AAV-mediated gene transfer in the perinatal period results in expression of FVII at levels that protect against fatal spontaneous hemorrhage

We explored adeno-associated viral vector (AAV)–mediated gene transfer in the perinatal period in animal models of severe congenital factor VII (FVII) deficiency, a disease associated with early postnatal life-threatening hemorrhage. In young adult mice with plasma FVII < 1% of normal, a single t...

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Detalles Bibliográficos
Autores principales: Binny, Christopher, McIntosh, Jenny, Della Peruta, Marco, Kymalainen, Hanna, Tuddenham, Edward G. D., Buckley, Suzanne M. K., Waddington, Simon N., McVey, John H., Spence, Yunyu, Morton, Christopher L., Thrasher, Adrian J., Gray, John T., Castellino, Francis J., Tarantal, Alice F., Davidoff, Andrew M., Nathwani, Amit C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Hematology 2012
Materias:
Gene Therapy
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3271720/
https://www.ncbi.nlm.nih.gov/pubmed/22134170
http://dx.doi.org/10.1182/blood-2011-09-377630

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