Mutations in protein coding-sequence and regulatory elements can cause altered gene expression which can cause diseases. Mis-regulated gene expression can be corrected by direct editing of genomic elements. Indeed, artificial transcription factors can regulate gene expression resulting rescue phenotypes. The fact that less than 2% of our genome encodes protein suggests regulatory elements are promising candidates for therapeutics. We aim to identify and develop strategy for correction of disease-associated aberrations using CRISPR and its derivative technology.