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A Comprehensive Study of AAV Tropism in the Marmoset Brain

文献类型：期刊论文

作者Fang, Kailun1,8; Liu, Hailin1; Yao, Yuting1,2; Xu, Zhen1; Liu, Xinyu1; Feng, Canbin1; Liu, Yuanhua1,3,8; Li, Tong4; Geng, Guannan1,5,7,8; Wu, Ruoxi6
刊名NEUROSCIENCE BULLETIN
出版日期2026-02-17
页码10
关键词AAV tropism Marmoset Central nervous system Intraventricular injection Intrastriatal injection Intravenous injection
ISSN号1673-7067
DOI10.1007/s12264-026-01591-9
通讯作者Yang, Hui(yanghui@simm.ac.cn) ; Gong, Neng(ngong@ion.ac.cn)
英文摘要The common marmoset has emerged as an increasingly valuable non-human primate model in neuroscience and biomedical research. Recombinant adeno-associated viruses (AAVs) are powerful tools for gene delivery and gene therapy. However, a systematic comparison of different AAV capsids and delivery routes remains lacking in marmosets. In this study, we constructed a barcoded AAV library comprising 21 capsid variants and administered it to marmosets via intravenous, intraventricular, and intrastriatal injections. To evaluate the AAV tropism, we quantified vector DNA, viral RNA abundance, and tdTomato signals in the marmoset brain and other tissues. Intravenous administration led to limited brain transduction, while intraventricular and intrastriatal administrations demonstrated high transduction efficiency in the marmoset brain. Notably, some AAV capsids exhibited distinct transduction patterns in the marmoset brain. These results offer valuable guidance for optimizing AAV-based gene delivery strategies in marmoset models and support their utility in both basic neuroscience research and potential therapeutic applications.
WOS关键词NONHUMAN PRIMATE MODEL ; GENE-TRANSFER ; MODULATION
资助项目Brain Science and Brain-like Intelligence Technology - National Science and Technology Major Project[2021ZD0200900] ; Brain Science and Brain-like Intelligence Technology - National Science and Technology Major Project[2021ZD0203900] ; Shanghai Municipal Science and Technology Major Project The pathogenesis of Alzheimer's disease and the development of related innovative drugs, Project[22ZR1481500] ; Shanghai Municipal Science and Technology Major Project The pathogenesis of Alzheimer's disease and the development of related innovative drugs, Project[23HC1401100] ; Eastern Talent Plan Project ; National Natural Science Foundation of China[32371085] ; National Natural Science Foundation of China[82021001] ; Basic Frontier Scientific Research Program of Chinese Academy of Sciences[ZDBS-LY-SM001]
WOS研究方向Neurosciences & Neurology
语种英语
WOS记录号WOS:001692534200001
出版者SPRINGER
源URL[http://119.78.100.183/handle/2S10ELR8/322987]  
专题中国科学院上海药物研究所
通讯作者Yang, Hui; Gong, Neng
作者单位1.Chinese Acad Sci, Key Lab Brain Cognit & Brain Inspired Intelligence, Ctr Excellence Brain Sci & Intelligence Technol, Inst Neurosci,Int Ctr Primate Brain Res, Shanghai 200031, Peoples R China
2.Univ Chinese Acad Sci, Beijing 101408, Peoples R China
3.Tongji Univ, Shanghai East Hosp, State Key Lab Cardiol, Sch Med, Shanghai 200120, Peoples R China
4.Chinese Acad Sci, Shanghai Inst Mat Med, Shanghai 201203, Peoples R China
5.Fudan Univ, ENT Inst, Shanghai 200031, Peoples R China
6.HuidaGene Therapeut Co Ltd, Shanghai 200131, Peoples R China
7.Fudan Univ, Eye & ENT Hosp, Dept Otorhinolaryngol, Shanghai 200031, Peoples R China
8.Shanghai Ctr Brain Sci & Brain Inspired Technol, Shanghai 201602, Peoples R China
推荐引用方式
GB/T 7714		 Fang, Kailun,Liu, Hailin,Yao, Yuting,et al. A Comprehensive Study of AAV Tropism in the Marmoset Brain[J]. NEUROSCIENCE BULLETIN,2026:10.
APA Fang, Kailun.,Liu, Hailin.,Yao, Yuting.,Xu, Zhen.,Liu, Xinyu.,...&Gong, Neng.(2026).A Comprehensive Study of AAV Tropism in the Marmoset Brain.NEUROSCIENCE BULLETIN,10.
MLA Fang, Kailun,et al."A Comprehensive Study of AAV Tropism in the Marmoset Brain".NEUROSCIENCE BULLETIN (2026):10.

入库方式： OAI收割

来源：上海药物研究所

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