Self-inactivating AAV-CRISPR at different ages enables sustained amelioration of Huntington's disease deficits in BAC226Q mice
Yuanyi Dai, Zuliayeti Abudujielili, Yunyi Ding, Wanping Huang, Jianhang Yin, Liqiong Ou, Jiazhi Hu, Sushuang Zheng, Chenjian Li
Self-inactivating CRISPR targeting mutant huntingtin in HD mice achieved long-term rescue of neural pathology, motor deficits, weight loss and survival.
Science Advances · Mar 18, 2026Peer-reviewedNeuroscience