The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
...of off-target DNA editing and on-target indel formation.
For the safety of KJ’s experimental treatment...cellular models of KJ’s mutation, SpG Cas9 + ABE8e performed the best when paired with the highest-efficiency...produced efficient editors, the Kleinstiver set performed best when paired with the V106W mutation (described... assessed for off-target risks, such a modular platform could accelerate the development of genetic therapies...