The human striatum receives dopaminergic and glutamatergic inputs from different parts of the central nervous system, and is the primary input nucleus of the basal ganglia. The striatum plays key roles in coordinating various brain functions, including motivation and motor planning.
The vast majority of human striatal neurons are γ-amino butyric acid (GABAergic) projection medium spiny neurons (MSNs). These cells typically express dopamine- and cAMP-regulated phosphoprotein – DARPP32. It is these cells that specifically degenerate in the early phase of Huntington’s disease (HD) causing progressive loss of the patients’ motor and cognitive functions.
Axol has developed a medium formulation that can efficiently drive the differentiation of iPSC-derived neural stem cells to striatal neuronal lineage. The neurons derived using the medium kit are functionally active and express key markers of human MSNs, such as DARPP32, CTIP2, CALBINDIN and GABA.
By using Axol’s striatal neuron medium kit with Huntington’s disease patient-derived NSCs (ax0211), researchers can build their in vitro HD models with the disease relevant cell type (MSNs) and a real patient genetic background (with 50 GAC repeats).