Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice

Hemophilia A is an X-linked bleeding disorder caused by mutations in the gene encoding the factor VIII (FVIII) coagulation protein. Bleeding episodes in patients are reduced by prophylactic therapy or treated acutely using recombinant or plasma-derived FVIII. We have made an adeno-associated virus 5...

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Detalles Bibliográficos
Autores principales: Bunting, Stuart, Zhang, Lening, Xie, Lin, Bullens, Sherry, Mahimkar, Rajeev, Fong, Sylvia, Sandza, Krystal, Harmon, Danielle, Yates, Bridget, Handyside, Britta, Sihn, Choong-Ryoul, Galicia, Nicole, Tsuruda, Laurie, O’Neill, Charles A., Bagri, Anil, Colosi, Peter, Long, Shinong, Vehar, Gordon, Carter, Barrie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5835117/
https://www.ncbi.nlm.nih.gov/pubmed/29292164
http://dx.doi.org/10.1016/j.ymthe.2017.12.009

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