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Therapeutic applications of gene editing in chronic liver diseases: an update

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초록

Innovative genome editing techniques developed in recent decadeshave revolutionized the biomedical research field. Liveris the most favored target organ for genome editing owing toits ability to regenerate. The regenerative capacity of the liverenables ex vivo gene editing in which the mutated gene inhepatocytes isolated from the animal model of genetic diseaseis repaired. The edited hepatocytes are injected back into theanimal to mitigate the disease. Furthermore, the liver is consideredas the easiest target organ for gene editing as it absorbsalmost all foreign molecules. The mRNA vaccines, which havebeen developed to manage the COVID-19 pandemic, haveprovided a novel gene editing strategy using Cas mRNA. Asingle injection of gene editing components with Cas mRNA isreported to be efficient in the treatment of patients with geneticliver diseases. In this review, we first discuss previouslyreported gene editing tools and cases managed using them, aswell as liver diseases caused by genetic mutations. Next, wesummarize the recent successes of ex vivo and in vivo geneediting approaches in ameliorating liver diseases in animalsand humans.

Abstract

Innovative genome editing techniques developed in recent decadeshave revolutionized the biomedical research field. Liveris the most favored target organ for genome editing owing toits ability to regenerate. The regenerative capacity of the liverenables ex vivo gene editing in which the mutated gene inhepatocytes isolated from the animal model of genetic diseaseis repaired. The edited hepatocytes are injected back into theanimal to mitigate the disease. Furthermore, the liver is consideredas the easiest target organ for gene editing as it absorbsalmost all foreign molecules. The mRNA vaccines, which havebeen developed to manage the COVID-19 pandemic, haveprovided a novel gene editing strategy using Cas mRNA. Asingle injection of gene editing components with Cas mRNA isreported to be efficient in the treatment of patients with geneticliver diseases. In this review, we first discuss previouslyreported gene editing tools and cases managed using them, aswell as liver diseases caused by genetic mutations. Next, wesummarize the recent successes of ex vivo and in vivo geneediting approaches in ameliorating liver diseases in animalsand humans.

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