Homing endonucleases: from basics to therapeutic applications |
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Authors: | Maria J Marcaida Inés G Muñoz Francisco J Blanco Jesús Prieto Guillermo Montoya |
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Institution: | 1. Macromolecular Crystallography Group, Structural Biology and Biocomputing Programme, Spanish National Cancer Research Centre (CNIO), c/Melchor Fdez. Almagro 3, 28029, Madrid, Spain 2. Ikerbasque Professor Structural Biology Unit, CIC bioGUNE, Parque Tecnológico de Vizcaya, 48160, Derio, Spain
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Abstract: | Homing endonucleases (HE) are double-stranded DNAses that target large recognition sites (12–40 bp). HE-encoding sequences
are usually embedded in either introns or inteins. Their recognition sites are extremely rare, with none or only a few of
these sites present in a mammalian-sized genome. However, these enzymes, unlike standard restriction endonucleases, tolerate
some sequence degeneracy within their recognition sequence. Several members of this enzyme family have been used as templates
to engineer tools to cleave DNA sequences that differ from their original wild-type targets. These custom HEs can be used
to stimulate double-strand break homologous recombination in cells, to induce the repair of defective genes with very low
toxicity levels. The use of tailored HEs opens up new possibilities for gene therapy in patients with monogenic diseases that
can be treated ex vivo. This review provides an overview of recent advances in this field. |
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