Artificial transcription factor for the treatment of diseases caused by OPA1 haploinsufficiency

摘要

The present invention relates to an artificial transcription factor comprising a polydactyl zinc finger protein that specifically targets the OPA1 promoter fused to an activation protein domain and a nuclear localization sequence. Artificial transcription factors directed against the OPA1 promoter are diseases associated with OPA1 haploinsufficiency such as autosomal dominant optic atrophy plus symptomatic autosomal dominant optic atrophy plus and normal-tension glaucoma Useful for the treatment of