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Gene Therapy Targets a Hard-to-Treat Cystic Fibrosis Mutation
Key Takeaways
- Researchers targeted the 1717-1G >A mutation in cystic fibrosis.
- The mutation affects about 10% of patients and does not respond to some current therapies.
- The team describes an mRNA-based genome-editing strategy designed to permanently repair the defect.
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DT Editorial Team··via medicalxpress.com