Intracranial delivery of CLN2 reduces brain pathology in a mouse model of classical late infantile neuronal ceroid lipofuscinosis

Marco A. Passini*, James C. Dodge, Jie Bu, Wendy Yang, Qi Zhao, Dolan Sondhi, Neil R. Hackett, Stephen M. Kaminsky, Qinwen Mao, Lamya S. Shihabuddin, Seng H. Cheng, David E. Sleat, Gregory R. Stewart, Beverly L. Davidson, Peter Lobel, Ronald G. Crystal

*Corresponding author for this work

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Medicine & Life Sciences