Publications by authors named "Cathryn Mah"

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Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.
Thomas J Conlon, Cathryn S Mah, Christina A Pacak, Mary B Rucker Henninger, Kirsten E Erger

Hum Gene Ther Clin Dev· December 2016

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Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice.
Darin J Falk, Meghan S Soustek, Adrian Gary Todd, Cathryn S Mah, Denise A Cloutier

Mol Ther Methods Clin Dev· June 2015

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Adeno-associated virus-mediated gene therapy for metabolic myopathy.
Cathryn S Mah, Meghan S Soustek, A Gary Todd, Angela McCall, Barbara K Smith

Hum Gene Ther· November 2013

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Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.
Darin J Falk, Cathryn S Mah, Meghan S Soustek, Kun-Ze Lee, Mai K Elmallah

Mol Ther· September 2013

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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.
Barbara K Smith, Shelley W Collins, Thomas J Conlon, Cathryn S Mah, Lee Ann Lawson

Hum Gene Ther· June 2013

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Gene therapy approaches for lysosomal storage disease: next-generation treatment.
Barry J Byrne, Darin J Falk, Nathalie Clément, Cathryn S Mah

Hum Gene Ther· August 2012

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Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis.
Steven Polyak, Annette Mach, Stacy Porvasnik, Lisa Dixon, Thomas Conlon, Cathryn Mah

Am J Physiol Gastrointest Liver Physiol· February 2012

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Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency.
Meghan S Soustek, Darin J Falk, Cathryn S Mah, Matthew J Toth, Michael Schlame

Hum Gene Ther· July 2011

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Glycogen storage disease type Ia in canines: a model for human metabolic and genetic liver disease.
Andrew Specht, Laurie Fiske, Kirsten Erger, Travis Cossette, John Verstegen, Cathryn S Mah

J Biomed Biotechnol· June 2011

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Pompe disease gene therapy.
Barry J Byrne, Darin J Falk, Christina A Pacak, Sushrusha Nayak, Roland W Herzog, Cathryn S Mah

Hum Mol Genet· April 2011

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Targeting murine small bowel and colon through selective superior mesenteric artery injection.
Stacy L Porvasnik, Cathryn Mah, Steven Polyak

Microsurgery· September 2010

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Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.
David A Weinstein, Catherine E Correia, Thomas Conlon, Andrew Specht, John Verstegen, Cathryn S Mah

Hum Gene Ther· July 2010

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Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease.
Cathryn S Mah, Darin J Falk, Sean A Germain, Jeffry S Kelley, Melissa A Lewis

Mol Ther· March 2010

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Neural deficits contribute to respiratory insufficiency in Pompe disease.
Lara R DeRuisseau, David D Fuller, Kai Qiu, Keith C DeRuisseau, William H Donnelly, Cathryn Mah

Proc Natl Acad Sci U S A· June 2009

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Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression.
Li Zhong, Baozheng Li, Giridhararao Jayandharan, Cathryn S Mah, Lakshmanan Govindasamy

Virology· November 2008

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Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle.
Christina A Pacak, Thomas Conlon, Cathryn S Mah, Barry J Byrne

Genet Vaccines Ther· October 2008

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Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice.
Christina A Pacak, Yoshihisa Sakai, Bijoy D Thattaliyath, Cathryn S Mah, Barry J Byrne

Genet Vaccines Ther· September 2008

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Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses.
Li Zhong, Baozheng Li, Cathryn S Mah, Lakshmanan Govindasamy, Mavis Agbandje-McKenna

Proc Natl Acad Sci U S A· June 2008

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Gene delivery to intestinal epithelial cells in vitro and in vivo with recombinant adeno-associated virus types 1, 2 and 5.
Steven Polyak, Cathryn Mah, Stacy Porvasnik, John-David Herlihy, Martha Campbell-Thompson

Dig Dis Sci· May 2008

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Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D.
Christina A Pacak, Glenn A Walter, Gabe Gaidosh, Nathan Bryant, Melissa A Lewis, Cathryn S Mah

Mol Ther· October 2007

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Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.
Cathryn Mah, Christina A Pacak, Kerry O Cresawn, Lara R Deruisseau, Sean Germain

Mol Ther· March 2007

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Recombinant adeno-associated virus serotype 9 leads to preferential cardiac transduction in vivo.
Christina A Pacak, Cathryn S Mah, Bijoy D Thattaliyath, Thomas J Conlon, Melissa A Lewis

Circ Res· August 2006

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Sustained FVIII expression and phenotypic correction of hemophilia A in neonatal mice using an endothelial-targeted sleeping beauty transposon.
Li Liu, Cathryn Mah, Bradley S Fletcher

Mol Ther· May 2006

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Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.
Kerry O Cresawn, Thomas J Fraites, Clive Wasserfall, Mark Atkinson, Melissa Lewis, Cathryn Mah

Hum Gene Ther· January 2005

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A new method for recombinant adeno-associated virus vector delivery to murine diaphragm.
Cathryn Mah, Thomas J Fraites, Kerry O Cresawn, Irene Zolotukhin, Melissa A Lewis

Mol Ther· March 2004

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