The myelin mutants as models to study myelin repair in the leukodystrophies.

ABSTRACT: The leukodystrophies are rare and serious genetic disorders of the central nervous system that primarily affect children who frequently die early in life or have significantly delayed motor and mental milestones that result in long-term disability. Although with some of these disorders, early intervention with bone marrow or cord blood transplantation has been proven useful, it has not yet been determined that such therapies promote myelin repair of the central nervous system. Research on experimental therapies aimed at myelin repair is aided by the ability to test cell replacement strategies in genetic models in which the mutations and neuropathology match the human disorder. Thus, models exist of Pelizaeus-Merzbacher disease and the lysosomal storage disorder, Krabbe disease, which refle

SUBMITTER: Duncan ID 

PROVIDER: S-EPMC3250297 | biostudies-literature | 2011 Oct

REPOSITORIES: biostudies-literature