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All articles tagged: Surfactant mutations of the lung (J84.83)

Evalytics 06 November at 03.54 PM

FDA moves closer to sickle cell cure that uses gene editing

The FDA is evaluating a groundbreaking sickle cell disease treatment using CRISPR technology, potentially the first of its kind to be approved. The treatment, known as exa-cel, could eliminate the need for donor bone marrow transplants by editing patients' DNA to correct the mutation causing the disease. With the FDA's final decision expected by December 8, the treatment's efficacy and safety rema