Search Results - "Byrne, Bj"

B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study by Corti, M, Elder, Me, Falk, Dj, Lawson, L, Smith, Bk, Nayak, S, Conlon, Tj, Clément, N, Erger, K, Lavassani, E, Green, M, Doerfler, Pa, Herzog, Rw, Byrne, Bj

“…Gene therapy strategies for congenital myopathies may require repeat administration of adeno-associated viral (AAV) vectors in response to several limitations…”
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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease by Doerfler, Phillip A, Todd, Adrian G, Clément, Nathalie, Falk, Darin J, Nayak, Sushrusha, Herzog, Roland W, Byrne, Barry J

“…Pompe disease is a progressive neuromuscular disorder caused by lysosomal accumulation of glycogen from a deficiency in acid alpha-glucosidase (GAA)…”
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Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart by TOMA, Catalin, PITTENGER, Mark F, CAHILL, Kevin S, BYRNE, Barry J, KESSLER, Paul D

“…Cellular cardiomyoplasty has been proposed as an alternative strategy for augmenting the function of diseased myocardium. We investigated the potential of…”
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Pompe disease diagnosis and management guideline by Kishnani, Priya S, Steiner, Robert D, Bali, Deeksha, Berger, Kenneth, Byrne, Barry J, Case, Laura E, Case, Laura, Crowley, John F, Downs, Steven, Howell, R Rodney, Kravitz, Richard M, Mackey, Joanne, Marsden, Deborah, Martins, Anna Maria, Millington, David S, Nicolino, Marc, O'Grady, Gwen, Patterson, Marc C, Rapoport, David M, Slonim, Alfred, Spencer, Carolyn T, Tifft, Cynthia J, Watson, Michael S

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Recruitment of human muscleblind proteins to (CUG)(n) expansions associated with myotonic dystrophy by Miller, J W, Urbinati, C R, Teng-Umnuay, P, Stenberg, M G, Byrne, B J, Thornton, C A, Swanson, M S

“…Myotonic dystrophy (DM1) is an autosomal dominant neuromuscular disorder associated with a (CTG)(n) expansion in the 3'-untranslated region of the DM1 protein…”
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Adult hematopoietic stem cells provide functional hemangioblast activity during retinal neovascularization by Grant, Maria B, Scott, Edward W, 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

“…Adults maintain a reservoir of hematopoietic stem cells that can enter the circulation to reach organs in need of regeneration. We developed a novel model of…”
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BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease by Doerfler, Phillip A, Nayak, Sushrusha, Herzog, Roland W, Morel, Laurence, Byrne, Barry J

“…Abstract Antibodies formed against the therapeutic protein are a life-threatening complication that arises during enzyme replacement therapy for Pompe disease…”
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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield by ZOLOTUKHIN, S, BYRNE, B. J, MASON, E, ZOLOTUKHIN, I, POTTER, M, CHESNUT, K, SUMMERFORD, C, SAMULSKI, R. J, MUZYCZKA, N

“…Conventional methods for rAAV purification that are based on cesium chloride ultracentrifugation have often produced vector preparations of variable quality…”
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Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors by Zolotukhin, Sergei, Potter, Mark, Zolotukhin, Irene, Sakai, Yoshihisa, Loiler, Scott, Fraites, Thomas J, Chiodo, Vince A, Phillipsberg, Tina, Muzyczka, Nicholas, Hauswirth, William W, Flotte, Terance R, Byrne, Barry J, Snyder, Richard O

“…Recombinant adeno-associated viral (rAAV) vectors based on serotype 2 are currently being evaluated most extensively in animals and human clinical trials. rAAV…”
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Targeted approaches to induce immune tolerance for Pompe disease therapy by Doerfler, Phillip A, Nayak, Sushrusha, Corti, Manuela, Morel, Laurence, Herzog, Roland W, Byrne, Barry J

“…Enzyme and gene replacement strategies have developed into viable therapeutic approaches for the treatment of Pompe disease (acid α-glucosidase (GAA)…”
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Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent by Nayak, Sushrusha, Doerfler, Phillip A, Porvasnik, Stacy L, Cloutier, Denise D, Khanna, Richie, Valenzano, Ken J, Herzog, Roland W, Byrne, Barry J

“…Enzyme replacement therapy (ERT) with recombinant human acid-α-glucosidase (rhGAA) is the only FDA approved therapy for Pompe disease. Without ERT, severely…”
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Phase I trial of intramuscular injection of a recombinant adeno-associated virus alpha 1-antitrypsin (rAAV2-CB-hAAT) gene vector to AAT-deficient adults by Flotte, Terence R, Brantly, Mark L, Spencer, L Terry, Byrne, Barry J, Spencer, Carolyn T, Baker, Dawn J, Humphries, Margaret

“…A recombinant virus vector constructed from adeno-associated virus (AAV) that has been altered to carry the human alpha1-antitrypsin (hAAT) gene expressed from…”
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Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System by Kohlbrenner, Erik, Aslanidi, George, Nash, Kevin, Shklyaev, Stanislav, Campbell-Thompson, Martha, Byrne, Barry J, Snyder, Richard O, Muzyczka, Nicholas, Warrington, Jr, Kenneth H, Zolotukhin, Sergei

“…Scalable production of rAAV vectors remains a major obstacle to the clinical application of this prototypical gene therapy vector. A recently developed…”
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Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy by Mah, Cathryn, Fraites, Thomas J., Zolotukhin, Irene, Song, Sihong, Flotte, Terence R., Dobson, Jon, Batich, Christopher, Byrne, Barry J.

“…A major hurdle in most current gene therapy modalities is the ability to transduce target tissues at very high efficiencies that ultimately lead to therapeutic…”
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Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors by Song, Sihong, Morgan, Michael, Ellis, Tamir, Poirier, Amy, Chesnut, Kye, Wang, Jianming, Brantly, Mark, Muzyczka, Nicholas, Byrne, Barry J., Atkinson, Mark, Flotte, Terence R.

“…Recombinant adeno-associated virus (AAV) vectors have been used to transduce murine skeletal muscle as a platform for secretion of therapeutic proteins. The…”
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Virus-based gene delivery systems by MAH, Cathryn, BYRNE, Barry J, FLOTTE, Terence R

“…Within the past decade, gene therapy strategies have come to the forefront of novel therapeutics. Tremendous advances in vector technology along with deeper…”
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Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein by Kessler, Paul D., Podsakoff, Gregory M., Chen, Xiaojuan, McQuiston, Susan A., Colosi, Peter C., Matelis, Laura A., Kurtzman, Gary J., Byrne, Barry J.

“…Somatic gene therapy has been proposed as a means to achieve systemic delivery of therapeutic proteins. However, there is limited evidence that current methods…”
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Long-term correction of murine glycogen storage disease type Ia by recombinant adeno-associated virus-1-mediated gene transfer by Ghosh, A, Allamarvdasht, M, Pan, C-J, Sun, M-S, Mansfield, B C, Byrne, B J, Chou, J Y

“…Glycogen storage disease type Ia (GSD-Ia) is caused by a deficiency in glucose-6-phosphatase-alpha (G6Pase-alpha), a nine-transmembrane domain, endoplasmic…”
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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 by Xu, L, Daly, T, Gao, C, Flotte, T R, Song, S, Byrne, B J, Sands, M S, Parker Ponder, K

“…Although AAV vectors show promise for hepatic gene therapy, the optimal transcriptional regulatory elements have not yet been identified. In this study, we…”
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Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors by MAH, C, CRESAWN, K. O, FRAITES, T. J, PACAK, C. A, LEWIS, M. A, ZOLOTUKHIN, I., BYRNE, B J

“…Glycogen storage disease type II (GSDII) is caused by a lack of functional lysosomal acid alpha-glucosidase (GAA). Affected individuals store glycogen in…”
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