Abstract
Huntington’s disease (HD) is an autosomal dominantly inherited neurodegenerative disorder characterized by a profound loss of striatal neurons in the basal ganglia. Patients suffer from a range of motor, cognitive and psychiatric symptoms, with no cure to date. Therefore, the generation of reliable physiologically relevant functional striatal neurons in vitro is fundamental to advance in the research of the HD field.
At Axol Bioscience, we have developed human induced pluripotent stem cells (hiPSCs)-derived striatal neurons from healthy individuals and a 125 CAG HD patient. The current bank of HD iPSCs is now 143 CAG repeats because of its time in culture. We confirmed that both lines express key striatal neuron markers at RNA and protein level after 20 days in maturation.
