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

The immunodeficiency disorder Wiskott-Aldrich syndrome (WAS) leads to life-threatening hematopoietic cell dysfunction. We used WAS protein (WASp)–deficient mice to analyze the in vivo efficacy of lentiviral (LV) vectors using either a viral-derived promoter, MND, or the human proximal WAS promoter (...

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Hlavní autoři: Astrakhan, Alexander, Sather, Blythe D., Ryu, Byoung Y., Khim, Socheath, Singh, Swati, Humblet-Baron, Stephanie, Ochs, Hans D., Miao, Carol H., Rawlings, David J.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Hematology 2012
Témata:
Gene Therapy
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3362358/
https://ncbi.nlm.nih.gov/pubmed/22431569
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2011-03-340711
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