The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
...cause off-target effects in a variety of RNA transcripts independent of Cas9 targeting (Rees et al., 2019... be targeted (Walton et al., 2020).
The initial screen for base editors that could target KJ’s mutation...PAMs available, a large number of gRNAs could be screened for the most efficient editing. In these initial...sequence near this site is AGC, so in a secondary screen, the team tested Cas9 mutations that imposed a ...performed best when paired with the V106W mutation (described earlier) to minimize off-target editing.
Base ...