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Article

RGEN Editing of RNA and DNA: The Long and Winding Road from Catalytic RNAs to CRISPR to the Clinic.

Cell - 28 May 2020

Sullenger Bruce A

Abstract excerpt

The first clinical studies utilizing RNA-guided endonucleases (RGENs) to therapeutically edit RNA and DNA in cancer patients were recently published. These groundbreaking technological advances promise to revolutionize genetic therapy and, as I discuss, represent the culmination of decades of innovative work to engineer RGENs for such editing applications.

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Identifiers

PMID: 32470403
DOI: 10.1016/j.cell.2020.04.050

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