Liver Biopsy Confirms Gene Correction in Alpha-1 Antitrypsin Deficiency Patient

A liver biopsy has confirmed that adenine base editing successfully corrected the pathogenic SERPINA1 Z variant in a patient with alpha-1 antitrypsin deficiency (AATD). Ten weeks after a single YOLT-202 infusion, over half of the alleles in liver tissue were corrected, with sustained circulating AAT levels and preserved anti-elastase activity.

Originally published by gnews