A multifunctional peptide‐conjugated AIEgen for efficient and sequential targeted gene delivery into the nucleus

Gene therapy has immense potential as a therapeutic approach to serious diseases. However, efficient delivery and real‐time tracking of gene therapeutic agents haven't been solved well for successfully gene‐based therapeutics. Here we present a versatile gene delivery strategy to efficient and visualized deliver therapeutic gene into the targeted nucleus. We develop an integrin‐targeted, cell‐permeable and nucleocytoplasmic trafficking peptide‐conjugated AIEgen named TDNCP for efficient and sequential targeted delivery of antisense single‐stranded DNA oligonucleotide (ASO) and tracking the delivery process into the nucleus. Compared with TDNCP/siRNA‐NPs (siRNA functioned mainly in cytoplasm), TDNCP/ASO‐NPs (ASO functioned mainly in nucleus) exhibit better interference effect, which further testifies that TDNCP is a nucleus targeting vector. Moreover, TDNCP/ASO‐NPs show the favorable tumor suppressive effect in vivo, which demonstrates the multifunctional peptide‐conjugated AIEgen for sequential targeted gene delivery and realizes efficient gene interference therapy.

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