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
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