RNA Editing Revolutionizes Genetic Disorder Treatments
Summary: Wave Life Sciences has pioneered RNA editing in clinical trials, marking a significant step in genetic disorder treatments. RNA editing corrects mRNA errors, restoring functional protein production through enzymes like ADAR and guided RNA. This approach treats conditions such as α-1 antitrypsin deficiency and could extend to diseases like Huntington’s. Unlike DNA editing, RNA editing is reversible, minimizing risks. Challenges include specificity and delivery methods, but ongoing advancements and interest from major pharmaceuticals indicate RNA editing’s potential as a revolutionary gene therapy standard.
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