This program is developing suppressor tRNA delivery to treat 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 suppressor tRNA would allow lung cells to override these premature stop signals and make full-length, functional CFTR protein.
Laboratory studies to develop and test this potential therapy are underway.
This program is sponsored by ReCode Therapeutics and partially funded by the Cystic Fibrosis Foundation.
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