The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
Published
Sept. 16, 2025, 1:15 p.m.
...). The Heroes of CRISPR. Cell, 164(1), 18–28. https://doi.org/10.1016/j.cell.2015.12.041
Musunuru, K....birth. Within a seven-month whirlwind of developing cell and mouse model systems, testing base editing variants...reporting genome editing by CRISPR in mammalian cells appeared in 2013 (Cong et al., 2013; reviewed in...
The first ABE was about 50% efficient in human cells on average, but its effectiveness varied depending... the chromosomal location, sequence context, and cell type. Multiple labs worked to improve its effectiveness...would be tested for their base-editing efficacy in cellular models of KJ’s CPS1 mutation. Every genetic target...base editors that could target KJ’s mutation in cellular models tested both the SpG and SpRY variants of...