What is CRISPR?
CRISPR is an advanced DNA-editing technology that allows scientists to correct certain mutations in the CFTR gene, which is responsible for Cystic Fibrosis. Unlike current treatments, which improve CFTR protein function, CRISPR aims to correct the genetic cause of the disease itself.
How does it work?
The technology uses an enzyme (such as Cas9 or other editing systems) and a guide molecule that locates the mutation in DNA. The goal is to correct the mutation so that cells can once again produce a functional CFTR protein.
Where does research stand in 2026?
As of 2026, CRISPR is not yet an approved treatment for people with Cystic Fibrosis. Most studies are still laboratory-based or in early stages of development. However, results in recent years have been promising and have shown that some mutations can be successfully corrected in lung cells in the laboratory.
Key developments:
- New techniques have been developed, such as base editing and prime editing, which can correct certain mutations more precisely than conventional CRISPR.
- Researchers are working to deliver the editing system directly to the lungs using lipid nanoparticles (LNPs), without using viruses.
- These technologies are particularly important for patients who do not benefit from CFTR modulators because they produce no CFTR protein or have very rare mutations.