The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
Published
Sept. 16, 2025, 1:15 p.m.
...). Advancing from this invention to KJ’s clinical application in under a decade is an astounding feat ...double-stranded breaks in the DNA backbone, making them significantly less prone to indel mutations that plagued CRISPR... safety of KJ’s experimental treatment, it was critical to reduce off-target editing as much as possible... ABE8e) would be tested for their base-editing efficacy in cellular models of KJ’s CPS1 mutation. Every...of identifying a successful editor. For this application, ABE8e turned out to be most effective.
Targeting...editors with increased activity and therapeutic application. Nature Biotechnology, 38(7), 892–900. https:...