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Updated: Mar 15, 2026
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An In Vitro Model for the Study of Cellular Pathophysiology in Globoid Cell Leukodystrophy
Published on: October 21, 2014
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Perspective on innovative therapies for globoid cell leukodystrophy
Alessandra Ricca1, Angela Gritti2
1San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), Division of Regenerative Medicine, Stem Cells and Gene Therapy, IRCCS San Raffaele Scientific Institute, Milan, Italy.
Journal of Neuroscience Research
|September 18, 2016
Summary
Globoid cell leukodystrophy (GLD), also known as Krabbe's disease, is a severe neurological disorder. Research highlights early intervention and global tissue targeting as crucial for effective GLD treatments.
Area of Science:
- Biochemistry
- Genetics
- Neurology
Background:
- Globoid cell leukodystrophy (GLD), or Krabbe's disease, is a lysosomal storage disorder caused by galactosylceramidase deficiency.
- Infantile GLD presents with aggressive neurological deterioration and diverse complications.
Purpose of the Study:
- To review GLD pathology mechanisms and therapeutic implications.
- To evaluate evidence from animal models for effective treatment strategies.
Main Methods:
- Review of existing literature on GLD pathophysiology.
- Analysis of experimental data from GLD animal models.
Main Results:
- Novel mechanisms contributing to GLD pathology have been identified.
- Key requirements for therapeutic benefit include early intervention, high enzyme levels, and broad tissue targeting.
Conclusions:
- Understanding GLD pathophysiology and therapy interplay is vital for developing better treatments.
- Continued research may lead to innovative approaches and prioritized treatment strategies for GLD.
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