BioengineeringbioRxivPreprint
Preventing large deletions and chromosome loss in engineered human primary T cells by CasPlus with optimized guide RNAs
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NYU Langone HealthORCID 0000-0002-2353-6399Unclaimed ORCID profile · follow to get notified of new preprints
3 papers
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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.
Zhenzhen Chen, Jian‐Ying Wang, Yu Kang…·Yuyu Niu
The paper describes the generation of PINK1 mutant cynomolgus monkeys using paired truncated sgRNA/Cas9-D10A to create a primate model of Parkinson's disease without affecting PINK1 protein expression.