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Partial correction of the CNS lysosomal storage defect in a mouse model of juvenile neuronal ceroid lipofuscinosis by neonatal CNS administration of an adeno-associated virus serotype rh.10 vector expressing the human CLN3 gene.

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author: Susan L Cotman  Hemanth R Nelvagal  Jonathan D. Cooper 

Publication date March 4, 2014
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