CORDIS Project
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This project aims to develop targeted gene therapies for photoreceptor diseases by silencing the rhodopsin gene using engineered DNA-binding proteins. It combines gene silencing with gene replacement strategies to address mutations effectively.
Zinc finger (ZF) and transcription activator-like effector (TALE) based technologies are been allowing the tailored design of “artificial” DNA-binding proteins targeted to specific and unique DNA genomic sequences.
Coupling DNA binding proteins to effectors domains enables the constitution of DNA binding factors for genomic directed transcriptional modulation or targeted genomic editing.
We have demonstrated that pairing a ZF DNA binding protein to the transcriptional repressor Kruppel-associate…
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