Zinc-finger-based transcriptional repression of rhodopsin in a model of dominant retinitis pigmentosa.

ABSTRACT: Despite the recent success of gene-based complementation approaches for genetic recessive traits, the development of therapeutic strategies for gain-of-function mutations poses great challenges. General therapeutic principles to correct these genetic defects mostly rely on post-transcriptional gene regulation (RNA silencing). Engineered zinc-finger (ZF) protein-based repression of transcription may represent a novel approach for treating gain-of-function mutations, although proof-of-concept of this use is still lacking. Here, we generated a series of transcriptional repressors to silence human rhodopsin (hRHO), the gene most abundantly expressed in retinal photoreceptors. The strategy was designed to suppress both the mutated and the wild-type hRHO allele in a mutational-independent fashio

SUBMITTER: Mussolino C 

PROVIDER: S-EPMC3085076 | biostudies-literature | 2011 Mar

REPOSITORIES: biostudies-literature