Molecular BiologyMolecular Therapy — Nucleic AcidsPeer-reviewed
Modeling and Correction of Protein Conformational Disease in iPSC-derived Neurons through Personalized Base Editing
Colin T. Konishi, Nancy Mulaiese, Tanvi Butola…·Chengzu Long
This study corrects a pathogenic SERPINI1 mutation in iPSC-derived neurons using a personalized base editor to treat FENIB and related neurodegenerative diseases.
6 citations