Feasibility of gene therapy for late neuronal ceroid lipofuscinosis.

Publication Type Review
Authors Sondhi D, Hackett N, Apblett R, Kaminsky S, Pergolizzi R, Crystal R
Journal Arch Neurol
Volume 58
Issue 11
Pagination 1793-8
Date Published 11/01/2001
ISSN 0003-9942
Keywords Endopeptidases, Genetic Therapy, Neuronal Ceroid-Lipofuscinoses
Abstract Late infantile neuronal ceroid lipofuscinosis is a progressive childhood neurodegenerative disorder characterized by intracellular accumulation of autofluorescent material resembling lipofuscin in neuronal cells. This report summarizes the new therapies under consideration for late infantile neuronal ceroid lipofuscinosis, with a focus on strategies for in vivo gene therapy for the retinal and central nervous system manifestations of the disease.
DOI 10.1001/archneur.58.11.1793
PubMed ID 11708986
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