Anguela, Xavier M.

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

Hemophilia Treatment and ResearchVirus-based gene therapy research

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In vivo genome editing restores haemostasis in a mouse model of haemophilia

Li, H., Haurigot, V., Doyon, Y., Li, T., Wong, S. Y., Bhagwat, A. S., Malani, N., Anguela, X. M., Sharma, R., Ivanciu, L., et al. (2011) · Nature

Hemophilia Treatment and ResearchCRISPR and Genetic Engineering

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Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys

Mingozzi, F., Anguela, X. M., Pavani, G., Chen, Y., Davidson, R. J., Hui, D. J., Yazicioglu, M., Elkouby, L., Hinderer, C. J., Faella, A., et al. (2013) · Science Translational Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

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IgG-cleaving endopeptidase enables in vivo gene therapy in the presence of anti-AAV neutralizing antibodies

Leborgne, C., Barbon, E., Alexander, J. M., Hanby, H., Delignat, S., Cohen, D. M., Collaud, F., Muraleetharan, S., Lupo, D., Silverberg, J., et al. (2020) · Nature Medicine

Virus-based gene therapy researchRNA Interference and Gene Delivery

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In vivo genome editing of the albumin locus as a platform for protein replacement therapy

Sharma, R., Anguela, X. M., Doyon, Y., Wechsler, T., DeKelver, R. C., Sproul, S., Paschon, D. E., Miller, J. C., Davidson, R. J., Shivak, D., et al. (2015) · 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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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

Hemophilia Treatment and ResearchVirus-based gene therapy research

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