Andrew C. Jamieson

TL;DR: It is shown that zinc-finger nucleases designed against an X-linked severe combined immune deficiency mutation in the IL2Rγ gene yielded more than 18% gene-modified human cells without selection, raising the possibility of strategies based on zinc- finger nucleases for the treatment of disease.

TL;DR: In this article, the authors provide criteria and methods for selecting optimum subsequence(s) from a target for targeting by a zinc finger protein, which can be used following the preselection of target sites according to the procedures and criteria described above.

TL;DR: These data establish, for the first time, that specifically designed transcription factors can regulate an endogenous gene in vivo and evoke a potentially therapeutic biophysiologic effect.

TL;DR: The methods used to design these DNA-binding domains are summarized, how they are incorporated into novel transcription factors (and other useful molecules) are explained and some key applications in drug discovery are described.

TL;DR: This work presents evidence for an enhanced activation of VEGF-A gene transcription by ZFP transcription factors fused to VP16 and p65 targeted to two distinct chromosomal sites >500 base pairs upstream or downstream of the transcription start site.