Cas12a chRDNA-mediated in vivo genome editing for high specificity functional gene disruption
CRISPR hybrid guides containing a combination of RNA and DNA nucleotides (CRISPR hybrid RNA-DNA or chRDNA) enhance both Cas9 and Cas12a nuclease specificity and reduce off-target editing in vitro. CRISPR-Cas9 with all-RNA crRNA guides has been implemented for in vivo intervention of familial hypercholesterolemia, an in...
M. Losa, Prachi Rajmane, Morena Stanaway et al.
· Molecular Therapy: Nucleic A... · 0 citations