Adeno-Associated Virus Gene Transfer Ameliorates Progression of Skeletal Lesions in Mucopolysaccharidosis IVA Mice
Hum Gene Ther. 2024 Oct 25. doi: 10.1089/hum.2024.096. Online ahead of print. ABSTRACT Mucopolysaccharidosis type IVA (MPS IVA) is an autosomal congenital metabolic lysosomal disease …