This program is studying messenger RNA (mRNA) delivery to treat rare CFTR mutations, including nonsense mutations. Nonsense mutations (also known as “x” or “stop” mutations) in the CFTR gene cause the production of CFTR protein to stop prematurely. Delivery of mRNA would allow lung cells to make full-length, functional CFTR protein.

Status

Laboratory studies to develop and test this potential therapy are underway.

Sponsor

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