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Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome

Alexander Astrakhan, Blythe D. Sather, Byoung Y. Ryu, Socheath Khim, Swati Singh, Stephanie Humblet-Baron, Hans D. Ochs, Carol H. Miao, David J. Rawlings
Journal: Blood
Blood (2012) 119 (19): 4395–4407.
https://doi.org/10.1182/blood-2011-03-340711
Published: 2012
Includes: Supplemental data