJ, Soustek MS, Gary Todd A, Mah CS, Cloutier DA, Clement N, Fuller DD, Byrne BJ. 401. AAV Therapy Attenuates Respiratory Dysfunction and Glycogen Accumulation in a Murine Model of Glycogen Storage Disease Type II Molecular Therapy. 23: S159. DOI: 10.1016/S1525-0016(16)34010-2 |
0.488 |
|
| 2015 |
Todd AG, McElroy JA, Grange RW, Fuller DD, Walter GA, Byrne BJ, Falk DJ. 399. Early Correction of Neuromuscular Deficits in Pompe Disease Is Required for Restoration of Strength Following Gene Therapy Molecular Therapy. 23: S158. DOI: 10.1016/S1525-0016(16)34008-4 |
0.474 |
|
| 2015 |
Doerfler PA, Todd AG, Clément N, Falk DJ, Herzog RW, Byrne BJ. 368. Simultaneous Delivery of Dual AAV9 Vectors Induces Immune Tolerance and Correction of Pompe Disease Pathology Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33977-6 |
0.487 |
|
| 2015 |
Hwu W, Muramatsu S, Lee N, Chien Y, Tseng S, Tzen K, Snyder RO, Byrne BJ, Tai C, Wu R. C-11. An Update on Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Molecular Therapy. 23: S103. DOI: 10.1016/S1525-0016(16)33868-0 |
0.385 |
|
| 2015 |
Lee N, Muramatsu S, Chien Y, Chen P, Tzen K, Byrne BJ, Hwu W. 201. Neuron-Specific Systemic Gene Therapy for Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Molecular Therapy. 23. DOI: 10.1016/S1525-0016(16)33806-0 |
0.431 |
|
| 2015 |
Love-Leonor T, Corti M, Boye S, Donate A, Santostefano K, Cloutier D, Clemente N, Pacak C, Byrne B. 191. Frataxin: A Putative Biomarker for Minimal Effective Dosage of AAV Gene Therapy Molecular Therapy. 23: S76. DOI: 10.1016/S1525-0016(16)33796-0 |
0.698 |
|
| 2015 |
Donate AL, Soustek M, Rizzo SA, Santostefano K, Gosiker BJ, Lewin AS, Terada N, Cade T, Byrne BJ, Pacak CA. 187. AAV Delivery of Tafazzin for the Correction of Mitochondrial Dysfunction in Barth Syndrome Using IPS Differentiated Cardiomyocytes Molecular Therapy. 23: S75. DOI: 10.1016/S1525-0016(16)33792-3 |
0.688 |
|
| 2015 |
Hiwot T, Barohn R, Bratkovic D, Byrne B, Desnuelle C, Hughes D, Laforêt P, Mengel E, Roberts M, Yang K, Heusner C, Dummer W. Reveglucosidase alfa (BMN 701), a GILT-tagged recombinant human acid alpha glucosidase (rhGAA), evaluation in late-onset Pompe disease: Preliminary clinical efficacy and safety results of an extension study (72-week results) Neuromuscular Disorders. 25. DOI: 10.1016/J.Nmd.2015.06.453 |
0.313 |
|
| 2014 |
Potter M, Lins B, Mietzsch M, Heilbronn R, Van Vliet K, Chipman P, Agbandje-McKenna M, Cleaver BD, Clément N, Byrne BJ, Zolotukhin S. A simplified purification protocol for recombinant adeno-associated virus vectors. Molecular Therapy. Methods & Clinical Development. 1: 14034. PMID 26015974 DOI: 10.1038/Mtm.2014.34 |
0.312 |
|
| 2014 |
Corti M, Elder M, Falk D, Lawson L, Smith B, Nayak S, Conlon T, Clément N, Erger K, Lavassani E, Green M, Doerfler P, Herzog R, Byrne B. B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study. Molecular Therapy. Methods & Clinical Development. 1. PMID 25541616 DOI: 10.1038/Mtm.2014.33 |
0.406 |
|
| 2014 |
Forbes SC, Willcocks RJ, Triplett WT, Rooney WD, Lott DJ, Wang DJ, Pollaro J, Senesac CR, Daniels MJ, Finkel RS, Russman BS, Byrne BJ, Finanger EL, Tennekoon GI, Walter GA, et al. Magnetic resonance imaging and spectroscopy assessment of lower extremity skeletal muscles in boys with Duchenne muscular dystrophy: a multicenter cross sectional study. Plos One. 9: e106435. PMID 25203313 DOI: 10.1371/Journal.Pone.0106435 |
0.352 |
|
| 2014 |
Doerfler PA, Byrne BJ, Clément N. Copackaging of multiple adeno-associated viral vectors in a single production step. Human Gene Therapy Methods. 25: 269-76. PMID 25143183 DOI: 10.1089/Hgtb.2014.055 |
0.365 |
|
| 2014 |
Arpan I, Willcocks RJ, Forbes SC, Finkel RS, Lott DJ, Rooney WD, Triplett WT, Senesac CR, Daniels MJ, Byrne BJ, Finanger EL, Russman BS, Wang DJ, Tennekoon GI, Walter GA, et al. Examination of effects of corticosteroids on skeletal muscles of boys with DMD using MRI and MRS. Neurology. 83: 974-80. PMID 25098537 DOI: 10.1212/Wnl.0000000000000775 |
0.322 |
|
| 2014 |
Nayak S, Doerfler PA, Porvasnik SL, Cloutier DD, Khanna R, Valenzano KJ, Herzog RW, Byrne BJ. Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent. Plos One. 9: e98336. PMID 24897114 DOI: 10.1371/Journal.Pone.0098336 |
0.387 |
|
| 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 |
0.504 |
|
| 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 |
0.461 |
|
| 2014 |
Byrne B, Barohn R, Barshop B, Bratkovic D, Desnuelle C, Henderson R, Hiwot T, Hughes D, Laforet P, Mengel E, Roberts M, Lang W, LeBowitz J. Preliminary clinical efficacy and safety of BMN 701, GILT-tagged recombinant human acid alpha glucosidase (rhGAA), in late-onset Pompe disease: results of an extension study Molecular Genetics and Metabolism. 111. DOI: 10.1016/J.Ymgme.2013.12.050 |
0.333 |
|
| 2014 |
Benjamin ER, Wu X, Valle MCD, Sjoberg E, Sitaraman S, Stevens A, Gomez N, Katz E, Pruthi F, Kelley JS, Wustman B, Valenzano KJ, Byrne BJ, Barlow C, Lockhart DJ. Strategy to assess the effect of duvoglustat co-administered with alglucosidase alfa infusion on the immune response to enzyme replacement therapy for Pompe disease Molecular Genetics and Metabolism. 111. DOI: 10.1016/J.Ymgme.2013.12.038 |
0.327 |
|
| 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 |
0.398 |
|
| 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 |
0.427 |
|
| 2013 |
Byrne BJ. Pathway for approval of a gene therapy orphan product: treading new ground. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 1465-6. PMID 23903569 DOI: 10.1038/Mt.2013.157 |
0.344 |
|
| 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 |
0.424 |
|
| 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 |
0.527 |
|
| 2013 |
Elder ME, Nayak S, Collins SW, Lawson LA, Kelley JS, Herzog RW, Modica RF, Lew J, Lawrence RM, Byrne BJ. B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease. The Journal of Pediatrics. 163: 847-54.e1. PMID 23601496 DOI: 10.1016/J.Jpeds.2013.03.002 |
0.317 |
|
| 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 |
0.458 |
|
| 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 |
0.361 |
|
| 2013 |
Tremblay JP, Xiao X, Aartsma-Rus A, Barbas C, Blau HM, Bogdanove AJ, Boycott K, Braun S, Breakefield XO, Bueren JA, Buschmann M, Byrne BJ, Calos M, Cathomen T, Chamberlain J, et al. Translating the genomics revolution: the need for an international gene therapy consortium for monogenic diseases. Molecular Therapy : the Journal of the American Society of Gene Therapy. 21: 266-8. PMID 23369965 DOI: 10.1038/Mt.2013.4 |
0.382 |
|
| 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 |
0.443 |
|
| 2013 |
Smith B, Martin D, Fuller D, Lawson LA, Lottenberg L, Islam S, Byrne B. Diaphragm pacing augments ventilatory function in Pompe disease Molecular Genetics and Metabolism. 108: S86. DOI: 10.1016/J.Ymgme.2012.11.232 |
0.334 |
|
| 2013 |
Phillips D, Lawson LA, Smith B, Martin A, Byrne B. Fatigue and motor gains in children with Pompe disease undergoing gene replacement therapy for chronic ventilatory failure Molecular Genetics and Metabolism. 108. DOI: 10.1016/J.Ymgme.2012.11.196 |
0.306 |
|
| 2013 |
Fuller D, Elmallah M, Falk D, Lawson LA, Martin AD, Smith B, Byrne B. The importance of targeting the central nervous system for treatment of respiratory insufficiency in Pompe disease Molecular Genetics and Metabolism. 108: S41. DOI: 10.1016/J.Ymgme.2012.11.089 |
0.31 |
|
| 2013 |
Byrne B, Smith B, Lawson LA, Martin D, Mah C, Conlon T, Phillips D, Collins S. One year outcomes of diaphragm gene therapy in ventilator dependent children with Pompe disease Molecular Genetics and Metabolism. 108. DOI: 10.1016/J.Ymgme.2012.11.047 |
0.323 |
|
| 2013 |
Falk DJ, Todd AG, Soustek MS, Cloutier DC, ElMallah M, Fuller DD, Byrne BJ. P.17.9 AAV9 vector encoding hGAA improves synaptic pathology of the neuromuscular junction in Pompe disease Neuromuscular Disorders. 23: 829. DOI: 10.1016/J.Nmd.2013.06.659 |
0.457 |
|
| 2013 |
Corti M, Baligand C, Falk L, Walter GA, Byrne BJ. P.17.4 Magnetic Resonance Imaging (MRI) to evaluate the effect of enzyme replacement therapy in Late Onset Pompe Disease (LOPD) Neuromuscular Disorders. 23: 827. DOI: 10.1016/J.Nmd.2013.06.654 |
0.437 |
|
| 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 |
0.397 |
|
| 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 |
0.316 |
|
| 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 |
0.364 |
|
| 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 |
0.411 |
|
| 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 |
0.397 |
|
| 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 |
0.434 |
|
| 2012 |
Sack BK, Merchant S, Markusic DM, Nathwani AC, Davidoff AM, Byrne BJ, Herzog RW. Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. Plos One. 7: e37671. PMID 22655063 DOI: 10.1371/Journal.Pone.0037671 |
0.412 |
|
| 2012 |
Hwu WL, Muramatsu S, Tseng SH, Tzen KY, Lee NC, Chien YH, Snyder RO, Byrne BJ, Tai CH, Wu RM. Gene therapy for aromatic L-amino acid decarboxylase deficiency. Science Translational Medicine. 4: 134ra61. PMID 22593174 DOI: 10.1126/Scitranslmed.3003640 |
0.363 |
|
| 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 |
0.333 |
|
| 2012 |
Nayak S, Sivakumar R, Cao O, Daniell H, Byrne BJ, Herzog RW. Mapping the T helper cell response to acid α-glucosidase in Pompe mice. Molecular Genetics and Metabolism. 106: 189-95. PMID 22494547 DOI: 10.1016/J.Ymgme.2012.03.009 |
0.375 |
|
| 2012 |
Stedman HH, Byrne BJ. Signs of progress in gene therapy for muscular dystrophy also warrant caution. Molecular Therapy : the Journal of the American Society of Gene Therapy. 20: 249-51. PMID 22297820 DOI: 10.1038/Mt.2011.307 |
0.413 |
|
| 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 |
0.415 |
|
| 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 |
0.342 |
|
| 2012 |
Smith BK, Martin D, Lawson L, Collins S, Byrne BJ. Inspiratory Muscle Conditioning Improves Coordination But Not Strength Prior To Diaphragm Gene Therapy For Infantile Pompe Disease Cardiopulmonary Physical Therapy Journal. 23: 28-29. DOI: 10.1097/01823246-201223040-00010 |
0.422 |
|
| 2012 |
Byrne B, Smith B, Martin A, Mah C, Lawson LA, Islam S, Conlon T, Phillips D, Collins S. Phase I/II Trial of Adeno-associated Virus Acid-alpha-Glucosidase (AAV-GAA) Diaphragm Gene Therapy for Ventilatory Failure in Pompe Disease Molecular Genetics and Metabolism. 105: S24. DOI: 10.1016/J.Ymgme.2011.11.039 |
0.369 |
|
| 2012 |
Kishnani P, Tarnopolsky M, Sivakumar K, Byrne B, Goker-Alpan O, Guter K, Pervaiz M, Dasouki M, Levine T, Roberts M, Johnson F, Valenzano K, Lockhart D, Boudes P. T.P.46 A novel phase 2a study design to investigate drug-drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha-glucosidase in subjects with Pompe disease Neuromuscular Disorders. 22: 852. DOI: 10.1016/J.Nmd.2012.06.165 |
0.391 |
|
| 2012 |
Kishnani P, Tarnopolsky M, Sivakumar K, Byrne B, Goker-Alpan O, Guter K, Pervaiz M, Dasouki M, Levine T, Roberts M, Johnson F, Sitaraman S, Khanna R, Flanagan J, Sjoberg E, et al. T.P.45 An ongoing phase 2a study to investigate drug–drug interactions between escalating doses of AT2220 (duvoglustat hydrochloride) and acid alpha glucosidase in subjects with Pompe disease – Preliminary results Neuromuscular Disorders. 22: 852. DOI: 10.1016/J.Nmd.2012.06.164 |
0.367 |
|
| 2011 |
Nayak S, Sarkar D, Perrin GQ, Moghimi B, Hoffman BE, Zhou S, Byrne BJ, Herzog RW. Prevention and Reversal of Antibody Responses Against Factor IX in Gene Therapy for Hemophilia B. Frontiers in Microbiology. 2: 244. PMID 22279442 DOI: 10.3389/Fmicb.2011.00244 |
0.379 |
|
| 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 |
0.325 |
|
| 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 |
0.698 |
|
| 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 |
0.426 |
|
| 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 |
0.69 |
|
| 2011 |
Byrne BJ, Kishnani PS, Case LE, Merlini L, Müller-Felber W, Prasad S, van der Ploeg A. Pompe disease: design, methodology, and early findings from the Pompe Registry. Molecular Genetics and Metabolism. 103: 1-11. PMID 21439876 DOI: 10.1016/J.Ymgme.2011.02.004 |
0.375 |
|
| 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 |
0.367 |
|
| 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 |
0.311 |
|
| 2011 |
Ma W, Li B, Ling C, Jayandharan GR, Srivastava A, Byrne BJ. A simple method to increase the transduction efficiency of single-stranded adeno-associated virus vectors in vitro and in vivo. Human Gene Therapy. 22: 633-40. PMID 21219084 DOI: 10.1089/Hum.2010.243 |
0.388 |
|
| 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 |
0.448 |
|
| 2011 |
Falk D, Soustek MS, Mah CS, Cloutier DA, Germain SA, Kelley JS, Erger KE, Conlon TJ, Clement N, Byrne BJ. Next generation treatment of Pompe disease using systemic gene transfer with AAV9 Molecular Genetics and Metabolism. 102. DOI: 10.1016/J.Ymgme.2010.11.062 |
0.337 |
|
| 2011 |
Forbes S, Walter G, Rooney W, Wang D, DeVos S, Pollaro J, Triplett W, Lott D, Senesac C, Daniels M, Willcocks R, Byrne B, Russman B, Finkel R, Sweeney H, et al. P1.41 Design of a multi-center study to examine skeletal muscles of children with Duchenne muscular dystrophy using MRI/MRS Neuromuscular Disorders. 21: 653-654. DOI: 10.1016/J.Nmd.2011.06.801 |
0.318 |
|
| 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 |
0.381 |
|
| 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 |
0.375 |
|
| 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 |
0.42 |
|
| 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 |
0.804 |
|
| 2010 |
Sack B, Moghimi B, Byrne B, Herzog RW. Anti-CD20 to Control Antibody Formation Against FVIII In Gene and Protein Replacement Therapy Blood. 116: 711-711. DOI: 10.1182/Blood.V116.21.711.711 |
0.373 |
|
| 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 |
0.363 |
|
| 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 |
0.39 |
|
| 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 |
0.307 |
|
| 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 |
0.325 |
|
| 2009 |
Martino AT, Nayak S, Hoffman BE, Cooper M, Liao G, Markusic DM, Byrne BJ, Terhorst C, Herzog RW. Tolerance induction to cytoplasmic beta-galactosidase by hepatic AAV gene transfer: implications for antigen presentation and immunotoxicity. Plos One. 4: e6376. PMID 19652717 DOI: 10.1371/Journal.Pone.0006376 |
0.354 |
|
| 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 |
0.322 |
|
| 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 |
0.311 |
|
| 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 |
0.36 |
|
| 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 |
0.779 |
|
| 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 |
0.729 |
|
| 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 |
0.72 |
|
| 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 |
0.344 |
|
| 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 |
0.329 |
|
| 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 |
0.399 |
|
| 2008 |
Byrne B, Kishnani P, Case L, Merlini L, Müeller-Felber W, Ploeg AVd, Marsden D, Prasad S. The Pompe Registry: tracking Pompe disease symptoms in a broad patient population Pediatric Rheumatology. 6: 159. DOI: 10.1186/1546-0096-6-S1-P159 |
0.31 |
|
| 2008 |
Hauswirth W, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon T, Boye SL, Flotte TR, Byrne B, Jacobson SG. Phase I Trial of Leber Congenital Amaurosis due to RPE65 Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector: Short-Term Results Human Gene Therapy. 81015093227032. DOI: 10.1089/Hgt.2008.107 |
0.336 |
|
| 2007 |
Nam HJ, Lane MD, Padron E, Gurda B, McKenna R, Kohlbrenner E, Aslanidi G, Byrne B, Muzyczka N, Zolotukhin S, Agbandje-McKenna M. Structure of adeno-associated virus serotype 8, a gene therapy vector. Journal of Virology. 81: 12260-71. PMID 17728238 DOI: 10.1128/Jvi.01304-07 |
0.388 |
|
| 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 |
0.714 |
|
| 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 |
0.401 |
|
| 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 |
0.82 |
|
| 2007 |
Kishnani PS, Corzo D, Nicolino M, Byrne B, Mandel H, Hwu WL, Leslie N, Levine J, Spencer C, McDonald M, Li J, Dumontier J, Halberthal M, Chien YH, Hopkin R, et al. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease. Neurology. 68: 99-109. PMID 17151339 DOI: 10.1212/01.Wnl.0000251268.41188.04 |
0.395 |
|
| 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 |
0.373 |
|
| 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 |
0.734 |
|
| 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 |
0.323 |
|
| 2006 |
Howell RR, Byrne B, Darras BT, Kishnani P, Nicolino M, Ploeg Avd. Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome. Genetics in Medicine. 8: 289-296. PMID 16702878 DOI: 10.1097/01.Gim.0000204462.42910.B8 |
0.344 |
|
| 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 |
0.312 |
|
| 2006 |
DeRuisseau LR, Mah C, Fuller DD, Byrne BJ. 892. Neural Deficits Contribute to Respiratory Insufficiency in Pompe Disease: A Therapeutic Approach with AAV1 Molecular Therapy. 13: S344. DOI: 10.1016/J.Ymthe.2006.08.981 |
0.774 |
|
| 2006 |
Brantly ML, Spencer T, Spencer CT, Conlon TJ, Song S, Humphries M, Byrne BJ, Snyder RO, Flotte TR. 728. Safety and Bioactivity of rAAV2-hAAT in alpha-1 Antitrypsin-Deficient Patients in a Phase I Clinical Trial Molecular Therapy. 13: S281. DOI: 10.1016/J.Ymthe.2006.08.808 |
0.449 |
|
| 2006 |
Andino LM, Byrne BJ, Kasahara H, Lewin AS. 709. Adeno-Associated Virus Delivery of siRNAs Leads to a Reduction in Phospholamban Levels Molecular Therapy. 13: S274. DOI: 10.1016/J.Ymthe.2006.08.788 |
0.382 |
|
| 2006 |
Mah C, DeRuisseau LR, Pacak CA, Lewis MA, Fuller DD, Byrne BJ. 575. Correction of Ventilation in Glycogen Storage Disease Type II Mice after Gel-Mediated Delivery of Adeno-Associated Virus Serotype 1 Vectors Molecular Therapy. 13: S221-S222. DOI: 10.1016/J.Ymthe.2006.08.648 |
0.825 |
|
| 2006 |
Mah C, Pacak CA, Cresawn KO, DeRuisseau LR, Germain S, Lewis MA, Fuller DD, Byrne BJ. 410. Physiological Correction of Glycogen Storage Disease Type II Using Adeno-Associated Virus Serotype 1 Vectors Molecular Therapy. 13. DOI: 10.1016/J.Ymthe.2006.08.473 |
0.818 |
|
| 2006 |
Pacak CA, Mah C, Cresawn KO, Lewis MA, Germain S, Byrne BJ. 28. rAAV2/9 Mediated Gene Delivery of Acid α-Glucosidase Corrects the Cardiac Phenotype in a Mouse Model of Pompe Disease Molecular Therapy. 13: S12. DOI: 10.1016/J.Ymthe.2006.08.040 |
0.839 |
|
| 2005 |
Lane MD, Nam HJ, Padron E, Gurda-Whitaker B, Kohlbrenner E, Aslanidi G, Byrne B, McKenna R, Muzyczka N, Zolotukhin S, Agbandje-McKenna M. Production, purification, crystallization and preliminary X-ray analysis of adeno-associated virus serotype 8. Acta Crystallographica. Section F, Structural Biology and Crystallization Communications. 61: 558-61. PMID 16511095 DOI: 10.1107/S1744309105014132 |
0.31 |
|
| 2005 |
Spencer CT, Byrne BJ, Gewitz MH, Wechsler SB, Kao AC, Gerstenfeld EP, Merliss AD, Carboni MP, Bryant RM. Ventricular arrhythmia in the X-linked cardiomyopathy Barth syndrome Pediatric Cardiology. 26: 632-637. PMID 16235007 DOI: 10.1007/S00246-005-0873-Z |
0.312 |
|
| 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 |
0.372 |
|
| 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 |
0.823 |
|
| 2005 |
Walker MC, Mandell TC, Crawford PC, Simon GG, Cahill KS, Fernandes PJ, MacLeod JN, Byrne BJ, Levy JK. Expression of erythropoietin in cats treated with a recombinant adeno-associated viral vector. American Journal of Veterinary Research. 66: 450-6. PMID 15822590 DOI: 10.2460/Ajvr.2005.66.450 |
0.403 |
|
| 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 |
0.82 |
|
| 2005 |
Mohiuddin I, Loiler S, Zolotukhin I, Byrne BJ, Flotte TR, Snyder RO. Herpesvirus-based infectious titering of recombinant adeno-associated viral vectors. Molecular Therapy : the Journal of the American Society of Gene Therapy. 11: 320-6. PMID 15668144 DOI: 10.1016/J.Ymthe.2004.08.030 |
0.413 |
|
| 2005 |
Porvasnik SL, Onal T, Sakai Y, Torres R, Walter GA, Byrne BJ, Spencer CT. 1117. Gene Therapy in a Rat Model of Pulmonary Hypertension Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.665 |
0.333 |
|
| 2005 |
Brantly ML, Humphries M, Song S, Conlon T, Poirier A, Byrne BJ, Snyder R, Flotte TR. 1077. Phase I Clinical Trial of Recombinant Adeno-Associated Virus (rAAV)-Alpha-1 Antitrypsin Vectors Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.624 |
0.458 |
|
| 2005 |
Cresawn KO, Mah CS, Zolotukhin I, Lewis MA, Byrne BJ. 911. Comparison of rAAV Serotype, Promoter, and Treatment Age for the Correction of Glycogen Storage Disease Type II Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.454 |
0.839 |
|
| 2005 |
Cresawn KO, Mah CS, Fraites TJ, Lewis MA, Zolotukhin I, Byrne BJ. 909. Sustained Correction of Glycogen Storage Disease Type II by rAAV1 Vector-Mediated Gene Therapy Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.452 |
0.844 |
|
| 2005 |
DeRuisseau LR, Mah CS, Fraites TJ, Fuller DD, Byrne BJ. 818. Recombinant AAV1-Mediated Treatment of CNS Glycogen Accumulation in Mouse Model of Glycogen Storage Disease Type II Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.07.361 |
0.822 |
|
| 2005 |
Kohlbrenner E, Aslanidi G, Nash K, Shklayev S, Campbell-Thompson M, Byrne BJ, Snyder RO, Muzyczka N, Warrington KH, Zolotukhin S. 400. Successful Production of Pseudotyped Mosaic rAAV Vectors Using a Modified Baculovirus Expression System Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.403 |
0.375 |
|
| 2005 |
Mah C, Cresawn KO, DeRuisseau LR, Fuller D, Byrne BJ. 354. Correction of Respiratory Function by Recombinant AAV1 Mediated Gene Therapy in a Murine Model of Glycogen Storage Disease Type II Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.357 |
0.819 |
|
| 2005 |
Pacak CA, Mah C, Gaidosh G, Lewis M, Torres R, Campbell K, Walter GA, Byrne BJ. 44. Development of AAV-Mediated Gene Therapy for Murine Models of Genetic Diseases Affecting the Heart Molecular Therapy. 11. DOI: 10.1016/J.Ymthe.2005.06.072 |
0.744 |
|
| 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 |
0.829 |
|
| 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 |
0.807 |
|
| 2004 |
Cresawn KO, Raben N, Plotz PH, Pacak C, Glick A, Byrne BJ. 859. A Skin-Restricted Conditionally Expressing Mouse Model of Pompe Disease for Assessment of Gene and Enzyme Replacement Therapies Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.767 |
0.817 |
|
| 2004 |
Simon GG, Byrne BJ, Atkinson MA. 565. Modulation of the Acute Phase Response by Recombinant Adeno-Associated Viral Expression of the IL-1 Type 1 Soluble Receptor Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.489 |
0.33 |
|
| 2004 |
Rucker MB, Tarantal AF, Conlon T, Flotte TR, Byrne BJ. 377. Expression of Acid |[alpha]|-Glucosidase in Nonhuman Primate Diaphragm and Peritoneum after Direct In Utero Delivery of Recombinant Adeno-Associated Virus Type 1 Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.311 |
0.502 |
|
| 2004 |
Pacak CA, Cloutier D, Zolotukhin I, Gaidosh GS, Campbell K, Walter GA, Byrne BJ. 251. rAAV-Mediated Gene Therapy To Treat Limb Girdle Muscular Dystrophy Type 2D (LGMD-2D) Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.192 |
0.716 |
|
| 2004 |
Cresawn KO, Wasserfall C, Atkinson MA, Mah C, Fraites TJ, Zolotukhin I, Byrne BJ. 250. Cross-Correction in Pompe Mice after Immunomodulation and Hepatic Delivery of Recombinant AAA5 and AAV8 Vectors Molecular Therapy. 9. DOI: 10.1016/J.Ymthe.2004.06.191 |
0.822 |
|
| 2003 |
Post H, Kajstura J, Lei B, Sessa WC, Byrne B, Anversa P, Hintze TH, Recchia FA. Adeno-associated virus mediated gene delivery into coronary microvessels of chronically instrumented dogs. Journal of Applied Physiology (Bethesda, Md. : 1985). 95: 1688-94. PMID 12844500 DOI: 10.1152/Japplphysiol.00896.2002 |
0.388 |
|
| 2003 |
Mah C, Sarkar R, Zolotukhin I, Schleissing M, Xiao X, Kazazian HH, Byrne BJ. Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice. Human Gene Therapy. 14: 143-52. PMID 12614565 DOI: 10.1089/104303403321070838 |
0.787 |
|
| 2002 |
Asfour B, Baba HA, Scheld HH, Hruban RH, Hammel D, Byrne BJ. Uniform long-term gene expression using adeno-associated virus (AAV) by ex vivo recirculation in rat-cardiac isografts. Thoracic and Cardiovascular Surgeon. 50: 347-350. PMID 12457311 DOI: 10.1055/S-2002-35745 |
0.408 |
|
| 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 |
0.801 |
|
| 2002 |
Mah C, Byrne BJ, Flotte TR. Virus-based gene delivery systems Clinical Pharmacokinetics. 41: 901-911. PMID 12222993 DOI: 10.2165/00003088-200241120-00001 |
0.414 |
|
| 2002 |
Sun MS, Pan CJ, Shieh JJ, Ghosh A, Chen LY, Mansfield BC, Ward JM, Byrne BJ, Chou JY. Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice. Human Molecular Genetics. 11: 2155-64. PMID 12189168 DOI: 10.1093/Hmg/11.18.2155 |
0.448 |
|
| 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 |
0.811 |
|
| 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 |
0.816 |
|
| 2002 |
Raben N, Plotz P, Byrne BJ. Acid a-Glucosidase Deficiency (Glycogenosis Type II, Pompe Disease) Current Molecular Medicine. 2: 145-166. PMID 11949932 DOI: 10.2174/1566524024605789 |
0.411 |
|
| 2002 |
Kapturczak M, Zolotukhin S, Cross J, Pileggi A, Molano RD, Jorgensen M, Byrne B, Flotte TR, Ellis T, Inverardi L, Ricordi C, Nick H, Atkinson M, Agarwal A. Transduction of human and mouse pancreatic islet cells using a bicistronic recombinant adeno-associated viral vector. Molecular Therapy : the Journal of the American Society of Gene Therapy. 5: 154-60. PMID 11829522 DOI: 10.1006/Mthe.2002.0522 |
0.38 |
|
| 2002 |
Toma C, Pittenger MF, Cahill KS, Byrne BJ, Kessler PD. Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart. Circulation. 105: 93-8. PMID 11772882 DOI: 10.1161/Hc0102.101442 |
0.325 |
|
| 2001 |
Raben N, Lu N, Nagaraju K, Rivera Y, Lee A, Yan B, Byrne B, Meikle PJ, Umapathysivam K, Hopwood JJ, Plotz PH. Conditional tissue-specific expression of the acid alpha-glucosidase (GAA) gene in the GAA knockout mice: implications for therapy. Human Molecular Genetics. 10: 2039-47. PMID 11590121 DOI: 10.1093/Hmg/10.19.2039 |
0.49 |
|
| 2001 |
Xu L, Daly T, Gao C, Flotte TR, Song S, Byrne BJ, Sands MS, Parker Ponder K. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Human Gene Therapy. 12: 563-73. PMID 11268288 DOI: 10.1089/104303401300042500 |
0.415 |
|
| 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 |
0.815 |
|
| 1999 |
Asfour B, Byrne BJ, Baba HA, Hammel D, Hruban RH, Weyand M, Deng M, Scheld HH. Effective gene transfer in the rat myocardium via adenovirus vectors using a coronary recirculation model. Thoracic and Cardiovascular Surgeon. 47: 311-316. PMID 10599959 DOI: 10.1055/S-2007-1013164 |
0.368 |
|
| 1999 |
Conway JE, Rhys CM, Zolotukhin I, Zolotukhin S, Muzyczka N, Hayward GS, Byrne BJ. High-titer recombinant adeno-associated virus production utilizing a recombinant herpes simplex virus type I vector expressing AAV-2 Rep and Cap. Gene Therapy. 6: 986-93. PMID 10455400 DOI: 10.1038/Sj.Gt.3300937 |
0.379 |
|
| 1998 |
Song S, Morgan M, Ellis T, Poirier A, Chesnut K, Wang J, Brantly M, Muzyczka N, Byrne BJ, Atkinson M, Flotte TR. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vectors. Proceedings of the National Academy of Sciences of the United States of America. 95: 14384-8. PMID 9826709 DOI: 10.1073/Pnas.95.24.14384 |
0.476 |
|
| 1998 |
Harrison RL, Byrne BJ, Tung L. Electroporation-mediated gene transfer in cardiac tissue Febs Letters. 435: 1-5. PMID 9755847 DOI: 10.1016/S0014-5793(98)00987-9 |
0.355 |
|
| 1998 |
Georgakopoulos D, Mitzner WA, Chen CH, Byrne BJ, Millar HD, Hare JM, Kass DA. In vivo murine left ventricular pressure-volume relations by miniaturized conductance micromanometry. The American Journal of Physiology. 274: H1416-22. PMID 9575947 DOI: 10.1152/Ajpheart.1998.274.4.H1416 |
0.303 |
|
| 1997 |
Conway JE, Zolotukhin S, Muzyczka N, Hayward GS, Byrne BJ. Recombinant adeno-associated virus type 2 replication and packaging is entirely supported by a herpes simplex virus type 1 amplicon expressing Rep and Cap. Journal of Virology. 71: 8780-9. PMID 9343238 DOI: 10.1128/Jvi.71.11.8780-8789.1997 |
0.398 |
|
| 1997 |
Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proceedings of the National Academy of Sciences of the United States of America. 93: 14082-7. PMID 8943064 DOI: 10.1073/Pnas.93.24.14082 |
0.479 |
|
| 1984 |
Hartzell SW, Byrne BJ, Subramanian KN. Mapping of the late promoter of simian virus 40 Proceedings of the National Academy of Sciences of the United States of America. 81: 23-27. PMID 6320166 DOI: 10.1073/Pnas.81.1.23 |
0.316 |
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