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Drug delivery

Engineered, zinc finger domain-containing recombinases for site-specific delivery of therapeutic genes

An engineered recombinase could enable nontoxic, site-specific delivery of therapeutic genes to the human genome. Recombinases containing zinc finger, DNA-binding domains form heterodimers capable of site-specific gene delivery but also form homodimers that can modify off-target genes. In a human cell line, a recombinase containing an engineered variant of the zinc finger domain exhibited over 500-fold less homodimerization than a recombinase containing the unmodified domain. In the cell line, the engineered recombinase was used to deliver human factor IX or a-galactosidase A into the genome with no signs of off-target delivery. Future studies could include developing viral vectors for cell- or tissue-targeted in vivo delivery of the engineered recombinase.

SciBX 7(13); doi:10.1038/scibx.2014.383
Published online April 3, 2014

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Gaj, T. et al. J. Am. Chem. Soc.; published online March 10, 2014;
doi:10.1021/ja4130059
Contact: Carlos F. Barbas III, The Scripps Research Institute, La Jolla, Calif.
e-mail:

carlos@scripps.edu