Dataset related to the article: "Correcting CFTR mRNA splicing defects with the plant cytokine kinetin and its analogues"

This record contains raw data related to article "Correcting CFTR mRNA splicing defects  with the plant cytokine kinetin and its analogues"

ABSTRACT: Cystic Fibrosis (CF) results from CFTR gene mutations, including splicing defects like the polymorphic TGnTm repeat, which disrupts exon-10 inclusion and contributes to CF monosymptomatic forms. Recent advances in CF treatment yielded targeted therapies for specific CFTR defects. While these drugs offer hope to a subset of patients, the vast majority of splicing variants still lacks a treatment. Small molecules, like the plant cytokine kinetin, have demonstrated efficacy in treating other genetic diseases, offering potential for personalized CF treatments.

This study evaluated the efficacy of kinetin and its analogue, RECTAS, in correcting CFTR exon-10 splicing defects caused by TGnTm repeats. Both compounds enhanced exon-10 inclusion in cell models, with RECTAS demonstrating superior efficacy, achieving up to a fourfold increase in patient-derived cells compared to kinetin. RECTAS also consistently rescued exon-10 splicing across TG-T alleles and restored CFTR protein expression. These findings highlight RECTAS as a potent therapeutic candidate for CF, though further research is needed to optimize dosing, enhance safety, and explore new analogues for personalized splicing-targeted therapies.