Gene Therapies in the Fight against Liver Disease: A Comprehensive Review

Abstract

Introduction: The field of hepatology is rapidly evolving, with new treatments being investigated using gene therapy. Gene therapy involves using a patient's genetic code and altering it to create a desired phenotype/genotype. Methods: Within this emerging domain, there is a multitude of delivery mediums used to alter genes. It is essential to understand the mechanisms of genetic therapies and the risks and benefits associated with them. This review aims to break down the mechanisms of action of gene therapies, describe current research conducted, and discuss future implications for the field of hepatology. Discussion and Conclusion: Different methods of gene therapy include gene editing, gene silencing, gene transfer, and mRNA therapy. These genetic modulations are achieved through several modalities. Examples include zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), cluster-regulated interspaced short palindromic repeats with associated cast 9 proteins (CRISPR/Cas9), SiRNA particles, antisense oligonucleotides (ASO), and synthetically engineered mRNA. Initially, the method for gene therapy included injecting genetic code, usually in a vector, to create phenotypic production in gene-deficient states. Recently, gene therapy has included more precise editing of the genome with technologies such as CRISPR-Cas9 and silencing of pathogenic genes.