Article
Native homing endonucleases can target conserved genes in humans and in animal models.
Nucleic acids research - 1 Aug 2011
Barzel Adi, Privman Eyal, Peeri Michael, Naor Adit, Shachar Einat, Burstein David, Lazary Rona, Gophna Uri, Pupko Tal, Kupiec Martin
Abstract excerpt
In recent years, both homing endonucleases (HEases) and zinc-finger nucleases (ZFNs) have been engineered and selected for the targeting of desired human loci for gene therapy. However, enzyme engineering is lengthy and expensive and the off-target effect of the manufactured endonucleases is difficult to predict. Moreover, enzymes selected to cleave a human DNA locus may not cleave the homologous locus in the...
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