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mRNA and gene editing: Late breaking therapies in liver diseases

4 Mar 2022

Abstract excerpt

The efficient delivery of RNA molecules to restore the expression of a missing or inadequately functioning protein in a target cell and the intentional specific modification of the host genome using engineered nucleases represent therapeutic concepts that are revolutionizing modern medicine. The initiation of several clinical trials using these approaches to treat metabolic liver disorders as well as the recently...

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PMID: 35243655
DOI: 10.1002/hep.32441

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