Conditional Deoxyribozyme-Nanoparticle Conjugates for miRNA-Triggered Gene Regulation.

ABSTRACT: DNA-nanoparticle (NP) conjugates have been used to knockdown gene expression transiently and effectively, making them desirable tools for gene regulation therapy. Because DNA-NPs are constitutively active and are rapidly taken up by most cell types, they offer limited control in terms of tissue or cell type specificity. To take a step toward solving this issue, we incorporate toehold-mediated strand exchange, a versatile molecular programming modality, to switch the DNA-NPs from an inactive state to an active state in the presence of a specific RNA input. Because many transcripts are unique to cell subtype or disease state, this approach could one day lead to responsive nucleic acid therapeutics with enhanced specificity. As a proof of concept, we designed conditional deoxyribozyme-nanopar

SUBMITTER: Zhang J 

PROVIDER: S-EPMC8287654 | biostudies-literature | 2020 Aug

REPOSITORIES: biostudies-literature