Review

† Author for correspondence

Email the corresponding author at gyurcsik@chem.u-szeged.hu

Department of Inorganic and Analytical Chemistry, University of Szeged, Dóm tér 7, H-6720 Szeged, Hungary

Published Online:25 Oct 2011https://doi.org/10.4155/fmc.11.139

Abstract

The process of DNA targeting or repair of mutated genes within the cell, induced by specifically positioned double-strand cleavage of DNA near the mutated sequence, can be applied for gene therapy of monogenic diseases. For this purpose, highly specific artificial metallonucleases are developed. They are expected to be important future tools of modern genetics. The present state of art and strategies of research are summarized, including protein engineering and artificial ‘chemical’ nucleases. From the results, we learn about the basic role of the metal ions and the various ligands, and about the DNA binding and cleavage mechanism. The results collected provide useful guidance for engineering highly controlled enzymes for use in gene therapy.

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Published online 25 October 2011

Published in print November 2011

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Financial & competing interests disclosure

The financial support of the Hungarian Scientific Research Foundation (OTKA-NKTH CK80850) and TÁMOP-4.2.1/B-09/1 is greatly acknowledged. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

No writing assistance was utilized in the production of this manuscript.

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Towards artificial metallonucleases for gene therapy: recent advances and new perspectives

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