CONCEPTION D'ENDONUCLÉASES DE RESTRICTION À SITES RARES POUR LE CIBLAGE EFFICACE ET SPÉCIFIQUE DE SÉQUENCES ADN COMPRENANT DES MOTIFS TRÈS RÉPÉTITIFS

摘要

The present invention is in the field of genetic editing tools and methods of genetic engineering. It relates to the engineering of rare-cutting endonucleases designed to contract highly repetitive motives in chromosomes, which are at the origin of certain genetic diseases, in particular the so-called “triplet repeat diseases”, such as the Huntington disease. The invention encompasses the method for contracting the repetitive motives, the rare-cutting endonucleases for use to contract repetitive motives in a gene subjected to repeat disorder, the polynucleotides and vectors encoding thereof as well as the resulting pharmaceutical compositions.