Work

468. GLP-Compliant Non-Clinical Safety and Biodistribution of a Recombinant AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

Year: 2016

Type: article

Abstract: Mucopolysaccharidosis VI (MPS VI) is a lysosomal storage disorder caused by deficiency of the enzyme arylsulfatase B (ARSB), which results in widespread accumulation and excretion of toxic glycosaminoglycans. We recently developed a successful gene therapy approach based on a single systemic administration of AAV2/8 that targets liver of MPS VI animal models. In view of a gene therapy clinical trial for MPS VI, we performed GLP-compliant non-clinical studies to assess the safety and biodistribution of AAV2/8.

Source: Molecular Therapy

DOI: 10.1016/s1525-0016(16)33277-4