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Clinical Grade hNSCs Rescue HD-associated Deficits

In a new STEM CELLS study, researchers led by Jihwan Song (CHA University, Gyeonggi-do, South Korea) investigated the transplantation of a clinical‐grade human neural stem cell (hNSC) line manufactured from a conditionally immortalized cell line in an animal model of Huntington's disease (HD). The hNSC line, CTX0E03, is currently in phase IIb clinical trial in the USA for patients with moderate to severe disability after ischemic stroke. Yoon et al. demonstrated that the intracerebral transplantation of hNSCs prompted significant improvements in behavioral and pathological deficits by replacing lost cells and inducing host cell regeneration mechanisms. Overall, this study provides the basis for the clinical development of these cells to treat HD patients in the future.