WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation

WAS Promoter-Driven Lentiviral Vectors Mimic Closely the Lopsided WASP Expression during Megakaryocytic Differentiation

Transplant of gene-modified autologous hematopoietic progenitors cells has emerged as a new therapeutic approach for Wiskott-Aldrich syndrome (WAS), a primary immunodeficiency with microthrombocytopenia and abnormal lymphoid and myeloid functions. Despite the clinical benefits obtained in ongoing cl...

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Bibliographic Details
Main Authors: Pilar Muñoz, María Tristán-Manzano, Almudena Sánchez-Gilabert, Giorgia Santilli, Anne Galy, Adrian J. Thrasher, Francisco Martin
Format: Article
Language:English
Published: Elsevier 2020-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
Wiskott-Aldrich syndrome
alternative WAS promoter
hematopoietic stem and progenitor cells
HSPCs
WASKO cellular models
megakaryocytic differentiation
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050120301911

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http://www.sciencedirect.com/science/article/pii/S2329050120301911

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