The Advances Behind The World's First Personalized CRISPR Treatment
Type
Blog Post
...Baby KJ” was diagnosed with a rare and dangerous metabolic disease shortly after his birth. Within a seven-month...successful base editing intervention was created. Only about half of babies with KJ’s disorder survive long enough...effort was only possible because of a massive collaboration between research hospitals, universities, private...an astounding feat of public investment and collaborative scientific effort.
As we like to say at Addgene...activity of adenine base editors
The first ABE was about 50% efficient in human cells on average, but its...in the lab. We’re so happy to be a part of a collaborative community focused on CRISPR research, and we...