Scientists test genetic patch to fix cystic fibrosis at its source
NCT ID NCT05100823
Summary
This study tested a new type of genetic therapy designed to fix a specific type of error in the CFTR gene that causes cystic fibrosis. Researchers used cells from 16 patients to see if custom-made 'oligonucleotide blockers' could repair the gene's instructions and restore normal protein function. The goal was to validate this approach in the lab as a potential future treatment for patients with these specific genetic mutations.
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Locations
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Montpellier University Hospital
Montpellier, 34090, France
Conditions
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