Back to Search
Start Over
Treatment for Krabbe's disease: Finding the combination.
- Source :
-
Journal of neuroscience research [J Neurosci Res] 2016 Nov; Vol. 94 (11), pp. 1126-37. - Publication Year :
- 2016
-
Abstract
- Globoid cell leukodystrophy (GLD) is an autosomal recessive neurodegenerative disorder caused by a deficiency of the lysosomal enzyme galactocerebrosidase (GALC). GALC is responsible for catabolism of certain glycolipids, including the toxic compound galactosylsphingosine (psychosine). Histological signs of disease include the widespread loss of myelin in the central and peripheral nervous systems, profound neruroinflammation, and axonal degeneration. Patients suffering from GLD also display neurological deterioration. Many different individual therapies have been investigated in the murine model of the GLD, the Twitcher mouse, with minimal success. The current standard of care for GLD patients, hematopoietic stem cell transplantation, serves only to delay disease progression and is not an effective cure. However, combination therapies that target different pathogenic mechanisms/pathways have been more effective at reducing histological signs of disease, delaying disease onset, prolonging life span, and improving behavioral/cognitive functions in rodent models of Krabbe's disease. In some cases, dramatic synergy between the various therapies has been observed. © 2016 Wiley Periodicals, Inc.<br />Competing Interests: We have no conflicts of interest to report.<br /> (© 2016 Wiley Periodicals, Inc.)
- Subjects :
- Animals
Anti-Inflammatory Agents therapeutic use
Bone Marrow Transplantation
Cycloserine therapeutic use
Disease Models, Animal
Enzyme Replacement Therapy
Galactosylceramidase deficiency
Genetic Therapy
Humans
Leukodystrophy, Globoid Cell genetics
Leukodystrophy, Globoid Cell metabolism
Combined Modality Therapy methods
Leukodystrophy, Globoid Cell therapy
Subjects
Details
- Language :
- English
- ISSN :
- 1097-4547
- Volume :
- 94
- Issue :
- 11
- Database :
- MEDLINE
- Journal :
- Journal of neuroscience research
- Publication Type :
- Academic Journal
- Accession number :
- 27638598
- Full Text :
- https://doi.org/10.1002/jnr.23822