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 OPA 1 promoter fused to an activatory protein demain, and a nuclear localization sequence. Artificial transcription factors directed against the OPA 1 promoter are useful for the treatment of diseases associated with OPA 1 haploinsufficiency, such as autosomal dominant optic atrophy, syndromic autosomal dominant optic atrophy plus and normal tension glaucoma.