Gene Therapy Ameliorates Cardiovascular Disease in Dogs With Mucopolysaccharidosis VII

摘要: Background— Mucopolysaccharidosis VII (MPS VII) is a lysosomal storage disease caused by deficient β-glucuronidase (GUSB) activity resulting in defective catabolism of glycosaminoglycans (GAGs). Cardiac major cause death MPS because accumulation GAGs cardiovascular cells. Manifestations include cardiomyopathy, mitral and aortic valve thickening, root dilation may the early months life or be compatible with fairly normal lifespan. We previously reported that neonatal administration retroviral vector (RV) resulted transduction hepatocytes, which secreted GUSB into blood could taken up cells throughout body. The goal this study was to evaluate effect on cardiac disease. Methods Results— Six dogs were treated intravenously an RV-expressing canine GUSB. Echocardiographic parameters, lesions, biochemical parameters these compared those unt...