ARTIFICIAL TRANSCRIPTION FACTORS FOR THE TREATMENT OF DISEASES CAUSED BY OPA1 HAPLOINSUFFICIENCY

摘要

The invention relates to an artificial transcription factor comprising a polydactyl zinc finger protein targeting specifically the OPA1 promoter fused to an activatory protein domain, and a nuclear localization sequence. Artificial transcription factors directed against the OPA1 promoter are useful for the treatment of diseases associated with OPA1 haploinsufficiency, such as autosomal dominant optic atrophy, syndromic autosomal dominant optic atrophy plus and normal tension glaucoma.