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Therapeutic applications of gene editing in chronic liver diseases: an update
Ji Hyun Shin 1 (Research Assistant Professor), Jinho Lee 1 (Post doc researcher), Yun Kyung Jung 1 (Professor), Kyeong Sik Kim 1 (Professor), Jeamin Jeong 1 (Research Professor), Dongho Choi 1,* (Professor)
1Department of Surgery, Hanyang University College of Medicine, Seoul 04763, Republic of Korea,
2HY Indang Center of Regenerative Medicine and Stem Cell Research and 3Department of HY-KIST Bio-convergence, Hanyang University, Seoul 04763, Republic of Korea
Abstract
Innovative genome editing techniques developed in recent decades have revolutionized the biomedical research field. Liver is the most favored target organ for genome editing owing to its ability to regenerate. The regenerative capacity of the liver enables ex vivo gene editing in which the mutated gene in hepatocytes isolated from the animal model of genetic disease is repaired. The edited hepatocytes are injected back into the animal to mitigate the disease. Furthermore, the liver is considered as the easiest target organ for gene editing as it absorbs almost all foreign molecules. The mRNA vaccines, which have been developed to manage the COVID-19 pandemic, have provided a novel gene editing strategy using Cas mRNA. A single injection of gene editing components with Cas mRNA is reported to be efficient in the treatment of patients with genetic liver diseases. In this review, we first discuss previously reported gene editing tools and cases managed using them, as well as liver diseases caused by genetic mutations. Next, we summarize the recent successes of ex vivo and in vivo gene editing approaches in ameliorating liver diseases in animals and humans.
Abstract, Accepted Manuscript [Submitted on February 22, 2022, Accepted on April 22, 2022]
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