Edit the Disease - or Replace the Liver?

Trenches in Transplant Surgery · Sep 24, 2026 · 56m

Edit the Disease - or Replace the Liver? examines whether mRNA therapy, gene replacement, CRISPR and base editing could eventually prevent the need for liver transplantation.

Human studies already show that therapeutic mRNA can temporarily replace missing hepatic enzymes, while gene editors can modify hepatocytes after intravenous delivery. Early BEAM-302 results in alpha-1 antitrypsin deficiency are particularly relevant: correcting the PiZ mutation may address both toxic protein accumulation in the liver and inadequate functional protein in the circulation.

The central distinction is that…