The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
Published
Sept. 16, 2025, 1:15 p.m.
...shortly after his birth. Within a seven-month whirlwind of developing cell and mouse model systems, testing...backbone, making them significantly less prone to indel mutations that plagued CRISPR editing at the time...off-target effects in a variety of RNA transcripts independent of Cas9 targeting (Rees et al., 2019). The Liu... levels of off-target DNA editing and on-target indel formation.
For the safety of KJ’s experimental treatment...McCague, S., Burke, S. M., Wright, C. M., Bick, S., Indurthi, V., Sharma, S., … Ahrens-Nicklas, R. C. (2025...