Jayshen Arudkumar

This study shows that shortening the 4T poly-T tract in gRNA scaffolds improves transcript levels and editing efficiency, especially under low vector conditions, for both SpCas9 and SaCas9.

The paper provides a protocol for generating and characterizing mouse models of Duchenne Muscular Dystrophy using CRISPR-Cas9 and describes methods for phenotypic and molecular analysis.

The paper generates a single-copy humanized DMD mouse model by deleting a duplicated transgene to improve the accuracy of CRISPR therapy testing.

This paper develops PEA1 plasmids and PE-nuclease systems for efficient prime editing in human and mouse cells, achieving high editing efficiency in some cell lines and fetal mice.