Rare Disease Report

Gene Therapy and Batten Disease - Preclinical Data

OCTOBER 08, 2016
Tammy Kielian, PhD



Tammy Kielian, PhD of the University of Nebraska Medical Center summarizes the recent study published in the Journal of Neuroscience testing gene therapy in a mouse model for a juvenile form of Batten disease (CLN3).
 
The data was presented at the 15th International Conference on Neuronal Ceroid Lipofuscinosis in Boston, MA.

Reference

Bosch ME, Aldrich A, Fallet R, et al. Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3). J Neurosci. 2016;36:9669-9682. doi: 10.1523/JNEUROSCI.1635-16.2016


This article was made possible by a sponsorship from BioMarin Pharmaceutical Inc. Rare Disease Report has sole control over the editorial content.
 


Stay informed on the latest rare disease news and developments by signing up for our newsletter.
Copyright © RareDR 2013-2018 Rare Disease Communications. All Rights Reserved.