A Multifunctional Peptide-Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus
Yong Cheng1,2, Chunli Sun2, Rui Liu1
1Engineering Research Center of Nano-Geomaterials of the Ministry of Education, Faculty of Materials Science and Chemistry, China University of Geosciences, Wuhan, 430074, China.
Abstract:
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 TD NCP 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 TD NCP/siRNA-NPs (siRNA functions mainly in the cytoplasm), TD NCP/ASO-NPs (ASO functions mainly in the nucleus) exhibited a better interference effect, which further indicates that TD NCP is a nucleus-targeting vector. Moreover, TD NCP/ASO-NPs showed a favorable tumor-suppressive effect in vivo.
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