2002•Molecular TherapyOpen access

754. A Corrective Gene Delivery Strategy for the Neurodegenerative Lysosomal Storage Disease Late Infantile Neuronal Ceroid Lipofuscinosis

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Abstract

Dolan Sondhi,1 Barbara S. Mendez,1 Benjamin R. Bergman,1 Michael Lam,1 Elizabeth Salvin,1 Philip L. Leopold,1 Neil R. Hackett,1 Stephen M. Kaminsky,1 Daniel A. Peterson,2 John R. Sladek, Jr.,3 Ronald G. Crystal.1

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Key concepts: Neuronal ceroid lipofuscinosis, Batten disease, Lysosomal storage disease, Lysosomal storage disorders, Biology, Disease, Gene, Medicine

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754. A Corrective Gene Delivery Strategy for the Neurodegenerative Lysosomal Storage Disease Late Infantile Neuronal Ceroid Lipofuscinosis — Research Paper | ScholarLens