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Successful Phenotype Improvement following Gene Therapy for Severe Hemophilia A in Privately Owned Dogs

Severe hemophilia A (HA) is an inherited bleeding disorder characterized by <1% of residual factor VIII (FVIII) clotting activity. The disease affects several mammals including dogs, and, like humans, is associated with high morbidity and mortality. In gene therapy using adeno-associated viral (A...

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Detalles Bibliográficos
Autores principales: Callan, Mary Beth, Haskins, Mark E., Wang, Ping, Zhou, Shangzhen, High, Katherine A., Arruda, Valder R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4807047/
https://www.ncbi.nlm.nih.gov/pubmed/27011017
http://dx.doi.org/10.1371/journal.pone.0151800

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