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Aptazyme-embedded guide RNAs enable ligand-responsive genome editing and transcriptional activation

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  • Weixin Tang

    (Harvard University
    Howard Hughes Medical Institute, Harvard University
    Broad Institute of MIT and Harvard)

  • Johnny H. Hu

    (Harvard University
    Howard Hughes Medical Institute, Harvard University
    Broad Institute of MIT and Harvard)

  • David R. Liu

    (Harvard University
    Howard Hughes Medical Institute, Harvard University
    Broad Institute of MIT and Harvard)

Abstract

Programmable sequence-specific genome editing agents such as CRISPR-Cas9 have greatly advanced our ability to manipulate the human genome. Although canonical forms of genome-editing agents and programmable transcriptional regulators are constitutively active, precise temporal and spatial control over genome editing and transcriptional regulation activities would enable the more selective and potentially safer use of these powerful technologies. Here, by incorporating ligand-responsive self-cleaving catalytic RNAs (aptazymes) into guide RNAs, we developed a set of aptazyme-embedded guide RNAs that enable small molecule-controlled nuclease-mediated genome editing and small molecule-controlled base editing, as well as small molecule-dependent transcriptional activation in mammalian cells.

Suggested Citation

  • Weixin Tang & Johnny H. Hu & David R. Liu, 2017. "Aptazyme-embedded guide RNAs enable ligand-responsive genome editing and transcriptional activation," Nature Communications, Nature, vol. 8(1), pages 1-8, August.
    • Handle: RePEc:nat:natcom:v:8:y:2017:i:1:d:10.1038_ncomms15939
    DOI: 10.1038/ncomms15939
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    Cited by:

    1. Yang Liu & Filipe Pinto & Xinyi Wan & Zhugen Yang & Shuguang Peng & Mengxi Li & Jonathan M. Cooper & Zhen Xie & Christopher E. French & Baojun Wang, 2022. "Reprogrammed tracrRNAs enable repurposing of RNAs as crRNAs and sequence-specific RNA biosensors," Nature Communications, Nature, vol. 13(1), pages 1-12, December.

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