Development of a Tethered mRNA Amplifier to increase protein expression.

Biotechnol J

Department of Molecular Biology and Genetics and Department of Biology, Johns Hopkins University, Baltimore, Maryland, USA.

Published: October 2022

Herein, we present a novel method to specifically increase a messenger RNA's (mRNA) expression at the post-transcriptional level. This is accomplished using what we term a "Tethered mRNA Amplifier." The Tethered mRNA Amplifier specifically binds an mRNA's 3' untranslated region and enhances its stability/translation, often doubling protein output. We test this approach on several transcripts associated with haploinsufficiency disorders and increase their steady-state expression in cell culture. We suggest this approach may be a tenable therapeutic modality with precise activity and broad-spectrum application.

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http://dx.doi.org/10.1002/biot.202200214DOI Listing

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