In the fourth month of the boy, researchers were meeting with the Food and Drug Administration to discuss regulatory approval for clinical trials. He was also working with the Institutional Review Board (IRB) at the Children’s Hospital in Philadelphia to achieve clinical protocols, safety and moral aspects of treatment. Researchers described the unprecedented speed of surveillance measures as “alternative procedures”.
Within five months, they began testing toxins in mice. In mice, experimental therapy corrected KJ’s variation, and changed the error in the base pair with the right GC pair in animal cells. The first dose provided 42 % of the entire liver improvement rate. At the beginning of the sixth month of KJ, researchers’ monkeys found the results of safety testing: their custom base editing therapy, which was delivered through a Lipid Nano Particle as MRNA, did not produce any toxic effects in the monkeys.
A clinical grade beach for treatment was developed. In seven months, further treatment tests revealed the genetic changes far from the lower level. Researchers submitted FDA paperwork for KJ for approval of “investigative new drugs,” or eggs. The FDA approved it within a week. The researchers then started the KJ on the treatment of immune pressure to ensure that its immune system would not react to the therapy that modify the gene. Then, when KJ was still only 6 months old, it got the first low dose of its custom gene editing therapy.
“Change”
After treatment, he was able to start eating more protein, which causes her ammonia level to cause sky cricket. But it could not be released from drug treatment that is used to keep its ammonia levels down (nitrogen skiing drugs). After the first dose, no safety concerns were seen, KJ got two more doses of gene therapy and now they are low in nitrogen squiding medicines. With more protein in his diet, he has gone from 9Third Weight 60 % 35Third Or 40Third The percentage of the percentage is now about 9 and a half months, and his doctors are preparing to allow him to go home from the hospital for the first time. Although he has to be closely monitored and he may still need liver transplantation, his family and doctors are still celebrating improvement.