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, that are designed to bind to RNA and change its properties in specific ways. In the case of a splicing mutation, the short nucleotide is designed to ensure that the RNA is cut and stitched correctly, allowing functional CFTR protein to be made.

Status

Laboratory studies to develop and test these compounds are underway.

Sponsor

This program is sponsored by SpliSense and partially funded by the Cystic Fibrosis Foundation.