Genetic engineering treatment with zinc-finger nucleases

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The most lethal etiologic agent of malaria is the Plasmodium falciparum parasite. Straimer et al. (2012) report on the successful genetic engineering of Plasmodium falciparum using zinc-finger nucleases (ZFNs), as a potential treatment for malaria refractory to existing medication. ZFNs produce a double-strand break in a user-defined locus and trigger homology-directed repair.

To realize the full potential of ZFNs in genome engineering the development of safe and effective methods for delivering ZFNs into cells are required. Gaj et al. (2012) report on the cell-penetrating property of ZFNs allowing them to be delivered to several cell types as proteins. Gaj et al. show that direct delivery of ZFNs as proteins leads to a dose-dependent endogenous gene disruption in various mammalian cell types, which is efficient and has minimal off-target effects.

References:

Site-specific genome editing in Plasmodium falciparum using engineered zinc-finger nucleases. Straimer et al. Nat Methods. 2012 Oct;9(10):993-8. doi: 10.1038/nmeth.2143.
Epub 2012 Aug 26. PMID: 22922501

Targeted gene knockout by direct delivery of zinc-finger nuclease proteins. Gaj et al. Nat Methods. 2012 Jul 1;9(8):805-7. PMID: 22751204

 

Yannis Trakadis, MD

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