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

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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Bibliographic Details
Main Authors: Swati Singh, Iram Khan, Socheath Khim, Brenda Seymour, Karen Sommer, Matthew Wielgosz, Zachary Norgaard, Hans-Peter Kiem, Jennifer Adair, Denny Liggitt, Arthur Nienhuis, David J. Rawlings
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Wiskott-Aldrich Syndrome
gene therapy
insulated lentivirus
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050116301292

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http://www.sciencedirect.com/science/article/pii/S2329050116301292

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