C. Mah

发表

Improved method of recombinant AAV2 delivery for systemic targeted gene therapy.

Jon Dobson, Sihong Song, Terence R Flotte, 2002, Molecular therapy : the journal of the American Society of Gene Therapy.

Adeno-Associated Virus Type 2-Mediated Gene Transfer: Role of Epidermal Growth Factor Receptor Protein Tyrosine Kinase in Transgene Expression

Arun Srivastava, S. Ponnazhagan, D. Kube, 1998, Journal of Virology.

Impaired Intracellular Trafficking of Adeno-Associated Virus Type 2 Vectors Limits Efficient Transduction of Murine Fibroblasts

Arun Srivastava, J. Hansen, A. Srivastava, 2000, Journal of Virology.

Long-term skeletal muscle protection after gene transfer in a mouse model of LGMD-2D.

B. Byrne, K. Campbell, G. Walter, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Sustained FVIII expression and phenotypic correction of hemophilia A in neonatal mice using an endothelial-targeted sleeping beauty transposon.

C. Mah, B. Fletcher, Li Liu, 2006, Molecular therapy : the journal of the American Society of Gene Therapy.

Gene therapy for hemophilia

C. Mah, 2005 .

A new method for recombinant adeno-associated virus vector delivery to murine diaphragm.

B. Byrne, C. Mah, T. J. Fraites, 2004, Molecular therapy : the journal of the American Society of Gene Therapy.

Virus-Based Gene Delivery Systems

T. Flotte, B. Byrne, C. Mah, 2002, Clinical pharmacokinetics.

Adeno-associated virus type 2-mediated transduction in primary human bone marrow-derived CD34+ hematopoietic progenitor cells: donor variation and correlation of transgene expression with cellular differentiation

S. Ponnazhagan, D. Kube, K. Qing, 1997, Journal of virology.

Relative persistence of AAV serotype 1 vector genomes in dystrophic muscle

T. Conlon, B. Byrne, C. Mah, 2008, Genetic vaccines and therapy.

Gene therapy for cardiovascular applications

B. Byrne, C. Mah, C. A. Pacak, 2005 .

Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice

B. Byrne, D. Fuller, N. Clément, 2015, Molecular Therapy: Methods & Clinical Development.

Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors

B. Byrne, C. Mah, T. J. Fraites, 2005, Gene Therapy.

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors.

B. Byrne, D. Fuller, L. DeRuisseau, 2007, Molecular therapy : the journal of the American Society of Gene Therapy.

Adeno-associated virus-mediated gene therapy for metabolic myopathy.

B. Byrne, Angela L McCall, C. Mah, 2013, Human gene therapy.

Gel-mediated delivery of AAV1 vectors corrects ventilatory function in Pompe mice with established disease.

T. Conlon, B. Byrne, D. Fuller, 2010, Molecular therapy : the journal of the American Society of Gene Therapy.

Dual vectors expressing murine factor VIII result in sustained correction of hemophilia A mice.

B. Byrne, H. Kazazian, R. Sarkar, 2003, Human gene therapy.

Characterization of a transgenic short hairpin RNA-induced murine model of Tafazzin deficiency.

B. Byrne, C. Mah, M. Schlame, 2011, Human gene therapy.

Gene Delivery to Intestinal Epithelial Cells In vitro and In vivo with Recombinant Adeno-Associated Virus Types 1, 2 and 5

B. Byrne, J. Valentine, C. Mah, 2008, Digestive Diseases and Sciences.

Intrapleural administration of AAV9 improves neural and cardiorespiratory function in Pompe disease.

B. Byrne, D. Fuller, M. ElMallah, 2013, Molecular therapy : the journal of the American Society of Gene Therapy.

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.

B. Byrne, M. Atkinson, C. Mah, 2005, Human gene therapy.

Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors.

T. Conlon, B. Byrne, C. Mah, 2016, Human gene therapy. Clinical development.

Muscle as a Metabolic Factory for Gene Therapy

B. Byrne, C. Mah, C. A. Pacak, 2010 .

Indoleamine 2,3-dioxygenase attenuates inhibitor development in gene-therapy-treated hemophilia A mice

C. Mah, B. Fletcher, H. Liu, 2009, Gene Therapy.

Central nervous system pathology contributes to respiratory deficits in Glycogen Storage Disease Type II (GSDII)

L. DeRuisseau, C. Mah, D. Fuller, 2006 .

Gene Therapy for Inherited Metabolic Storage Diseases

C. Mah, 2010 .

Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis.

T. Conlon, C. Mah, I. Zolotukhin, 2012, American journal of physiology. Gastrointestinal and liver physiology.

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.

T. Conlon, B. Byrne, D. Fuller, 2013, Human gene therapy.

Recombinant Adeno-Associated Virus Serotype 9 Leads to Preferential Cardiac Transduction In Vivo

T. Conlon, B. Byrne, C. Mah, 2006, Circulation research.

GUT MICROBIOTA AND INFLAMMATORY BOWEL DISEASE TREATMENT

H. Sokol, N. Benech, C. Mah, 2021 .

Pompe disease gene therapy.

T. Conlon, B. Byrne, D. Fuller, 2011, Human molecular genetics.

Neural deficits contribute to respiratory insufficiency in Pompe disease

B. Byrne, P. Reier, D. Fuller, 2009, Proceedings of the National Academy of Sciences.

Adeno-associated virus-mediated correction of a canine model of glycogen storage disease type Ia.

T. Conlon, B. Byrne, N. Clément, 2010, Human gene therapy.

Next generation of adeno-associated virus 2 vectors: Point mutations in tyrosines lead to high-efficiency transduction at lower doses

N. Muzyczka, L. Govindasamy, M. Agbandje-McKenna, 2008, Proceedings of the National Academy of Sciences.

Gene therapy approaches for lysosomal storage disease: next-generation treatment.

B. Byrne, N. Clément, C. Mah, 2012, Human gene therapy.

Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2

Keyun Qing, Arun Srivastava, J. Hansen, 1999, Nature Medicine.

Adeno-Associated Virus Type 2-Mediated Gene Transfer: Correlation of Tyrosine Phosphorylation of the Cellular Single-Stranded D Sequence-Binding Protein with Transgene Expression in Human Cells In Vitro and Murine Tissues In Vivo

S. Ponnazhagan, D. Kube, V. Dwarki, 1998, Journal of Virology.

Gene transfer with adeno-associated virus 2 vectors: the growth factor receptor connection

J. Hansen, A. Srivastava, K. Qing, 1999 .

Glycogen Storage Disease Type Ia in Canines: A Model for Human Metabolic and Genetic Liver Disease

T. Conlon, B. Byrne, J. Chou, 2011, Journal of biomedicine & biotechnology.

Tissue specific promoters improve specificity of AAV9 mediated transgene expression following intra-vascular gene delivery in neonatal mice

B. Byrne, C. Mah, C. A. Pacak, 2008, Genetic vaccines and therapy.

Tyrosine-phosphorylation of AAV2 vectors and its consequences on viral intracellular trafficking and transgene expression.

N. Muzyczka, L. Govindasamy, M. Agbandje-McKenna, 2008, Virology.

Targeting murine small bowel and colon through selective superior mesenteric artery injection

C. Mah, S. Porvasnik, S. Polyak, 2010, Microsurgery.

Induction of tolerance to factor VIII by transient co‐administration with rapamycin

B. Sack, R. Herzog, C. Mah, 2011, Journal of thrombosis and haemostasis : JTH.

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

B. Byrne, D. Fuller, L. DeRuisseau, 2006 .

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

B. Byrne, C. Mah, C. A. Pacak, 2006 .