Ozelo, Margareth C.

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

Virus-based gene therapy researchHemophilia Treatment and Research

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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

Hemophilia Treatment and ResearchVirus-based gene therapy research

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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

Virus-based gene therapy researchHemophilia Treatment and Research

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Eradication of neutralizing antibodies to factor VIII in canine hemophilia a after liver gene therapy

Finn, J. D., Ozelo, M. C., Sabatino, D. E., Franck, H. W. G., Merricks, E. P., Crudele, J. M., Zhou, S., Kazazian, H. H., Lillicrap, D., Nichols, T. C., et al. (2010) · Blood

Hemophilia Treatment and ResearchVirus-based gene therapy research

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Global seroprevalence of pre-existing immunity against AAV5 and other AAV serotypes in people with hemophilia a

Klamroth, R., Hayes, G., Andreeva, T., Gregg, K., Suzuki, T., Mitha, I. H., Hardesty, B., Shima, M., Pollock, T., Slev, P., et al. (2022) · Human Gene Therapy

Hemophilia Treatment and ResearchVirus-based gene therapy research

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