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

ConclusionsThe current 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 potentially an effective means to treat CNS pathology in MPS IIIA patients. This article is protected by copyright. All rights reserved.

http://onlinelibrary.wiley.com/resolve/doi?DOI=10.1002%2Fjgm.2816

Source: The Journal of Gene Medicine - September 1, 2014 Category: Genetics & Stem Cells Authors: Chantelle McIntyre, Ainslie L. K. Derrick Roberts, Sharon Byers, Donald S. Anson Tags: Research Article Source Type: research