MRNA and gene editing: Late breaking therapies in liver diseases.

ABSTRACT: 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 reported remarkable results obtained by patients with transthyretin amyloidosis highlight the advances in this field and show the potential of these therapies to treat these diseases safely and efficaciously. These advances have been possible due, firstly, to significant improvements made in RNA chemistry that increase its stability and prevent activation of the innate immune resp

SUBMITTER: Zabaleta N 

PROVIDER: S-EPMC9546265 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature