Cystic fibrosis plain language summary: exploring gene therapy possibilities

This Plain Language Summary of Publication article (PLSP) from Future Rare Diseases explains cystic fibrosis (CF), a genetic disease affecting the lungs and other organs. While new CFTR modulator therapies help some CF patients, not everyone can benefit from them, creating an urgent need for additional treatments. Gene therapy offers a promising alternative approach, and this article explains CF, how gene therapy could treat it, and current gene therapy developments for CF patients.

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This PLSP is based on an article called ‘Lentiviral Gene Therapy for Cystic Fibrosis: A Promising Approach and First-in-Human Trial’ and was published in the American Journal of Respiratory and Critical Care Medicine.

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• clinical trials
• Cystic fibrosis
• gene therapy
• lay summary
• plain language summary
• viral vectors