Article

Modeling and Correction of Protein Conformational Disease in iPSC-derived Neurons through Personalized Base Editing

2024-01-18

Abstract excerpt

<h4> Abstract </h4> 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 autosomal dominant progressive myoclonic epilepsy that progresses to dementia and premature...

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Literature Corpus work: 8f391407-cdda-5436-b516-a2e156b32b60
DOI: 10.1101/2024.01.17.576134

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