How Can Human Pluripotent Stem Cells Help Decipher and Cure Huntington’s Disease?

Pluripotent stem cell (PSC) technologies are becoming a key asset for deciphering pathological cascades and for developing new treatments against many neurodegenerative disorders, including Huntington’s disease (HD). This perspective discusses the challenges and opportunities facing the use of PSCs for treating HD, focusing on four major applications: namely, the use of PSCs as a substitute source of human striatal cells for current HD cell therapy, as a cellular model of HD for the validation of human-specific gene therapies, for deciphering molecular mechanisms underlying HD, and in drug discovery.