Cell-Based Therapy for Canavan Disease Using Human iPSC-Derived NPCs and OPCs.

Canavan disease (CD) is a fatal leukodystrophy caused by mutation of the aspartoacylase () gene, which leads to deficiency in ASPA activity, accumulation of the substrate N-acetyl--aspartate (NAA), demyelination, and spongy degeneration of the brain. There is neither a cure nor a standard treatment for this disease. In this study, human induced pluripotent stem cell (iPSC)-based cell therapy is developed for CD.