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January 23, 2026Advanced Science0 citationsOpen Access

A Modular DNAzyme for Precise Visualization and Intervention of Alternative Splicing Isoforms in Live Cells

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MLMengru LinJSJiale SunYMYuqing Mao

Key Points

  • To develop a platform for real-time imaging and intervention of mRNA splicing isoforms in living cells.
  • Implemented a split-DNAzyme system for precise imaging of splicing events.
  • Utilized isoform-specific intron-exon junctions for high-fidelity discrimination.
  • Employed dual-site-activated fluorescence design to minimize background noise.
  • Achieved accurate imaging of closely related mRNA splicing variants.
  • Enabled real-time tracking of isoform integrity and decay.
  • Demonstrated the potential for therapeutic activation of aptamers without off-target effects.

Abstract

ABSTRACT Alternative splicing is a fundamental mechanism that enhances proteomic diversity and modulates gene function, with its dysregulation being a hallmark of numerous diseases. Despite its biological significance, the real‐time monitoring of spliced mRNA isoforms in living cells remains challenging due to limited specificity and sensitivity in existing methods. Herein, we present a Stringent dUPlex‐activated Error‐Robust (SUPER) platform, an in situ, split‐DNAzyme‐based system enabling precise imaging of mRNA splicing events in live cells. SUPER employs an identical parental DNAzyme reassembled via isoform‐specific intron‐exon junctions, providing high‐fidelity discrimination of closely related splicing variants. Its dual‐site‐activated fluorescence design ensures error‐robust, background‐minimized imaging with spatial colocalization as an intrinsic validation mechanism. Beyond dynamic isoform profiling, the programmable nature of SUPER enables its conversion into a spatially confined catalytic antenna, locally activating therapeutic aptamers without affecting off‐target transcripts. This approach further allows for real‐time tracking of variant integrity and decay by monitoring subtle changes in probe colocalization. Our platform offers a powerful tool for dissecting splicing mechanisms and holds promise for therapeutic intervention in splicing‐associated diseases by enabling isoform‐selective gene regulation while mitigating oligonucleotide toxicity.

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Cite This Study

Lin et al. (2026) studied this question.

synapsesocial.com/papers/69731047c8125b09b0d1ffa6https://doi.org/10.1002/advs.202517895
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