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Dolan Sondhi, PhD; Neil R. Hackett, PhD; Robin L. Apblett, BA; Stephen M. Kaminsky, PhD; Robert G. Pergolizzi, PhD; Ronald G. Crystal, MD

Arch Neurol. 2001;58:1793-1798.

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.

From the Institute of Genetic Medicine and Belfer Gene Therapy Core Facility, Weill Medical College of Cornell University, New York, NY.

Corresponding author and reprints: Ronald G. Crystal, MD, Institute of Genetic Medicine, Weill Medical College of Cornell University, 515 E 71st St, Suite 1000, New York, NY 10021 (e-mail: geneticmedicine{at}med.cornell.edu).

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