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Livres
Articles (33)
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Targeting murine small bowel and colon through selective superior mesenteric artery injection

Stacy L. Porvasnik, Cathryn Mah, Steven Polyak

Journal:

Microsurgery

Année:

2010

Langue:

english

Fichier:

PDF, 349 KB

english, 2010

Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle

Christina A Pacak, Thomas Conlon, Cathryn S Mah, Barry J Byrne

Journal:

Genetic Vaccines and Therapy

Année:

2008

Langue:

english

Fichier:

PDF, 459 KB

english, 2008

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

Journal:

Genetic Vaccines and Therapy

Année:

2008

Langue:

english

Fichier:

PDF, 483 KB

english, 2008

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

Journal:

Genetic Vaccines and Therapy

Année:

2009

Langue:

english

Fichier:

PDF, 77 KB

english, 2009

Gene Delivery to Intestinal Epithelial Cells In

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

Journal:

Digestive Diseases and Sciences

Année:

2008

Langue:

english

Fichier:

PDF, 588 KB

english, 2008

10.1038/4758

Srivastava, Arun, Qing, Keyun, Mah, Cathryn, Hansen, Jonathan, Zhou, Shangzhen, Dwarki, Varavani

Journal:

Nature Medicine

Année:

1999

Langue:

english

Fichier:

PDF, 227 KB

english, 1999

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, Mavis Agbandje-McKenna, Roland W. Herzog, Kirsten A. Weigel-Van Aken, Jacqueline A. Hobbs, Sergei Zolotukhin, N

Journal:

Virology

Année:

2008

Langue:

english

Fichier:

PDF, 875 KB

english, 2008

Next generation treatment of Pompe disease using systemic gene transfer with AAV9

Darin Falk, Meghan S. Soustek, Cathryn S. Mah, Denise A. Cloutier, Sean A. Germain, Jeffry S. Kelley, Kirsten E. Erger, Thomas J. Conlon, Nathalie Clement, Barry J. Byrne

Journal:

Molecular Genetics and Metabolism

Année:

2011

Langue:

english

Fichier:

PDF, 60 KB

english, 2011

Phase I/II Trial of Adeno-associated Virus Acid-alpha-Glucosidase (AAV-GAA) Diaphragm Gene Therapy for Ventilatory Failure in Pompe Disease

Barry Byrne, Barbara Smith, Anatole Martin, Cathryn Mah, Lee Ann Lawson, Saleem Islam, Thomas Conlon, Dawn Phillips, Shelley Collins

Journal:

Molecular Genetics and Metabolism

Année:

2012

Langue:

english

Fichier:

PDF, 55 KB

english, 2012

598 Efficient Gene Delivery and Sustained Gene Expression in the Intestinal Epithelium of the Small Bowel and Colon

Steven F. Polyak, Andres Acosta, Stacy L. Porvasnik, Thomas Conlon, Annette Mach, John F. Valentine, Martha Campbell-Thompson, Cathryn Mah

Journal:

Gastroenterology

Année:

2008

Langue:

english

Fichier:

PDF, 67 KB

english, 2008

Virus-Based Gene Delivery Systems

Cathryn Mah, Barry J. Byrne, Dr Terence R. Flotte

Journal:

Clinical Pharmacokinetics

Année:

2002

Langue:

english

Fichier:

PDF, 174 KB

english, 2002

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,

Journal:

Human Gene Therapy

Année:

2013

Langue:

english

Fichier:

PDF, 530 KB

english, 2013

Gene Therapy Approaches for Lysosomal Storage Disease: Next-Generation Treatment

Byrne, Barry J., Falk, Darin J., Clément, Nathalie, Mah, Cathryn S.

Journal:

Human Gene Therapy

Année:

2012

Langue:

english

Fichier:

PDF, 240 KB

english, 2012

[Laboratory Techniques in Biochemistry and Molecular Biology] Adeno-Associated Viral Vectors for Gene Therapy Volume 31 || Gene therapy for hemophilia

Mah, Cathryn

Année:

2005

Langue:

english

Fichier:

PDF, 203 KB

english, 2005

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

Journal:

Molecular Therapy

Année:

2013

Langue:

english

Fichier:

PDF, 634 KB

english, 2013

One year outcomes of diaphragm gene therapy in ventilator dependent children with Pompe disease

Byrne, Barry, Smith, Barbara, Lawson, Lee Ann, Martin, Daniel, Mah, Cathryn, Conlon, Thomas, Phillips, Dawn, Collins, Shelley

Journal:

Molecular Genetics and Metabolism

Année:

2013

Langue:

english

Fichier:

PDF, 62 KB

english, 2013

T1769 Mucosal Cytokine Levels Normalize Despite Continued Clinical and Histopathologic Evidence of Chronic Colitis in a Murine DSS Model of Colitis

Polyak, Steven, Dixon, Lisa R., Wasserfall, Clive, Mach, Annette, Mah, Cathryn, Kevil, Chris G., Valentine, John F.

Journal:

Gastroenterology

Année:

2009

Langue:

english

Fichier:

PDF, 96 KB

english, 2009

W1344 Intestinal Directed Adeno-Associated Virus Expressing IL-10 Improves Enterocolitis in a Murine Model of Inflammatory Bowel Disease

Polyak, Steven, Porvasnik, Stacy L., Conlon, Thomas, Mach, Annette, Wasserfall, Clive, Dixon, Lisa R., Mah, Cathryn

Journal:

Gastroenterology

Année:

2010

Langue:

english

Fichier:

PDF, 72 KB

english, 2010

Impact of Humoral Immune Response on Distribution and Efficacy of Recombinant Adeno-Associated Virus-Derived Acid α-Glucosidase in a Model of Glycogen Storage Disease Type II

Cresawn, Kerry O., Fraites, Thomas J., Wasserfall, Clive, Atkinson, Mark, Lewis, Melissa, Porvasnik, Stacy, Liu, Chen, Mah, Cathryn, Byrne, Barry J.

Journal:

Human Gene Therapy

Année:

2005

Langue:

english

Fichier:

PDF, 425 KB

english, 2005

Characterization of a Transgenic Short Hairpin RNA-Induced Murine Model of Tafazzin Deficiency

Soustek, Meghan S., Falk, Darin J., Mah, Cathryn S., Toth, Matthew J., Schlame, Michael, Lewin, Alfred S., Byrne, Barry J.

Journal:

Human Gene Therapy

Année:

2011

Langue:

english

Fichier:

PDF, 436 KB

english, 2011

Dual Vectors Expressing Murine Factor VIII Result in Sustained Correction of Hemophilia A Mice

Mah, Cathryn, Sarkar, Rita, Zolotukhin, Irene, Schleissing, Mary, Xiao, Xiao, Kazazian, Haig H., Byrne, Barry J.

Journal:

Human Gene Therapy

Année:

2003

Langue:

english

Fichier:

PDF, 389 KB

english, 2003

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.

Journal:

Human Gene Therapy

Année:

2013

Langue:

english

Fichier:

PDF, 189 KB

english, 2013

892. Neural Deficits Contribute to Respiratory Insufficiency in Pompe Disease: A Therapeutic Approach with AAV1

DeRuisseau, Lara R., Mah, Cathryn, Fuller, David D., Byrne, Barry J.

Journal:

Molecular Therapy

Année:

2006

Langue:

english

Fichier:

PDF, 83 KB

english, 2006

28. rAAV2/9 Mediated Gene Delivery of Acid α-Glucosidase Corrects the Cardiac Phenotype in a Mouse Model of Pompe Disease

Pacak, Christina A., Mah, Cathryn, Cresawn, Kerry O., Lewis, Melissa A., Germain, Sean, Byrne, Barry J.

Journal:

Molecular Therapy

Année:

2006

Langue:

english

Fichier:

PDF, 91 KB

english, 2006

575. Correction of Ventilation in Glycogen Storage Disease Type II Mice after Gel-Mediated Delivery of Adeno-Associated Virus Serotype 1 Vectors

Mah, Cathryn, DeRuisseau, Lara R., Pacak, Christina A., Lewis, Melissa A., Fuller, David D., Byrne, Barry J.

Journal:

Molecular Therapy

Année:

2006

Langue:

english

Fichier:

PDF, 135 KB

english, 2006

410. Physiological Correction of Glycogen Storage Disease Type II Using Adeno-Associated Virus Serotype 1 Vectors

Mah, Cathryn, Pacak, Christina A., Cresawn, Kerry O., DeRuisseau, Lara R., Germain, Sean, Lewis, Melissa A., Fuller, David D., Byrne, Barry J.

Journal:

Molecular Therapy

Année:

2006

Langue:

english

Fichier:

PDF, 82 KB

english, 2006

Phase I/II Trial of Diaphragm Delivery of Recombinant Adeno-Associated Virus Acid Alpha-Glucosidase (rAAV1-CMV- GAA ) Gene Vector in Patients with Pompe Disease

Byrne, Barry J., Smith, Barbara, Mah, Cathryn, Lawson, Lee Ann, Islam, Saleem, Corti, Manuela, Martin, Daniel, Dobija, Nicole, Irwin, Maria V., Conlon, Thomas, Cleaver, Brian, Clement, Nathalie, Colli

Journal:

Human Gene Therapy Clinical Development

Année:

2014

Langue:

english

Fichier:

PDF, 1011 KB

english, 2014

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

Journal:

Molecular Therapy

Année:

2007

Langue:

english

Fichier:

PDF, 688 KB

english, 2007

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

Journal:

Molecular Therapy

Année:

2007

Langue:

english

Fichier:

PDF, 241 KB

english, 2007

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,

Journal:

Molecular Therapy

Année:

2010

Langue:

english

Fichier:

PDF, 419 KB

english, 2010

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.R., Rucker Henninger, Mary B., Erger, Kirsten E., Jorgensen, Marda L., Lee, Charles C., Tarantal, Alice F, Byrne, Barry J.

Journal:

Human Gene Therapy Clinical Development

Année:

2016

Langue:

english

Fichier:

PDF, 3.96 MB

english, 2016

401. AAV Therapy Attenuates Respiratory Dysfunction and Glycogen Accumulation in a Murine Model of Glycogen Storage Disease Type II

Falk, Darin J., Soustek, Meghan S., Gary Todd, A., Mah, Cathryn S., Cloutier, Denise A., Clement, Nathalie, Fuller, David D., Byrne, Barry J.

Journal:

Molecular Therapy

Année:

2015

Langue:

english

Fichier:

PDF, 306 KB

english, 2015

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

Journal:

Molecular Therapy — Methods & Clinical Development

Année:

2015

Langue:

english

Fichier:

PDF, 1.47 MB

english, 2015