| abstract |
Provided herein are methods for inducing CRISPR/Cas-based gene regulation (eg, genome editing or gene expression) of a target nucleic acid (eg, target DNA or target RNA) in a cell. The methods include the use of modified single guide RNAs (sgRNAs) that enhance gene regulation of target nucleic acids in primary cells for ex vivo treatment or in subject cells for ex vivo treatment. Additionally, provided herein are methods of preventing or treating a genetic disease in a subject by administering a sufficient amount of the modified sgRNA to correct a mutation in a target gene associated with the genetic disease. |