Ontology highlight
ABSTRACT:
SUBMITTER: Shen MW
PROVIDER: S-EPMC6517069 | biostudies-literature | 2018 Nov
REPOSITORIES: biostudies-literature
Shen Max W MW Arbab Mandana M Hsu Jonathan Y JY Worstell Daniel D Culbertson Sannie J SJ Krabbe Olga O Cassa Christopher A CA Liu David R DR Gifford David K DK Sherwood Richard I RI
Nature 20181107 7733
Following Cas9 cleavage, DNA repair without a donor template is generally considered stochastic, heterogeneous and impractical beyond gene disruption. Here, we show that template-free Cas9 editing is predictable and capable of precise repair to a predicted genotype, enabling correction of disease-associated mutations in humans. We constructed a library of 2,000 Cas9 guide RNAs paired with DNA target sites and trained inDelphi, a machine learning model that predicts genotypes and frequencies of 1 ...[more]