Mah, Cathryn S.

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

Zhong, L., Li, B., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Cooper, M., Herzog, R. W., Zolotukhin, I., Warrington, K. H., Weigel-Van Aken, K. A., et al. (2008) · Proceedings of the National Academy of Sciences

Virus-based gene therapy researchRNA Interference and Gene Delivery

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

Qing, K., Mah, C., Hansen, J., Zhou, S., Dwarki, V., & Srivastava, A. (1999) · Nature Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

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

Pacak, C. A., Mah, C. S., Thattaliyath, B. D., Conlon, T. J., Lewis, M. A., Cloutier, D. E., Zolotukhin, I., Tarantal, A. F., & Byrne, B. J. (2006) · Circulation Research

Virus-based gene therapy researchRNA Interference and Gene Delivery

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

Zhong, L., Li, B., Jayandharan, G., Mah, C. S., Govindasamy, L., Agbandje-McKenna, M., Herzog, R. W., Weigel-Van Aken, K. A., Hobbs, J. A., Zolotukhin, S., et al. (2008) · Virology

Virus-based gene therapy researchRNA Interference and Gene Delivery

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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

Smith, B. K., Collins, S. W., Conlon, T. J., Mah, C. S., Lawson, L. A., Martin, A. D., Fuller, D. D., Cleaver, B. D., Clément, N., Phillips, D., et al. (2013) · Human Gene Therapy

Lysosomal Storage Disorders ResearchVirus-based gene therapy research

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Pompe disease gene therapy

Byrne, B. J., Falk, D. J., Pacak, C. A., Nayak, S., Herzog, R. W., Elder, M. E., Collins, S. W., Conlon, T. J., Clement, N., Cleaver, B. D., et al. (2011) · Human Molecular Genetics

Lysosomal Storage Disorders ResearchGlycogen Storage Diseases and Myoclonus

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