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

MOLECULAR THERAPY NUCLEIC ACIDS（2025）

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MT: RNA/DNA Editing,protein conformational disease,protein aggregation,iPSC-derived neurons,CRISPR-Cas9,adenine base editing

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