Ragni, Margaret V.

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

Manno, C. S., Pierce, G. F., Arruda, V. R., Glader, B., Ragni, M., Rasko, J. J. E., Ozelo, M. C., Hoots, K., Blatt, P., Konkle, B., et al. (2006) · Nature Medicine

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Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

George, L. A., Sullivan, S. K., Giermasz, A., Rasko, J. E. J., Samelson-Jones, B. J., Ducore, J., Cuker, A., Sullivan, L. M., Majumdar, S., Teitel, J., et al. (2017) · New England Journal of Medicine

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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, C. S., Chew, A. J., Hutchison, S., Larson, P. J., Herzog, R. W., Arruda, V. R., Tai, S. J., Ragni, M. V., Thompson, A., Ozelo, M., et al. (2003) · Blood

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Cd8(+) T-Cell Responses to Adeno-Associated Virus Capsid in Humans

Mingozzi, F., Maus, M. V., Hui, D. J., Sabatino, D. E., Murphy, S. L., Rasko, J. E. J., Ragni, M. V., Manno, C. S., Sommer, J., Jiang, H., et al. (2007) · Nature Medicine

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Long-term follow-up of the first in human intravascular delivery of AAV for gene transfer: AAV2-hFIX16 for severe hemophilia b

George, L. A., Ragni, M. V., Rasko, J. E. J., Raffini, L. J., Samelson-Jones, B. J., Ozelo, M., Hazbon, M., Runowski, A. R., Wellman, J. A., Wachtel, K., et al. (2020) · Molecular Therapy

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A Phase 1/2 Trial of Investigational Spk-8011 in Hemophilia a Demonstrates Durable Expression and Prevention of Bleeds

High, K. A., George, L. A., Eyster, M. E., Sullivan, S. K., Ragni, M. V., Croteau, S. E., Samelson-Jones, B. J., Evans, M., Joseney-Antoine, M., Macdougall, A., et al. (2018) · Blood

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Extrahepatic factor VIII production in transplant recipient of hemophilia donor liver

Madeira, C. L., Layman, M. E., de Vera, R. E., Fontes, P. A., & Ragni, M. V. (2009) · Blood

Hemophilia Treatment and ResearchOrgan Transplantation Techniques and Outcomes

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