Recent advances in CF treatment aim to develop specific drugs targeting classes of molecular defects underlying CF. No such therapies are available for patients carrying splicing mutations. About 13% of CF mutations are classified as splicing defects. The plant hormone kinetin was shown to correct RNA aberrant splicing in 2 severe genetic diseases (familial dysautonomia and neurofibromatosis). This project aims to investigate the therapeutic potential of kinetin to treat CF splicing defects and to perform a search of other molecules similar to kinetin. The ability of kinetin to normalize splicing in different cell model systems and subsequently in cell lines derived from patients carrying splicing defects will be tested. In parallel, novel compounds similar to kinetin by a computational screening of a virtual library will be searched.
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€ 60.000
€ 25.000
€ 20.000