EMBRYONIC DEVELOPMENT & STEM CELL COMPENDIUM
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Pelizaeus-Merzbacher Disease
 - Cell Therapy Approaches

Pelizaeus–Merzbacher disease (PMD) is a congenital hypomyelination disorder that belongs to a group of genetic disorders called leukodystrophies that affect myelin sheath growth. PMD is caused by a mutation in the myelin protein proteolipid protein 1 (PLP1).

Cell Therapy Approaches


The objective of PMD cell therapy is to achieve myelin sheath repair. Glial progenitor cells (GPCs), which can give rise to new myelinogenic oligodendrocytes, are potential therapeutic cells for the restoration of myelin to the hypomyelinated or dysmyelinated central nervous system axons.

Pelizaeus-Merzbacher Disease