We are developing human striatal medium spiny neurons (MSNs) for a cell transplantation therapy targeting Huntington’s Disease.

Huntington’s Disease is an autosomal dominant neurodegenerative disorder caused by an expanded polyglutamine repeat in the huntingtin gene. The neuropathology of HD is characterized by the decline of a specific neuronal population within the brain - the human striatal medium spiny neurons (MSNs).

We are developing these specialized human striatal medium spiny neuronal cells (MSNs) from induced pluripotent stem cells (iPSCs) as the basis for a cell transplantation-based therapy for Huntington’s Disease.