Modeling and correction of protein conformational disease in iPSC-derived neurons through personalized base editing

Modeling and correction of protein conformational disease in iPSC-derived neurons through personalized base editing

Altered protein conformation can cause incurable neurodegenerative disorders. Mutations in SERPINI1, the gene encoding neuroserpin, can alter protein conformation resulting in cytotoxic aggregation leading to neuronal death. Familial encephalopathy with neuroserpin inclusion bodies (FENIB) is a rare...

Full description

Saved in:
Bibliographic Details
Main Authors: Colin T. Konishi, Nancy Mulaiese, Tanvi Butola, Qinkun Zhang, Dana Kagan, Qiaoyan Yang, Mariel Pressler, Brooke G. Dirvin, Orrin Devinsky, Jayeeta Basu, Chengzu Long
Format: Article
Language:English
Published: Elsevier 2025-03-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
protein conformational disease
protein aggregation
iPSC-derived neurons
CRISPR-Cas9
adenine base editing
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253124003287
Tags: Add Tag
No Tags, Be the first to tag this record!

Internet

http://www.sciencedirect.com/science/article/pii/S2162253124003287

Similar Items