Scientific Frontline
Base editing, a highly precise gene-editing technology, has successfully corrected the PEX1 genetic mutation responsible for Zellweger spectrum disorder in a mouse model, significantly restoring liver and peroxisome function.
#MolecularGenetics #BiomedicalEngineering #GeneTherapy #Genetics #sflorg
https://www.sflorg.com/2026/04/gen04142601.html
Base editing repairs mutation and liver function in mouse model of Zellweger spectrum disorder

The base editing enzyme used in this study is the same one that fixed Baby KJ Muldoon’s disease-causing mutation and saved his life in 2025.

Apr 14 at 7:35pmWeb