The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
Published
Sept. 16, 2025, 1:15 p.m.
...was born with a C→T mutation on one allele of his CPS1 gene, which could be repaired with an A→G edit on...base-editing efficacy in cellular models of KJ’s CPS1 mutation. Every genetic target is slightly different...