SUPT4H1-Edited Stem Cell Therapy Rescues Neuronal Dysfunction in a Mouse Model for Huntington’s Disease

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Transplanting SUPT4H1-edited Huntington’s disease (HD)-induced pluripotent stem cell-derived neural precursor cells (iPSC-NPCs) into the YAC128 HD transgenic mouse model improved motor function compared to unedited HD iPSC-NPCs.
[NPJ Regenerative Medicine]
Abstract