Feasibility of gene therapy for late neuronal ceroid lipofuscinosis

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

Research output: Contribution to journalReview article

22 Citations (Scopus)

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.

Original languageEnglish
Pages (from-to)1793-1798
Number of pages6
JournalArchives of Neurology
Volume58
Issue number11
Publication statusPublished - 20 Nov 2001
Externally publishedYes

Fingerprint

Neuronal Ceroid-Lipofuscinoses
Genetic Therapy
Lipofuscin
Central Nervous System Diseases
Neurodegenerative Diseases
Central Nervous System
Manifestation
Therapy
Childhood
Cells
Gene Therapy
Therapeutics

ASJC Scopus subject areas

  • Arts and Humanities (miscellaneous)
  • Clinical Neurology

Cite this

Sondhi, D., Hackett, N. R., Apblett, R. L., Kaminsky, S. M., Pergolizzi, R. G., & Crystal, R. (2001). Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. Archives of Neurology, 58(11), 1793-1798.

Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. / Sondhi, Dolan; Hackett, Neil R.; Apblett, Robin L.; Kaminsky, Stephen M.; Pergolizzi, Robert G.; Crystal, Ronald.

In: Archives of Neurology, Vol. 58, No. 11, 20.11.2001, p. 1793-1798.

Research output: Contribution to journalReview article

Sondhi, D, Hackett, NR, Apblett, RL, Kaminsky, SM, Pergolizzi, RG & Crystal, R 2001, 'Feasibility of gene therapy for late neuronal ceroid lipofuscinosis', Archives of Neurology, vol. 58, no. 11, pp. 1793-1798.
Sondhi D, Hackett NR, Apblett RL, Kaminsky SM, Pergolizzi RG, Crystal R. Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. Archives of Neurology. 2001 Nov 20;58(11):1793-1798.
Sondhi, Dolan ; Hackett, Neil R. ; Apblett, Robin L. ; Kaminsky, Stephen M. ; Pergolizzi, Robert G. ; Crystal, Ronald. / Feasibility of gene therapy for late neuronal ceroid lipofuscinosis. In: Archives of Neurology. 2001 ; Vol. 58, No. 11. pp. 1793-1798.
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