Concise Review: Stem Cell-Based Treatment of Pelizaeus-Merzbacher Disease.

Abstract excerpt

Pelizaeus-Merzbacher disease (PMD) is an X-linked disorder caused by mutation in the proteolipid protein-1 (PLP1) gene, which encodes the proteolipid protein of myelinating oligodendroglia. PMD exhibits phenotypic variability that reflects its considerable genotypic heterogeneity, but all forms of the disease result in central hypomyelination, associated in most cases with early neurological dysfunction,...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Disease Models, Animal
• Share to PLP1 gene
• Share to Mutation