Works matching AU Byrne, Barry J.

Results: 103

AAV-mediated knockdown of phospholamban leads to improved contractility and calcium handling in cardiomyocytes.

Published in:

Journal of Gene Medicine, 2008, v. 10, n. 2, p. 132, doi. 10.1002/jgm.1131

By:

Andino, Lourdes M.;
Takeda, Morihiko;
Kasahara, Hideko;
Jakymiw, Andrew;
Byrne, Barry J.;
Lewin, Alfred S.

Publication type:

Article

Longitudinal Observational Study of Cardiac Outcome Risk Factor Prediction in Children, Adolescents, and Adults with Barth Syndrome.

Published in:

Pediatric Cardiology, 2022, v. 43, n. 6, p. 1251, doi. 10.1007/s00246-022-02846-8

By:

Chowdhury, Shahryar;
Jackson, Lanier;
Byrne, Barry J.;
Bryant, Randall M.;
Cade, W. Todd;
Churchill, Tammy Lane;
Buchanan, Julia;
Taylor, Carolyn

Publication type:

Article

Cardiac responses in paediatric Pompe disease in the ADVANCE patient cohort.

Published in:

Cardiology in the Young, 2022, v. 32, n. 3, p. 364, doi. 10.1017/S1047951121002079

By:

Byrne, Barry J.;
Colan, Steven D.;
Kishnani, Priya S.;
Foster, Meredith C.;
Sparks, Susan E.;
Gibson, James B.;
An Haack, Kristina;
Stockton, David W.;
Peña, Loren D. M.;
Hahn, Si Houn;
Johnson, Judith;
Tanpaiboon, Pranoot X.;
Leslie, Nancy D.;
Kronn, David;
Hillman, Richard E.;
Wang, Raymond Y.

Publication type:

Article

Analysis of regional congenital cardiac surgical outcomes in Florida using The Society of Thoracic Surgeons Congenital Heart Surgery Database.

Published in:

Cardiology in the Young, 2009, v. 19, n. 4, p. 360, doi. 10.1017/S1047951109990151

By:

Jacobs, Jeffrey P.;
Quintessenza, James A.;
Burke, Redmond P.;
Bleiweis, Mark S.;
Byrne, Barry J.;
Ceithaml, Eric L.;
DeCampli, William M.;
Giroud, Jorge M.;
Perryman, Richard A.;
Rosenkranz, Eliot R.;
Wolff, Grace;
Posner, Vicki;
Steverson, Sue;
Blanchard, William B.;
Schiebler, Gerry L.

Publication type:

Article

Peak oxygen uptake (VO<sub>2peak</sub>) across childhood, adolescence and young adulthood in Barth syndrome: Data from cross-sectional and longitudinal studies.

Published in:

PLoS ONE, 2018, v. 13, n. 5, p. 1, doi. 10.1371/journal.pone.0197776

By:

Cade, William Todd;
Bohnert, Kathryn L.;
Reeds, Dominic N.;
Peterson, Linda R.;
Bittel, Adam J.;
Bashir, Adil;
Byrne, Barry J.;
Taylor, Carolyn L.

Publication type:

Article

Skeletal muscle magnetic resonance biomarkers correlate with function and sentinel events in Duchenne muscular dystrophy.

Published in:

PLoS ONE, 2018, v. 13, n. 3, p. 1, doi. 10.1371/journal.pone.0194283

By:

Barnard, Alison M.;
Willcocks, Rebecca J.;
Finanger, Erika L.;
Daniels, Michael J.;
Triplett, William T.;
Rooney, William D.;
Lott, Donovan J.;
Forbes, Sean C.;
Wang, Dah-Jyuu;
Senesac, Claudia R.;
Harrington, Ann T.;
Finkel, Richard S.;
Russman, Barry S.;
Byrne, Barry J.;
Tennekoon, Gihan I.;
Walter, Glenn A.;
Sweeney, H. Lee;
Vandenborne, Krista

Publication type:

Article

Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization.

Published in:

Nature Medicine, 2002, v. 8, n. 6, p. 607, doi. 10.1038/nm0602-607

By:

Grant, Maria B.;
May, W. Stratford;
Caballero, Sergio;
Brown, Gary A. J.;
Guthrie, Steven M.;
Mames, Robert N.;
Byrne, Barry J.;
Vaught, Timothy;
Spoerri, Polyxenie E.;
Peck, Ammon B.;
Scott, Edward W.

Publication type:

Article

Stimulation of Respiratory Motor Output and Ventilation in a Murine Model of Pompe Disease by Ampakines.

Published in:

American Journal of Respiratory Cell & Molecular Biology, 2015, v. 53, n. 3, p. 326, doi. 10.1165/rcmb.2014-0374OC

By:

ElMallah, Mai K.;
Pagliardini, Silvia;
Turner, Sara M.;
Cerreta, Anthony J.;
Falk, Darin J.;
Byrne, Barry J.;
Greer, John J.;
Fuller, David D.

Publication type:

Article

Management of Neuroinflammatory Responses to AAV-Mediated Gene Therapies for Neurodegenerative Diseases.

Published in:

Brain Sciences (2076-3425), 2020, v. 10, n. 2, p. 119, doi. 10.3390/brainsci10020119

By:

Perez, Barbara A.;
Shutterly, Alison;
Chan, Ying Kai;
Byrne, Barry J.;
Corti, Manuela

Publication type:

Article

Oral delivery of Acid Alpha Glucosidase epitopes expressed in plant chloroplasts suppresses antibody formation in treatment of Pompe mice.

Published in:

Plant Biotechnology Journal, 2015, v. 13, n. 8, p. 1023, doi. 10.1111/pbi.12413

By:

Su, Jin;
Sherman, Alexandra;
Doerfler, Phillip A.;
Byrne, Barry J.;
Herzog, Roland W.;
Daniell, Henry

Publication type:

Article

Magnetic Resonance Imaging and Spectroscopy Assessment of Lower Extremity Skeletal Muscles in Boys with Duchenne Muscular Dystrophy: A Multicenter Cross Sectional Study.

Published in:

PLoS ONE, 2014, v. 9, n. 9, p. 1, doi. 10.1371/journal.pone.0106435

By:

Forbes, Sean C.;
Willcocks, Rebecca J.;
Triplett, William T.;
Rooney, William D.;
Lott, Donovan J.;
Wang, Dah-Jyuu;
Pollaro, Jim;
Senesac, Claudia R.;
Daniels, Michael J.;
Finkel, Richard S.;
Russman, Barry S.;
Byrne, Barry J.;
Finanger, Erika L.;
Tennekoon, Gihan I.;
Walter, Glenn A.;
Sweeney, H. Lee;
Vandenborne, Krista

Publication type:

Article

Immune Responses and Hypercoagulation in ERT for Pompe Disease Are Mutation and rhGAA Dose Dependent.

Published in:

PLoS ONE, 2014, v. 9, n. 6, p. 1, doi. 10.1371/journal.pone.0098336

By:

Nayak, Sushrusha;
Doerfler, Phillip A.;
Porvasnik, Stacy L.;
Cloutier, Denise D.;
Khanna, Richie;
Valenzano, Ken J.;
Herzog, Roland W.;
Byrne, Barry J.

Publication type:

Article

Transient B Cell Depletion or Improved Transgene Expression by Codon Optimization Promote Tolerance to Factor VIII in Gene Therapy.

Published in:

PLoS ONE, 2012, v. 7, n. 5, p. 1, doi. 10.1371/journal.pone.0037671

By:

Sack, Brandon K.;
Merchant, Sherin;
Markusic, David M.;
Nathwani, Amit C.;
Davidoff, Andrew M.;
Byrne, Barry J.;
Herzog, Roland W.

Publication type:

Article

The Effect of DNA-Dependent Protein Kinase on Adeno-Associated Virus Replication.

Published in:

PLoS ONE, 2010, v. 5, n. 12, p. 1, doi. 10.1371/journal.pone.0015073

By:

Young-Kook Choi;
Nash, Kevin;
Byrne, Barry J.;
Muzyczka, Nicholas;
Sihong Song

Publication type:

Article

Tolerance Induction to Cytoplasmic β-Galactosidase by Hepatic AAV Gene Transfer - Implications for Antigen Presentation and Immunotoxicity.

Published in:

PLoS ONE, 2009, v. 4, n. 8, p. 1, doi. 10.1371/journal.pone.0006376

By:

Martino, Ashley T.;
Nayak, Sushrusha;
Hoffman, Brad E.;
Cooper, Mario;
Liao, Gongxian;
Markusic, David M.;
Byrne, Barry J.;
Terhorst, Cox;
Herzog, Roland W.

Publication type:

Article

Hypoglossal Motoneuron (XII MN) Survival Over the Lifespan in Pompe Disease.

Published in:

FASEB Journal, 2022, v. 36, p. N.PAG, doi. 10.1096/fasebj.2022.36.S1.R3681

By:

Barral, Brian E.;
Singer, Michele L.;
Rana, Sabhya;
Lavassani, Emmanuelle;
Pope, Megan K.;
Byrne, Barry J.;
Fuller, David D.

Publication type:

Article

Mutation-adapted U1 snRNA corrects a splicing error of the dopa decarboxylase gene.

Published in:

Human Molecular Genetics, 2016, v. 25, n. 23, p. 5142, doi. 10.1093/hmg/ddw323

By:

Ni-Chung Lee;
Yu-May Lee;
Pin-Wen Chen;
Byrne, Barry J.;
Wuh-Liang Hwu

Publication type:

Article

Peripheral nerve and neuromuscular junction pathology in Pompe disease.

Published in:

Human Molecular Genetics, 2015, v. 24, n. 3, p. 625, doi. 10.1093/hmg/ddu476

By:

Falk, Darin J.;
Todd, Adrian Gary;
Lee, Sooyeon;
Soustek, Meghan S.;
ElMallah, Mai K.;
Fuller, David D.;
Notterpek, Lucia;
Byrne, Barry J.

Publication type:

Article

Virus-based gene delivery systems.

Published in:

2002

By:

Mah, C.;
Byrne, B.J.;
Flotte, T.R.;
Mah, Cathryn;
Byrne, Barry J;
Flotte, Terence R

Publication type:

journal article

Corrigendum: Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Published in:

2025

By:

Byrne, Barry J.;
Parenti, Giancarlo;
Schoser, Benedikt;
van der Ploeg, Ans T.;
Do, Hung;
Fox, Brian;
Goldman, Mitchell;
Johnson, Franklin K.;
Kang, Jia;
Mehta, Nickita;
Mondick, John;
Sheikh, M. Osman;
Sitaraman Das, Sheela;
Tuske, Steven;
Brudvig, Jon;
Weimer, Jill M.;
Mozaffar, Tahseen

Publication type:

Correction Notice

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Published in:

Frontiers in Neurology, 2025, p. 1, doi. 10.3389/fneur.2024.1451512

By:

Byrne, Barry J.;
Parenti, Giancarlo;
Schoser, Benedikt;
van der Ploeg, Ans T.;
Do, Hung;
Fox, Brian;
Goldman, Mitchell;
Johnson, Franklin K.;
Kang, Jia;
Mehta, Nickita;
Mondick, John;
Sheikh, M. Osman;
Sitaraman Das, Sheela;
Tuske, Steven;
Brudvig, Jon;
Weimer, Jill M.;
Mozaffar, Tahseen

Publication type:

Article

Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease.

Published in:

Frontiers in Neurology, 2024, p. 1, doi. 10.3389/fneur.2024.1451512

By:

Byrne, Barry J.;
Parenti, Giancarlo;
Schoser, Benedikt;
van der Ploeg, Ans T.;
Hung Do;
Fox, Brian;
Goldman, Mitchell;
Johnson, Franklin K.;
Jia Kang;
Mehta, Nickita;
Mondick, John;
Sheikh, M. Osman;
Das, Sheela Sitaraman;
Tuske, Steven;
Brudvig, Jon;
Weimer, Jill M.;
Mozaffar, Tahseen

Publication type:

Article

Effect of Intracoronary Delivery of Autologous Bone Marrow Mononuclear Cells 2 to 3 Weeks Following Acute Myocardial Infarction on Left Ventricular Function.

Published in:

JAMA: Journal of the American Medical Association, 2011, v. 306, n. 19, p. 2110, doi. 10.1001/jama.2011.1670

By:

Traverse, Jay H.;
Henry, Timothy D.;
Ellis, Stephen G.;
Pepine, Carl J.;
Willerson, James T.;
Zhao, David X. M.;
Forder, John R.;
Byrne, Barry J.;
Hatzopoulos, Antonis K.;
Penn, Marc S.;
Perin, Emerson C.;
Baran, Kenneth W.;
Chambers, Jeffrey;
Lambert, Charles;
Raveendran, Ganesh;
Simon, Daniel I.;
Vaughan, Douglas E.;
Simpson, Lara M.;
Gee, Adrian P.;
Taylor, Doris A.

Publication type:

Article

The Barth Syndrome Registry: Distinguishing disease characteristics and growth data from a longitudinal study.

Published in:

American Journal of Medical Genetics. Part A, 2012, v. 158A, n. 11, p. 2726, doi. 10.1002/ajmg.a.35609

By:

Roberts, Amy E.;
Nixon, Connie;
Steward, Colin G.;
Gauvreau, Kimberly;
Maisenbacher, Melissa;
Fletcher, Matthew;
Geva, Judith;
Byrne, Barry J.;
Spencer, Carolyn T.

Publication type:

Article

Multicenter prospective longitudinal study of magnetic resonance biomarkers in a large duchenne muscular dystrophy cohort.

Published in:

2016

By:

Willcocks, Rebecca J.;
Rooney, William D.;
Triplett, William T.;
Forbes, Sean C.;
Lott, Donovan J.;
Senesac, Claudia R.;
Daniels, Michael J.;
Wang, Dah‐Jyuu;
Harrington, Ann T.;
Tennekoon, Gihan I.;
Russman, Barry S.;
Finanger, Erika L.;
Byrne, Barry J.;
Finkel, Richard S.;
Walter, Glenn A.;
Sweeney, H. Lee;
Vandenborne, Krista;
Wang, Dah-Jyuu

Publication type:

journal article

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy.

Published in:

2016

By:

Mendell, Jerry R.;
Goemans, Nathalie;
Lowes, Linda P.;
Alfano, Lindsay N.;
Berry, Katherine;
Shao, James;
Kaye, Edward M.;
Mercuri, Eugenio;
Hamid, Hoda Abdel;
Byrne, Barry J.;
Connolly, Anne M.;
Dracker, Robert A.;
Matthew Frank, L.;
Heydemann, Peter T.;
O'Brien, Kevin C.;
Sparks, Susan E.;
Specht, Linda A.;
Rodino‐Klapac, Louise;
Sahenk, Zarife;
Al‐Zaidy, Samiah

Publication type:

journal article

Correcting Neuromuscular Deficits With Gene Therapy in Pompe Disease.

Published in:

Annals of Neurology, 2015, v. 78, n. 2, p. 222, doi. 10.1002/ana.24433

By:

Todd, Adrian G.;
McElroy, Jessica A.;
Grange, Robert W.;
Fuller, David D.;
Walter, Glenn A.;
Byrne, Barry J.;
Falk, Darin J.

Publication type:

Article

The Clinical Development of Taldefgrobep Alfa: An Anti-Myostatin Adnectin for the Treatment of Duchenne Muscular Dystrophy.

Published in:

Neurology & Therapy, 2024, v. 13, n. 1, p. 183, doi. 10.1007/s40120-023-00570-w

By:

Muntoni, Francesco;
Byrne, Barry J.;
McMillan, Hugh J.;
Ryan, Monique M.;
Wong, Brenda L.;
Dukart, Juergen;
Bansal, Amita;
Cosson, Valerie;
Dreghici, Roxana;
Guridi, Maitea;
Rabbia, Michael;
Staunton, Hannah;
Tirucherai, Giridhar S.;
Yen, Karl;
Yuan, Xiling;
Wagner, Kathryn R.;
the Taldefgrobep Alfa Study Group;
Carvajal, Irvith;
Chimalakonda, Anjaneya;
Gokemeijer, Jochem

Publication type:

Article

Cough Effectiveness and Pulmonary Hygiene Practices in Patients with Pompe Disease.

Published in:

Lung (0341-2040), 2019, v. 197, n. 1, p. 1, doi. 10.1007/s00408-018-0171-1

By:

Pitts, Teresa;
Bordelon, Rachel;
Huff, Alyssa;
Byrne, Barry J.;
Smith, Barbara K.

Publication type:

Article

A study on the safety and efficacy of reveglucosidase alfa in patients with late-onset Pompe disease.

Published in:

2017

By:

Byrne, Barry J.;
Geberhiwot, Tarekegn;
Barshop, Bruce A.;
Barohn, Richard;
Hughes, Derralynn;
Bratkovic, Drago;
Desnuelle, Claude;
Laforet, Pascal;
Mengel, Eugen;
Roberts, Mark;
Haroldsen, Peter;
Reilley, Kristin;
Jayaram, Kala;
Ke Yang;
Walsh, Liron;
Yang, Ke;
POM-001/002 Investigators

Publication type:

journal article

Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.

Published in:

Annals of Neurology, 2010, v. 68, n. 5, p. 629, doi. 10.1002/ana.22251

By:

Mendell, Jerry R.;
Rodino-Klapac, Louise R.;
Rosales, Xiomara Q.;
Coley, Brian D.;
Galloway, Gloria;
Lewis, Sarah;
Malik, Vinod;
Shilling, Chris;
Byrne, Barry J.;
Conlon, Thomas;
Campbell, Katherine J.;
Bremer, William G.;
Taylor, Laura E.;
Flanigan, Kevin M.;
Gastier-Foster, Julie M.;
Astbury, Caroline;
Kota, Janaiah;
Sahenk, Zarife;
Walker, Christopher M.;
Clark, K. Reed

Publication type:

Article

Limb-girdle muscular dystrophy type 2D gene therapy restores α-sarcoglycan and associated proteins.

Published in:

Annals of Neurology, 2009, v. 66, n. 3, p. 290, doi. 10.1002/ana.21732

By:

Mendell, Jerry R.;
Rodino-Klapac, Louise R.;
Rosales-Quintero, Xiomara;
Kota, Janaiah;
Coley, Brian D.;
Galloway, Gloria;
Craenen, Josepha M.;
Lewis, Sarah;
Malik, Vinod;
Shilling, Christopher;
Byrne, Barry J.;
Conlon, Thomas;
Campbell, Katherine J.;
Bremer, William G.;
Viollet, Laurence;
Walker, Christopher M.;
Sahenk, Zarife;
Clark, K. Reed

Publication type:

Article

Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5.

Published in:

2008

By:

Polyak, Steven;
Mah, Cathryn;
Porvasnik, Stacy;
Herlihy, John-David;
Campbell-Thompson, Martha;
Byrne, Barry J;
Valentine, John F

Publication type:

journal article

Glycogen Storage Disease Type Ia in Canines: A Model for Human Metabolic and Genetic Liver Disease.

Published in:

Journal of Biomedicine & Biotechnology, 2011, p. 1, doi. 10.1155/2011/646257

By:

Specht, Andrew;
Fiske, Laurie;
Erger, Kirsten;
Cossette, Travis;
Verstegen, John;
Campbell-Thompson, Martha;
Struck, Maggie B.;
Lee, Young Mok;
Chou, Janice Y.;
Byrne, Barry J.;
Correia, Catherine E.;
Mah, Cathryn S.;
Weinstein, David A.;
Conlon, Thomas J.

Publication type:

Article

Blunted fat oxidation upon submaximal exercise is partially compensated by enhanced glucose metabolism in children, adolescents, and young adults with Barth syndrome.

Published in:

Journal of Inherited Metabolic Disease, 2019, v. 42, n. 3, p. 480, doi. 10.1002/jimd.12094

By:

Cade, William Todd;
Bohnert, Kathryn L.;
Peterson, Linda R.;
Patterson, Bruce W.;
Bittel, Adam J.;
Okunade, Adewole L.;
de las Fuentes, Lisa;
Steger‐May, Karen;
Bashir, Adil;
Schweitzer, George G.;
Chacko, Shaji K.;
Wanders, Ronald J.;
Pacak, Christina A.;
Byrne, Barry J.;
Reeds, Dominic N.

Publication type:

Article

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.

Published in:

Journal of Inherited Metabolic Disease Reports, 2021, v. 62, n. 1, p. 74, doi. 10.1002/jmd2.12244

By:

Bohnert, Kathryn L.;
Ditzenberger, Grace;
Bittel, Adam J.;
de las Fuentes, Lisa;
Corti, Manuela;
Pacak, Christina A.;
Taylor, Carolyn;
Byrne, Barry J.;
Reeds, Dominic N.;
Cade, W. Todd

Publication type:

Article

Pompe disease gene therapy.

Published in:

Human Molecular Genetics, 2011, v. 20, n. R1, p. R61, doi. 10.1093/hmg/ddr174

By:

Byrne, Barry J.;
Falk, Darin J.;
Pacak, Christina A.;
Nayak, Sushrusha;
Herzog, Roland W.;
Elder, Melissa E.;
Collins, Shelley W.;
Conlon, Thomas J.;
Clement, Nathalie;
Cleaver, Brian D.;
Cloutier, Denise A.;
Porvasnik, Stacy L.;
Islam, Saleem;
Elmallah, Mai K.;
Martin, Anatole;
Smith, Barbara K.;
Fuller, David D.;
Lawson, Lee Ann;
Mah, Cathryn S.

Publication type:

Article

Sustained hepatic and renal glucose-6-phosphatase expression corrects glycogen storage disease type Ia in mice.

Published in:

Human Molecular Genetics, 2002, v. 11, n. 18, p. 2155, doi. 10.1093/hmg/11.18.2155

By:

Sun, Mao-Sen;
Pan, Chi-Jiunn;
Shieh, Jeng-Jer;
Ghosh, Abhijit;
Chen, Li-Yuan;
Mansfield, Brian C.;
Ward, Jerrold M.;
Byrne, Barry J.;
Chou, Janice Yang

Publication type:

Article

Recruitment of human muscleblind proteins to (CUG)<sub>n</sub> expansions associated with myotonic dystrophy.

Published in:

EMBO Journal, 2000, v. 19, n. 17, p. 4439, doi. 10.1093/emboj/19.17.4439

By:

Miller, Jill W.;
Urbinati, Carl R.;
Patana Teng-umnuay;
Stenberg, Myrna G.;
Byrne, Barry J.;
Thornton, Charles A.;
Swanson, Maurice S.

Publication type:

Article

A Neuron-Specific Gene Therapy Relieves Motor Deficits in Pompe Disease Mice.

Published in:

Molecular Neurobiology, 2018, v. 55, n. 6, p. 5299, doi. 10.1007/s12035-017-0763-4

By:

Lee, Ni-Chung;
Hwu, Wuh-Liang;
Muramatsu, Shin-Ichi;
Falk, Darin J.;
Byrne, Barry J.;
Cheng, Chia-Hao;
Shih, Nien-Chu;
Chang, Kai-Ling;
Tsai, Li-Kai;
Chien, Yin-Hsiu

Publication type:

Article

Benefits of Neuronal Preferential Systemic Gene Therapy for Neurotransmitter Deficiency.

Published in:

Molecular Therapy, 2015, v. 23, n. 10, p. 1572, doi. 10.1038/mt.2015.122

By:

Lee, Ni-Chung;
Muramatsu, Shin-Ichi;
Chien, Yin-Hsiu;
Liu, Wen-Shin;
Wang, Wei-Hua;
Cheng, Chia-Hao;
Hu, Meng-Kai;
Chen, Pin-Wen;
Tzen, Kai-Yuan;
Byrne, Barry J;
Hwu, Wuh-Liang

Publication type:

Article

Charting a Clear Path: The ASGCT Standardized Pathways Conference.

Published in:

Molecular Therapy, 2014, v. 22, n. 7, p. 1235, doi. 10.1038/mt.2014.95

By:

Kiem, Hans-Peter;
Baum, Christopher;
Bushman, Frederic D;
Byrne, Barry J;
Carter, Barrie J;
Cavagnaro, Joy;
Malech, Harry L;
Mendell, Jerry R;
Naldini, Luigi M;
Sorrentino, Brian P;
Williams, David A;
Flotte, Terence R

Publication type:

Article

Sustained Correction of Motoneuron Histopathology Following Intramuscular Delivery of AAV in Pompe Mice.

Published in:

Molecular Therapy, 2014, v. 22, n. 4, p. 702, doi. 10.1038/mt.2013.282

By:

ElMallah, Mai K;
Falk, Darin J;
Nayak, Sushrusha;
Federico, Roland A;
Sandhu, Milapjit S;
Poirier, Amy;
Byrne, Barry J;
Fuller, David D

Publication type:

Article

Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease.

Published in:

Molecular Therapy, 2013, v. 21, n. 9, p. 1661, doi. 10.1038/mt.2013.96

By:

Falk, Darin J;
Mah, Cathryn S;
Soustek, Meghan S;
Lee, Kun-Ze;
ElMallah, Mai K;
Cloutier, Denise A;
Fuller, David D;
Byrne, Barry J

Publication type:

Article

Pathway for Approval of a Gene Therapy Orphan Product: Treading New Ground.

Published in:

Molecular Therapy, 2013, v. 21, n. 8, p. 1465, doi. 10.1038/mt.2013.157

By:

Byrne, Barry J

Publication type:

Article

Translating the Genomics Revolution: The Need for an International Gene Therapy Consortium for Monogenic Diseases.

Published in:

Molecular Therapy, 2013, v. 21, n. 2, p. 266, doi. 10.1038/mt.2013.4

By:

Tremblay, Jacques P;
Xiao, Xiao;
Aartsma-Rus, Annemieke;
Barbas, Carlos;
Blau, Helen M;
Bogdanove, Adam J;
Boycott, Kym;
Braun, Serge;
Breakefield, Xandra O;
Bueren, Juan A;
Buschmann, Michael;
Byrne, Barry J;
Calos, Michele;
Cathomen, Toni;
Chamberlain, Jeffrey;
Chuah, Marinee;
Cornetta, Kenneth;
Davies, Kay E;
Dickson, J George;
Duchateau, Philippe

Publication type:

Article

Signs of Progress in Gene Therapy for Muscular Dystrophy Also Warrant Caution.

Published in:

2012

By:

Stedman, Hansell H;
Byrne, Barry J

Publication type:

Opinion

Spinal Delivery of AAV Vector Restores Enzyme Activity and Increases Ventilation in Pompe Mice.

Published in:

Molecular Therapy, 2012, v. 20, n. 1, p. 21, doi. 10.1038/mt.2011.214

By:

Qiu, Kai;
Falk, Darin J;
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Article

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Molecular Therapy, 2011, v. 19, n. 9, p. 1582, doi. 10.1038/mt.2011.124

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Molecular Therapy, 2011, v. 19, n. 2, p. 231, doi. 10.1038/mt.2010.306

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