Correction of SCID-X1 using an enhancerless Vav promoter.

  title={Correction of SCID-X1 using an enhancerless Vav promoter.},
  author={Elena Almarza and Feijie Zhang and Giorgia Santilli and Michael Patrick Blundell and Steven J Howe and Susannah I. Thornhill and Juan Antonio Bueren and Adrian J. Thrasher},
  journal={Human gene therapy},
  volume={22 3},
The efficacy of gene therapy for the treatment of inherited immunodeficiency has been highlighted in recent clinical trials, although in some cases complicated by insertional mutagenesis and silencing of vector genomes through methylation. To minimize these effects, we have evaluated the use of regulatory elements that confer reliability of gene expression, but also lack potent indiscriminate enhancer activity. The Vav1 proximal promoter is particularly attractive in this regard and may be… CONTINUE READING