Article
Precisely defining disease variant effects in CRISPR-edited single cells.
Nature - 1 Oct 2025
Baglaenko Yuriy, Mu Zepeng, Curtis Michelle, Mire Hafsa M, Jayanthi Vidyashree, Al Suqri Majd, Liu Cassidy, Agnew Ryan, Nathan Aparna, Mah-Som Annelise Yoo, Liu David R, Newby Gregory A, Raychaudhuri Soumya
Abstract excerpt
Genetic studies have identified thousands of individual disease-associated non-coding alleles, but the identification of the causal alleles and their functions remains a critical bottleneck1. CRISPR-Cas editing has enabled targeted modification of DNA to introduce and test disease alleles. However, the combination of inefficient editing, heterogeneous editing outcomes in individual cells and nonspecific...
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