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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Bibliografische gegevens
Gepubliceerd in:Mol Ther Methods Clin Dev
Hoofdauteurs: 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.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: American Society of Gene & Cell Therapy 2016
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Original Article
Online toegang: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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