Journal
CELLULAR AND MOLECULAR LIFE SCIENCES
Volume 67, Issue 5, Pages 727-748Publisher
SPRINGER BASEL AG
DOI: 10.1007/s00018-009-0188-y
Keywords
Homing endonucleases; Protein engineering; Monogenic diseases; Double-strand break; DNA repair; Gene therapy; Protein structure; Protein-DNA interaction
Categories
Funding
- European Union [LSHG-CT-2006-037226]
- Ministerio de Ciencia e Inovacion (MICINN) [BFU2007-30703-E, BFU2008-01344]
- ETORTEK-2008
- Spanish MICINN
Ask authors/readers for more resources
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.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available