Article
Highly efficient endogenous human gene correction using designed zinc-finger nucleases.
Nature - 2 Jun 2005
Urnov Fyodor D, Miller Jeffrey C, Lee Ya-Li, Beausejour Christian M, Rock Jeremy M, Augustus Sheldon, Jamieson Andrew C, Porteus Matthew H, Gregory Philip D, Holmes Michael C
Abstract excerpt
Permanent modification of the human genome in vivo is impractical owing to the low frequency of homologous recombination in human cells, a fact that hampers biomedical research and progress towards safe and effective gene therapy. Here we report a general solution using two fundamental biological processes: DNA recognition by C2H2 zinc-finger proteins and homology-directed repair of DNA double-strand breaks....
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