Cell therapy in Huntington's disease: Taking stock of past studies to move the field forward.

ABSTRACT: Huntington's disease (HD) is a rare inherited neurodegenerative disease that manifests mostly in adulthood with progressive cognitive, behavioral, and motor dysfunction. Neuronal loss occurs predominantly in the striatum but also extends to other brain regions, notably the cortex. Most patients die around 20 years after motor onset, although there is variability in the rate of progression and some phenotypic heterogeneity. The most advanced experimental therapies currently are huntingtin-lowering strategies, some of which are in stage 3 clinical trials. However, even if these approaches are successful, it is unlikely that they will be applicable to all patients or will completely halt continued loss of neural cells in all cases. On the other hand, cellular therapies have the potential to r

SUBMITTER: Bachoud-Levi AC 

PROVIDER: S-EPMC10234449 | biostudies-literature | 2021 Feb

REPOSITORIES: biostudies-literature