B-Cell Depletion is Protective Against Anti-AAV Capsid Immune Response: A Human Subject Case Study von 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

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Human mesenchymal stem cells differentiate to a cardiomyocyte phenotype in the adult murine heart von TOMA, Catalin, PITTENGER, Mark F, CAHILL, Kevin S, BYRNE, Barry J, KESSLER, Paul D

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Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease von Doerfler, Phillip A, Todd, Adrian G, Clément, Nathalie, Falk, Darin J, Nayak, Sushrusha, Herzog, Roland W, Byrne, Barry J

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Pompe disease diagnosis and management guideline von 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 von Miller, J W, Urbinati, C R, Teng-Umnuay, P, Stenberg, M G, Byrne, B J, Thornton, C A, Swanson, M S

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

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Recombinant adeno-associated virus purification using novel methods improves infectious titer and yield von ZOLOTUKHIN, S, BYRNE, B. J, MASON, E, ZOLOTUKHIN, I, POTTER, M, CHESNUT, K, SUMMERFORD, C, SAMULSKI, R. J, MUZYCZKA, N

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BAFF blockade prevents anti-drug antibody formation in a mouse model of Pompe disease von Doerfler, Phillip A, Nayak, Sushrusha, Herzog, Roland W, Morel, Laurence, Byrne, Barry J

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Immune responses and hypercoagulation in ERT for Pompe disease are mutation and rhGAA dose dependent von Nayak, Sushrusha, Doerfler, Phillip A, Porvasnik, Stacy L, Cloutier, Denise D, Khanna, Richie, Valenzano, Ken J, Herzog, Roland W, Byrne, Barry J

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Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors von 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

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Targeted approaches to induce immune tolerance for Pompe disease therapy von Doerfler, Phillip A, Nayak, Sushrusha, Corti, Manuela, Morel, Laurence, Herzog, Roland W, Byrne, Barry J

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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 von Flotte, Terence R, Brantly, Mark L, Spencer, L Terry, Byrne, Barry J, Spencer, Carolyn T, Baker, Dawn J, Humphries, Margaret

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Successful Production of Pseudotyped rAAV Vectors Using a Modified Baculovirus Expression System von 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

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Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy von Mah, Cathryn, Fraites, Thomas J., Zolotukhin, Irene, Song, Sihong, Flotte, Terence R., Dobson, Jon, Batich, Christopher, Byrne, Barry J.

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Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein von Kessler, Paul D., Podsakoff, Gregory M., Chen, Xiaojuan, McQuiston, Susan A., Colosi, Peter C., Matelis, Laura A., Kurtzman, Gary J., Byrne, Barry J.

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Sustained Secretion of Human Alpha-1-Antitrypsin from Murine Muscle Transduced with Adeno-Associated Virus Vectors von Song, Sihong, Morgan, Michael, Ellis, Tamir, Poirier, Amy, Chesnut, Kye, Wang, Jianming, Brantly, Mark, Muzyczka, Nicholas, Byrne, Barry J., Atkinson, Mark, Flotte, Terence R.

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CMV-β-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1α promoter and results in therapeut... von LINGFEI XU, DALY, Thomas, CUIHUA GAO, FLOTTE, Terence R, SIHONG SONG, BYRNE, Barry J, SANDS, Mark S, PONDER, Katherine Parker

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Virus-based gene delivery systems von MAH, Cathryn, BYRNE, Barry J, FLOTTE, Terence R

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Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors von MAH, C, CRESAWN, K. O, FRAITES, T. J, PACAK, C. A, LEWIS, M. A, ZOLOTUKHIN, I., BYRNE, B J

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In vivo murine left ventricular pressure-volume relations by miniaturized conductance micromanometry von Georgakopoulos, Dimitrios, Mitzner, Wayne A, Chen, Chen-Huan, Byrne, Barry J, Millar, Huntly D, Hare, Joshua M, Kass, David A

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