Molecular therapy : the journal of the American Society of Gene Therapy
Comparative evaluation of liver-directed knockin strategies with viral and nonviral vectors in mouse inherited disease models.
Xiu Jin, Xiaoyi Wu, Junwei Song, Mei Luo, Qin Ye, Chengda Ren, Li Song, Manjun Li, Ming Hu, Yifang An, Jing Su, Jiamei Fu, Qiuxia Xu, Min Luo, Fanfei Liu
Published: 202510.1016/j.ymthe.2025.12.012
Abstract
CRISPR-Cas9-mediated gene knockin has emerged as a promising strategy for early-onset genetic disease intervention. However, the therapeutic efficacy and editing outcomes of different knockin strategies remain incompletely understood. Here, we system…
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