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