Gene-editing therapeutic YOLT-202 shown to restore α1-antitrypsin deficiency
May 22, 2024
α1-Antitrypsin deficiency is a hereditary disorder that affects the liver in children and adults, as well as the lungs in adults. The disease is mostly caused by the Z allele mutation in the SERPINA1 gene, where a glutamic acid amino acid is substituted by lysine (E342K) leading to protein misfolding and aggregation.