cross-posted from: https://slrpnk.net/post/22218016
The work, they said, began decades ago with federal funding for basic research on bacterial immune systems. That led eventually, with more federal support, to the discovery of CRISPR. Federal investment in sequencing the human genome made it possible to identify KJ’s mutation. U.S. funding supported Dr. Liu’s lab and its editing discovery. A federal program to study gene editing supported Dr. Musunuru’s research. Going along in parallel was federally funded work that led to an understanding of KJ’s disease.
Personalized gene editing isn’t new, is it? IIRC they did this successfully for sickle cell anemia or something; but it’s stupid expensive, so if you’re not crazy rich you don’t have access to this kind of healthcare.
I think its the personalized nature of the treatment, but I could be wrong.
It is. The crispr had a warm rapport with the patient and knew the parents on a first name basis.