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Safe and Effective Gene Therapy for Murine Wiskott-Aldrich Syndrome Using an Insulated Lentiviral Vector

Wiskott-Aldrich syndrome (WAS) is a life-threatening immunodeficiency caused by mutations within the WAS gene. Viral gene therapy to restore WAS protein (WASp) expression in hematopoietic cells of patients with WAS has the potential to improve outcomes relative to the current standard of care, allog...

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Detalles Bibliográficos
Autores principales:	Singh, Swati, Khan, Iram, Khim, Socheath, Seymour, Brenda, Sommer, Karen, Wielgosz, Matthew, Norgaard, Zachary, Kiem, Hans-Peter, Adair, Jennifer, Liggitt, Denny, Nienhuis, Arthur, Rawlings, David J.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2016
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363182/ https://www.ncbi.nlm.nih.gov/pubmed/28344987 http://dx.doi.org/10.1016/j.omtm.2016.11.001

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