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)


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
Issue number11
Publication statusPublished - 20 Nov 2001
Externally publishedYes


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.