Scientists test genetic patch to fix cystic fibrosis flaw
NCT ID NCT05100823
Summary
This study aimed to test if a new type of genetic medicine, called oligonucleotide blockers, could fix a specific type of error in the DNA of people with cystic fibrosis. Researchers grew airway and rectal cells from 16 patients in the lab to see if the treatment could restore the function of the faulty protein that causes the disease. The study was terminated and did not involve giving the treatment directly to patients.
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Locations
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Montpellier University Hospital
Montpellier, 34090, France
Conditions
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