Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

D Sondhi; N R Hackett; R L Apblett; S M Kaminsky; R G Pergolizzi; R G Crystal

doi:10.1001/archneur.58.11.1793

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

Arch Neurol. 2001 Nov;58(11):1793-8. doi: 10.1001/archneur.58.11.1793.

Authors

D Sondhi¹, N R Hackett, R L Apblett, S M Kaminsky, R G Pergolizzi, R G Crystal

Affiliation

¹ Institute of Genetic Medicine and Belfer Gene Therapy Core Facility, Weill Medical College of Cornell University, 515 E 71st St, Suite 1000, New York, NY 10021, USA.

PMID: 11708986
DOI: 10.1001/archneur.58.11.1793

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.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Adult
Aminopeptidases
Animals
Child
Child, Preschool
Clinical Trials as Topic
Dipeptidyl-Peptidases and Tripeptidyl-Peptidases
Endopeptidases / genetics*
Endopeptidases / therapeutic use
Genetic Therapy*
Genetic Vectors
Humans
Neuronal Ceroid-Lipofuscinoses / genetics*
Neuronal Ceroid-Lipofuscinoses / therapy*
Serine Proteases
Stem Cell Transplantation
Tripeptidyl-Peptidase 1

Substances

Tripeptidyl-Peptidase 1
Endopeptidases
Serine Proteases
Aminopeptidases
Dipeptidyl-Peptidases and Tripeptidyl-Peptidases