The invention relates to the treatment of Huntington's disease (HD) using the Clustered- Regularly Interspaced Short Palindromic Repeats (CRISPR) system. This technology offers the possibility to design a small RNA (sg RNA), which is incorporated into a CRISPR- associated protein (Cas9) to recognize and induce DNA double-strand breaks at a specific target location. In the context of HD, this allows to block the expression of the mutant huntingtin or repair the CAG expansion causing the disease.
GENOME EDITING FOR THE TREATMENT OF HUNTINGTON's DISEASE