Immunological considerations for treating globoid cell leukodystrophy

Subha Karumuthil-Melethil, Steven J. Gray

Research output: Contribution to journalReview article

3 Scopus citations

Abstract

Globoid cell leukodystrophy (GLD, or Krabbe's disease) is a severe inherited neurodegenerative disease caused by the lack of a lysosomal enzyme, GALC. The disease has been characterized in humans as well as three naturally occurring animal models, murine, canine, and nonhuman primate. Multiple treatment strategies have been explored for GLD, including enzyme replacement therapy, small-molecule pharmacological approaches, gene therapy, and bone marrow transplant. No single therapeutic approach has proved to be entirely effective, and the reason for this is not well understood. It is unclear whether initiation of a neuroinflammatory cascade in GLD precedes demyelination, a hallmark of the disease, but it does precede overt symptoms. This Review explores what is known about the role of inflammation and the immune response in the progression of GLD as well as how various treatment strategies might interplay with innate and adaptive immune responses involved in GLD. The focus of this Review is on GLD, but these concepts may have relevance for other, related diseases.

Original languageEnglish (US)
Pages (from-to)1349-1358
Number of pages10
JournalJournal of Neuroscience Research
Volume94
Issue number11
DOIs
StatePublished - Nov 1 2016

Keywords

  • GALC
  • globoid cell leukodystrophy
  • immune
  • Krabbe's disease
  • neuroinflammation
  • psychosine

ASJC Scopus subject areas

  • Cellular and Molecular Neuroscience

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