Modeling and Correction of Protein Conformational Disease in iPSC-derived Neurons through Personalized Base Editing
Colin T. Konishi, Nancy Mulaiese, Tanvi Butola, Qinkun Zhang, Dana Kagan, Qiaoyan Yang, Mariel Pressler, Brooke G Dirvin, Orrin Devinsky, Jayeeta Basu, 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.
Molecular Therapy — Nucleic Acids · Dec 21, 2024Peer-reviewedMolecular Biology