Ontology highlight
ABSTRACT:
SUBMITTER: Ponder KP
PROVIDER: S-EPMC3345988 | biostudies-literature | 2012 May
REPOSITORIES: biostudies-literature
Ponder Katherine P KP O'Malley Thomas M TM Wang Ping P O'Donnell Patricia A PA Traas Anne M AM Knox Van W VW Aguirre Gustavo A GA Ellinwood N Matthew NM Metcalf Jason A JA Wang Bin B Parkinson-Lawrence Emma J EJ Sleeper Meg M MM Brooks Doug A DA Hopwood John J JJ Haskins Mark E ME
Molecular therapy : the journal of the American Society of Gene Therapy 20120306 5
Mucopolysaccharidosis (MPS) VI is due to a deficiency in the activity of N-acetylgalactosamine 4-sulfatase (4S), also known as arylsulfatase B. Previously, retroviral vector (RV)-mediated neonatal gene therapy reduced the clinical manifestations of MPS I and MPS VII in mice and dogs. However, sulfatases require post-translational modification by sulfatase-modifying factors. MPS VI cats were injected intravenously (i.v.) with a gamma RV-expressing feline 4S, resulting in 5 ± 3 copies of RV per 10 ...[more]