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• W4385368859 abstract "Gene therapy is a potential approach to deal with complicated diseases that are now untreatable. A critical step in the effective gene therapy is the successful delivery of therapeutic genes into target cells. The fast development of gene regulation toolboxes and diverse gene vectors greatly increase the likelihood of delivering functional gene modules to multiple organs and expand the therapeutic scope of complex diseases. In the design of the optimal gene vectors for therapeutic applications, the gene loading capacity, shape, size, and safety are critical factors to be considered. In this chapter, the design, construction, function, applications, and future prospects of a new type of nonviral gene nanovector with Archimedes solid-like truncated octahedron nanostructures (ASN-TO) constructed from polymerase chain reaction based on branched primers (branch-PCR) were described. ASN-TO gene nanovector exhibits its unique feature in the improvement of high DNA loading capacity, serum stability, particle size controllability, and long-lasting bioactivity, especially multiplex gene regulation. ASN-TO gene nanovector offers us new opportunity to develop the strategy of genome therapy through integrating various kinds of gene regulation toolboxes (gene overexpression, gene silencing, gene editing, etc.) as an all-in-one vector, fine-tuning gene expression in multiple levels and synergistically adjusting network targets in multiple signaling pathways, which is different from single gene therapy and would potentially expand therapeutic applications. For complex diseases involving multiple genes, ASN-TO gene nanovector would hopefully act as a chromosome-like payload to carry diverse artificially designed gene regulation toolboxes to synergistically regulate the network targets for genome therapy." @default.
• W4385368859 created "2023-07-29" @default.
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• W4385368859 date "2023-01-01" @default.
• W4385368859 modified "2023-10-16" @default.
• W4385368859 title "Gene Nanovector for Genome Therapy" @default.
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• W4385368859 doi "https://doi.org/10.1007/978-981-19-9776-1_60" @default.
• W4385368859 hasPublicationYear "2023" @default.
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