Gene Therapy and Batten Disease - Preclinical Data

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.


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.
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