Jonathan Lee

The paper develops fully modified SpyCas9 guide RNAs that maintain or enhance genome editing efficacy in vitro and in vivo.

CRYPTID-exon identifies cryptic exons in human RNA-seq data, showing their impact on mRNA stability and gene expression through ASO targeting.

This study demonstrates the use of protecting oligos to enhance crRNA stability and potency for in vivo genome editing via AAV co-delivery in mice.