| abstract |
The present invention is in the field of genetic editing tools and methods of genetic engineering. This relates to the manipulation of rare-cut nucleases designed to shorten highly repetitive motifs in chromosomes that are responsible for certain genetic diseases, especially so-called “triplet repeat diseases” such as Huntington's disease. The present invention includes a method for shortening a repetitive motif, a rare-cutting endonuclease used to shorten a repetitive motif of a repetitive disorder gene, a polynucleotide and vector encoding the same, and the resulting pharmaceutical composition To do. |