Pediatric Liver Diseases: Next-Generation Therapies

Abstract

With recent advances in molecular medicine, there has been a transformation of the landscape of pediatric liver disease therapeutics. The advent of gene editing technologies, RNA therapeutics, and molecular chaperone therapies has led to precise targeting of liver pathology. This review aims to shed light on recent breakthroughs in liver-directed nucleic acid therapies such as small interfering RNA, mRNA editing, and CRISPR-based approaches, with a special focus on their application in alpha-1 antitrypsin deficiency, hepatitis B, cystic fibrosis-related liver disease, metabolic dysfunction-associated steatotic liver disease, Alagille syndrome, progressive familial intrahepatic cholestasis, and acute hepatic porphyria.

Keywords: CRISPR; Gene editing; Molecular medicine; Nucleic acid therapy; Oligonucleotide drugs; Pediatric hepatology; RNA therapeutics.