Comparative evaluation of hepatic-targeted knock-in strategies using viral and nonviral vector combinations in mouse inherited disease models Article Swipe
Xiu Jin
,
Xiaoyi Wu
,
Junwei Song
,
Mei Luo
,
Qin Ye
,
Chengda Ren
,
Song Li
,
Manjun Li
,
Ming Hu
,
Yifan An
,
Jing Su
,
Jiamei Fu
,
Qiuxia Xu
,
Min Luo
,
Fanfei Liu
,
Man Liu
,
Qiqi Li
,
Shaohua Yao
,
Lu Chen
,
Yang Yang
·
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.1016/j.ymthe.2025.12.012
YOU?
·
· 2025
· Open Access
·
· DOI: https://doi.org/10.1016/j.ymthe.2025.12.012
CRISPR-Cas9-mediated gene knock-in has emerged as a promising strategy for early-onset genetic disease intervention. However, the therapeutic efficacy and editing outcomes of different knock-in strategies remain incompletely understood. Here, we systematically evaluated three major liver-directed knock-in strategies, namely, homology-directed repair (HDR), homology-independent targeted integration (HITI), and homology-mediated end joining (HMEJ), using neonatal mouse models of mucopolysaccharidosis type I and hemophilia B. Although all three approaches effectively rescued disease phenotypes, we observed distinct editing outcomes. Notably, the HMEJ approach, delivered via a combined adeno-associated virus-lipid nanoparticle (AAV-LNP) system, exhibited superior integration efficiency (5.8-5.9%) and fidelity (97-98%) compared with HDR and HITI. In contrast, whole-genome sequencing indicated that HITI induced a higher risk of random AAV donor integration than HDR or HMEJ. Furthermore, long-read sequencing analyses revealed that the frequencies of inverted terminal repeat (ITR)-mediated transgene integration differed between the 5' and 3' genomic junctions among the three strategies. Specifically, in HDR- and HMEJ-treated mice, ITR-mediated integration events were 7.7-19.7-fold more common at the 3' junctions than at the 5' junctions. These findings highlight the comprehensive advantages of the AAV-LNP-mediated HMEJ approach for liver-directed knock-in therapy and suggest its strong potential for clinical translation.
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- OA Status
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https://openalex.org/W4417187917Canonical identifier for this work in OpenAlex
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https://doi.org/10.1016/j.ymthe.2025.12.012Digital Object Identifier
- Title
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Comparative evaluation of hepatic-targeted knock-in strategies using viral and nonviral vector combinations in mouse inherited disease modelsWork title
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articleOpenAlex work type
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enPrimary language
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2025Year of publication
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2025-12-01Full publication date if available
- Authors
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Xiu Jin, Xiaoyi Wu, Junwei Song, Mei Luo, Qin Ye, Chengda Ren, Song Li, Manjun Li, Ming Hu, Yifan An, Jing Su, Jiamei Fu, Qiuxia Xu, Min Luo, Fanfei Liu, Man Liu, Qiqi Li, Shaohua Yao, Lu Chen, Yang YangList of authors in order
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https://doi.org/10.1016/j.ymthe.2025.12.012Publisher landing page
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YesWhether a free full text is available
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hybridOpen access status per OpenAlex
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https://doi.org/10.1016/j.ymthe.2025.12.012Direct OA link when available
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0Total citation count in OpenAlex
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