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

@article{Astrakhan2012UbiquitousHG,
  title={Ubiquitous high-level gene expression in hematopoietic lineages provides effective lentiviral gene therapy of murine Wiskott-Aldrich syndrome.},
  author={Alexander Astrakhan and Blythe D. Sather and Byoung Y. Ryu and Socheath Khim and Swati Singh and St{\'e}phanie Humblet-Baron and Hans D. Ochs and Carol H. Miao and David J Rawlings},
  journal={Blood},
  year={2012},
  volume={119 19},
  pages={
          4395-407
        }
}
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 (WS1.6) for human WASp expression. Transplantation of stem cells transduced with MND-huWASp LV resulted in sustained, endogenous levels of WASp in all hematopoietic lineages, progressive selection for… CONTINUE READING
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References

Publications referenced by this paper.
Showing 1-10 of 51 references

Solving the problem of γ-retroviral vectors containing long terminal repeats.

Molecular therapy : the journal of the American Society of Gene Therapy • 2011