The invention relates to an artificial transcription factor comprising a polydactyl zinc finger protein targeting specifically a gene promoter, engineered to overcome endosomal entrapment after transduction into cells. Such artificial transcription factor comprises a polydactyl zinc finger protein fused to an inhibitory or activatory protein domain, a nuclear localization sequence, a protein transduction domain, and an endosome-specific protease-recognition site. These transducible artificial transcription factors are particularly useful for the treatment of diseases caused or modulated by membrane-bound receptor proteins, nuclear receptor proteins or products of haploinsufficient genes.本發明係關於一種經工程改造以在轉導至細胞中後克服內體捕獲之人工轉錄因子,其包含特異性以基因啟動子為目標之多指鋅指蛋白。該人工轉錄因子包含與抑制性或活化性蛋白質域、核定位序列、蛋白轉導域及內體特異性蛋白酶識別位點融合之多指鋅指蛋白。此等可轉導人工轉錄因子尤其適用於治療由膜結合受體蛋白、核受體蛋白或單倍體不足基因產物造成或調節的疾病。
