Biochemical, Pathological, and Skeletal Improvement of Mucopolysaccharidosis VI After Gene Transfer to Liver but Not to Muscle

Abstract excerpt

Mucopolysaccharidosis VI (MPS VI) is caused by deficient activity of arylsulfatase B (ARSB), resulting in intralysosomal storage of dermatan sulfate (DS) and multisystem disease without central nervous system involvement. After gene transfer, muscle or liver can theoretically be converted into factories for systemic ARSB secretion, leading to uptake by non-transduced cells. We have injected newborn MPS VI rats...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Cytomegalovirus and herpesvirus research
• Share to Lysosomal Storage Disorders Research
• Share to Virus-based gene therapy research