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What is the mechanism of action of KRRO-110?

Asked by Anonymous Sep 02, 2026 0 views 1 answers
Find out What is the mechanism of action of KRRO-110 and explore helpful information and answers.
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Answered Sep 02, 2026

KRRO-110 operates by co-opting an endogenous cellular enzyme known as Adenosine Deaminase Acting on RNA (ADAR) to execute precise, targeted single-base RNA editing. Specifically engineered to treat alpha-1 antitrypsin deficiency, the oligonucleotide is designed to target the mutant Z allele variant on the SERPINA1 messenger RNA transcript. By directing the ADAR enzyme to catalyze a precise adenosine-to-inosine conversion at the specific site of the genetic mutation, the translational machinery reads the altered code correctly, restoring the secretion and function of normal, healthy wild-type alpha-1 antitrypsin protein without introducing permanent, irreversible modifications to the underlying genomic DNA.

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