CORDIS Project
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The ALLELECHOKER project aims to develop a gene therapy for treating inherited dominant disorders, particularly focusing on retinitis pigmentosa caused by mutations in the RHODOPSIN gene. It utilizes synthetic transcription factors to silence the faulty gene and replace it with a functional copy.
At present therapies for inherited dominant disorders are not available, thus representing an urgent unmet medical and social need.
The ERC Grant ALLELECHOKER enabled the PI to discover three modes to generate transcriptional repression by transcription factors (TFs) based on Zinc-finger scaffold: i- synthetic transcription factor (TF;
Mussolino et al., EMBO Mol.
Med. 2011 Mar;3(3):118-28;
Botta S. et al.
Elife. 2016 Mar 14;5), ii- synthetic DNA-binding protein (Botta S. et al.
Elife. 2016 Mar 1…
UNIVERSITA DEGLI STUDI DI NAPOLI FEDERICO II
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Italy, Roma
Type: Research institute
Activity type: Research Organisations
SME: No
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