ARTIFICIAL TRANSCRIPTION FACTORS ENGINEERED TO OVERCOME ENDOSOMAL ENTRAPMENT

摘要

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.

主权项

1. An artificial transcription factor comprising a polydactyl zinc finger protein targeting specifically a gene promoter fused to an activatory or inhibitory protein domain, a nuclear localization sequence, a protein transduction domain, and an endosome-specific protease recognition site.