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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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2016
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5363182/
https://ncbi.nlm.nih.gov/pubmed/28344987
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2016.11.001
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