Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery

Abstract

The present study demonstrates the efficacy of the intraventricular injection as a tool to target the brain with therapeutic genes in adult MPS IIIA mice, and provides evidence supporting this approach as a potentially effective means of treating CNS pathology in MPS IIIA patients.