Title:A Multifunctional Peptide-Conjugated AIEgen for Efficient and Sequential Targeted Gene Delivery into the Nucleus
Author(s):Cheng, Yong; Sun, Chunli; Liu, Rui; Yang, Juliang; Dai, Jun; Zhai, Tianyou; Lou, Xiaoding*; Xia, Fan*
Addresses:, China Univ Geosci, Fac Mat Sci & Chem, Engn Res Ctr Nanogeomat, Minist Educ, 388 Lumo Rd, Wuhan 430074, Hubei, Peoples R China
Published:14 February 2019
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 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.
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