Broad brain biodistribution conferred by an AAV to restore TDP-43 function mitigates Frontotemporal Demenia-like deficits
通过腺相关病毒(AAV)实现广泛的脑生物分布,从而恢复TDP-43功能,减轻额颞叶痴呆样症状。
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doi:10.1101/2025.08.28.672900
Cao, Tianyu; Thapa, Rashmi; Liu, Rongsong; Mallika, Aswathy Peethambaran; Baghel, Meghraj Singh; Wei, Yijun; Sinha, Irika R; Burns, Grace D; Wen, Xinrui; Pang, Bo; Ling, Jonathan P; Lin, Da-Ting; Li, Yun; Wong, Philip C