This program is working to develop potential therapies for people with CF who have splicing mutations. Splicing is an essential process in which RNA is cut into pieces and then stitched back together in a specific way. Splicing mutations in the CFTR gene cause the RNA to be cut or stitched incorrectly, leading to a mutated CFTR protein. This program is studying short nucleotides, or small pieces of genetic material, the short nucleotide is designed to ensure that the RNA is cut and stitched correctly, allowing functional CFTR protein to be made.
Laboratory studies to develop and test these compounds are underway.
This program is sponsored by SpliSense and partially funded by the Cystic Fibrosis Foundation.
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