Clinics in liver diseaseHumansGenetic TherapyLiver DiseasesGenetic VectorsGene Editing
Liver-Directed Molecular Therapies: Current and Upcoming Strategies.
Patrik Asp, Beata Malachowska, Chandan Guha, Jayanta Roy-Chowdhury
Published: 202510.1016/j.cld.2025.06.001
Abstract
Hundreds of rare monogenic metabolic liver diseases together affect 1 in 800 live births. Liver transplantation remains the only cure for many of these disorders. Liver-targeted DNA and RNA-based molecular therapies are potential alternatives, for wh…
Preview only. Read the full abstract at the source