Gene therapy has immense potential as a therapeutic approach to serious diseases. However, efficient delivery and real-time tracking of gene therapeutic agents have not been solved well for successful gene-based therapeutics. Herein we present a versatile gene-delivery strategy for efficient and visualized delivery of therapeutic genes into the targeted nucleus. We developed an integrin-targeted, cell-permeable, and nucleocytoplasmic trafficking peptide-conjugated AIEgen named TDNCP for the efficient and sequential targeted delivery of an antisense single-stranded DNA oligonucleotide (ASO) and tracking of the delivery process into the nucleus. As compared with TDNCP/siRNA-NPs (siRNA functions mainly in the cytoplasm), TDNCP/ASO-NPs (ASO functions mainly in the nucleus) exhibited a better interference effect, which further indicates that TDNCP is a nucleus-targeting vector. Moreover, TDNCP/ASO-NPs showed a favorable tumor-suppressive effect in vivo.
基金：
National Natural Science Foundation of ChinaNational Natural Science Foundation of China (NSFC) [21525523, 21722507, 21574048, 21874121, 21703210]; National Basic Research Program of China (973 Program)National Basic Research Program of China [2015CB932600]; National Key R&D Program of China [2017YFA0208000, 2016YFF0100800]; China Postdoctoral Science FoundationChina Postdoctoral Science Foundation [2017M620309, 2017M610492]
第一作者单位：[1]China Univ Geosci, Fac Mat Sci & Chem, Engn Res Ctr Nanogeomat, Minist Educ, Wuhan 430074, Hubei, Peoples R China;[2]Huazhong Univ Sci & Technol, Tongji Med Coll,Sch Mat Sci & Engn, Tongji Hosp,State Key Lab Mat Proc & & Mould Tech, Sch Chem & Chem Engn,Dept Obstet & Gynecol,Hubei, Wuhan 430074, Hubei, Peoples R China
通讯作者：
通讯机构：[1]China Univ Geosci, Fac Mat Sci & Chem, Engn Res Ctr Nanogeomat, Minist Educ, Wuhan 430074, Hubei, Peoples R China;[2]Huazhong Univ Sci & Technol, Tongji Med Coll,Sch Mat Sci & Engn, Tongji Hosp,State Key Lab Mat Proc & & Mould Tech, Sch Chem & Chem Engn,Dept Obstet & Gynecol,Hubei, Wuhan 430074, Hubei, Peoples R China
推荐引用方式(GB/T 7714)：
Cheng Yong,Sun Chunli,Liu Rui,et al.A Multifunctional Peptide-Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus[J].ANGEWANDTE CHEMIE-INTERNATIONAL EDITION.2019,58(15):5049-5053.doi:10.1002/anie.201901527.
APA：
Cheng, Yong,Sun, Chunli,Liu, Rui,Yang, Juliang,Dai, Jun...&Xia, Fan.(2019).A Multifunctional Peptide-Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus.ANGEWANDTE CHEMIE-INTERNATIONAL EDITION,58,(15)
MLA：
Cheng, Yong,et al."A Multifunctional Peptide-Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus".ANGEWANDTE CHEMIE-INTERNATIONAL EDITION 58..15(2019):5049-5053
化学