SpliSense solution treats the root cause of the disease – the mutation – to eliminate disease symptoms and improve patients’ Quality of Life (QOL). Our platform restores protein functionality by increasing the level of mature functional proteins. This is performed by modulating the splicing pattern of gene mutations related to pulmonary diseases. Our most advanced programs focus on Cystic Fibrosis treatments, modulating Cystic fibrosis Transmembrane Conductance Regulator (CFTR) RNA transcripts in patients carrying mutations to produce mature and functional CFTR proteins.
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