Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses
Zhong, Li, Li, Baozheng, Mah, Cathryn S, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Cooper, Mario, Herzog, Roland W, Zolotukhin, Irene, Warrington, Kenneth H. Jr, Weigel-Van Aken, Kirsten A, Hobbs, Jacqueline A, Zolotukhin, Sergei, Muzyczka, Nicholas, Srivastava, Arun
Published in Proceedings of the National Academy of Sciences - PNAS (03.06.2008)
Published in Proceedings of the National Academy of Sciences - PNAS (03.06.2008)
Get full text
Journal Article
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
Smith, Barbara K, Collins, Shelley W, Conlon, Thomas J, Mah, Cathryn S, Lawson, Lee Ann, Martin, Anatole D, Fuller, David D, Cleaver, Brian D, Clément, Nathalie, Phillips, Dawn, Islam, Saleem, Dobjia, Nicole, Byrne, Barry J
Published in Human gene therapy (01.06.2013)
Published in Human gene therapy (01.06.2013)
Get more information
Journal Article
Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo
Pacak, Christina A, Mah, Cathryn S, Thattaliyath, Bijoy D, Conlon, Thomas J, Lewis, Melissa A, Cloutier, Denise E, Zolotukhin, Irene, Tarantal, Alice F, Byrne, Barry J
Published in Circulation research (18.08.2006)
Published in Circulation research (18.08.2006)
Get full text
Journal Article
Intrapleural Administration of AAV9 Improves Neural and Cardiorespiratory Function in Pompe Disease
Falk, Darin J, Mah, Cathryn S, Soustek, Meghan S, Lee, Kun-Ze, ElMallah, Mai K, Cloutier, Denise A, Fuller, David D, Byrne, Barry J
Published in Molecular therapy (01.09.2013)
Published in Molecular therapy (01.09.2013)
Get full text
Journal Article
Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression
Zhong, Li, Li, Baozheng, Jayandharan, Giridhararao, Mah, Cathryn S, Govindasamy, Lakshmanan, Agbandje-McKenna, Mavis, Herzog, Roland W, Weigel-Van Aken, Kirsten A, Hobbs, Jacqueline A, Zolotukhin, Sergei, Muzyczka, Nicholas, Srivastava, Arun
Published in Virology (New York, N.Y.) (25.11.2008)
Published in Virology (New York, N.Y.) (25.11.2008)
Get full text
Journal Article
Neural deficits contribute to respiratory insufficiency in Pompe disease
DeRuisseau, Lara R, Fuller, David D, Qiu, Kai, DeRuisseau, Keith C, Donnelly, William H. Jr, Mah, Cathryn, Reier, Paul J, Byrne, Barry J
Published in Proceedings of the National Academy of Sciences - PNAS (09.06.2009)
Published in Proceedings of the National Academy of Sciences - PNAS (09.06.2009)
Get full text
Journal Article
Gene therapy approaches for lysosomal storage disease: next-generation treatment
Byrne, Barry J, Falk, Darin J, Clément, Nathalie, Mah, Cathryn S
Published in Human gene therapy (01.08.2012)
Published in Human gene therapy (01.08.2012)
Get more information
Journal Article
Pompe disease gene therapy: Gene Therapy
BYRNE, Barry J, FALK, Darin J, 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, PACAK, Christina A, NAYAK, Sushrusha, HERZOG, Roland W, ELDER, Melissa E, COLLINS, Shelley W, CONLON, Thomas J, CLEMENT, Nathalie, CLEAVER, Brian D
Published in Human molecular genetics (15.04.2011)
Published in Human molecular genetics (15.04.2011)
Get full text
Journal Article
Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2
Srivastava, Arun, Qing, Keyun, Mah, Cathryn, Hansen, Jonathan, Zhou, Shangzhen, Dwarki, Varavani
Published in Nature medicine (01.01.1999)
Published in Nature medicine (01.01.1999)
Get full text
Journal Article
Gel-mediated Delivery of AAV1 Vectors Corrects Ventilatory Function in Pompe Mice With Established Disease
Mah, Cathryn S, Falk, Darin J, Germain, Sean A, Kelley, Jeffry S, Lewis, Melissa A, Cloutier, Denise A, DeRuisseau, Lara R, Conlon, Thomas J, Cresawn, Kerry O, Fraites Jr, Thomas J, Campbell-Thompson, Martha, Fuller, David D, Byrne, Barry J
Published in Molecular therapy (01.03.2010)
Published in Molecular therapy (01.03.2010)
Get full text
Journal Article
Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice
Falk, Darin J, Soustek, Meghan S, Todd, Adrian Gary, Mah, Cathryn S, Cloutier, Denise A, Kelley, Jeffry S, Clement, Nathalie, Fuller, David D, Byrne, Barry J
Published in Molecular therapy. Methods & clinical development (01.01.2015)
Published in Molecular therapy. Methods & clinical development (01.01.2015)
Get full text
Journal Article
Adeno-associated virus-mediated gene therapy for metabolic myopathy
Mah, Cathryn S, Soustek, Meghan S, Todd, A Gary, McCall, Angela, Smith, Barbara K, Corti, Manuela, Falk, Darin J, Byrne, Barry J
Published in Human gene therapy (01.11.2013)
Published in Human gene therapy (01.11.2013)
Get more information
Journal Article
Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia
Weinstein, David A, Correia, Catherine E, Conlon, Thomas, Specht, Andrew, Verstegen, John, Onclin-Verstegen, Karine, Campbell-Thompson, Martha, Dhaliwal, Gurmeet, Mirian, Layla, Cossette, Holly, Falk, Darin J, Germain, Sean, Clement, Nathalie, Porvasnik, Stacy, Fiske, Laurie, Struck, Maggie, Ramirez, Harvey E, Jordan, Juan, Andrutis, Karl, Chou, Janice Y, Byrne, Barry J, Mah, Cathryn S
Published in Human gene therapy (01.07.2010)
Published in Human gene therapy (01.07.2010)
Get more information
Journal Article
Long-term Skeletal Muscle Protection After Gene Transfer in a Mouse Model of LGMD-2D
Pacak, Christina A, Walter, Glenn A, Gaidosh, Gabe, Bryant, Nathan, Lewis, Melissa A, Germain, Sean, Mah, Cathryn S, Campbell, Kevin P, Byrne, Barry J
Published in Molecular therapy (01.10.2007)
Published in Molecular therapy (01.10.2007)
Get full text
Journal Article
Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice
Pacak, Christina A, Sakai, Yoshihisa, Thattaliyath, Bijoy D, Mah, Cathryn S, Byrne, Barry J
Published in Genetic vaccines and therapy (23.09.2008)
Published in Genetic vaccines and therapy (23.09.2008)
Get full text
Journal Article
Physiological Correction of Pompe Disease by Systemic Delivery of Adeno-associated Virus Serotype 1 Vectors
Mah, Cathryn, Pacak, Christina A, Cresawn, Kerry O, DeRuisseau, Lara R, Germain, Sean, Lewis, Melissa A, Cloutier, Denise A, Fuller, David D, Byrne, Barry J
Published in Molecular therapy (01.03.2007)
Published in Molecular therapy (01.03.2007)
Get full text
Journal Article
Improved Method of Recombinant AAV2 Delivery for Systemic Targeted Gene Therapy
Mah, Cathryn, Fraites, Thomas J., Zolotukhin, Irene, Song, Sihong, Flotte, Terence R., Dobson, Jon, Batich, Christopher, Byrne, Barry J.
Published in Molecular therapy (01.07.2002)
Published in Molecular therapy (01.07.2002)
Get full text
Journal Article
Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors
Conlon, Thomas J, Mah, Cathryn S, Pacak, Christina A, Rucker Henninger, Mary B, Erger, Kirsten E, Jorgensen, Marda L, Lee, C Chang I, Tarantal, Alice F, Byrne, Barry J
Published in Human gene therapy. Clinical development (01.12.2016)
Published in Human gene therapy. Clinical development (01.12.2016)
Get more information
Journal Article