In the fourth month of the boy, the researchers met with the Food and Drug Administration to discuss organizational approval on a clinical trial – an experiment where KJ would be the only participant. They were also working with the institutional audit council (IRB) at the Children’s Hospital in Philadelphia to overcome the clinical protocol, safety and moral aspects of treatment. The researchers described the unprecedented speed of control steps as “through alternative procedures.”
In the fifth month, they started testing toxins in mice. In the mice, the experimental therapy corrected the KJ mutation, and replaced the sinner in the primary husband with the right GC’s husband in animal cells. The first dose provided 42 percent a full complete average in animals. At the beginning of the sixth month of KJ, the researchers had results from the monkeys safety test: the treatment of the liberalization of the designated base, which was delivered as a flexible via oily nanoparticles, was not caused by any toxic effects in the monkeys.
A clinical batch of treatment is prepared. In the seventh month, he found more tests for treatment, which are acceptable to genetic changes outside the target. The researchers presented paper works in the Food and Drug Administration to agree to a “new investigative property” or IND, to KJ. The Food and Drug Administration (FDA) agreed within a week. Then the researchers KJ began an immunosuppressive treatment to ensure that its immune system will not interact with the treatment of genes. Then, when KJ was still only 6 months old, he got a low dose of treating allocated genes.
“Transformation”
After treatment, he was able to start taking more protein, which might cause ammonia levels. But it was not possible to be lost from the treatment of drugs used to maintain ammonia levels (nitrogen that spoils). With no safety concerns after the first dose, KJ has since obtained two other doses of genetic therapy and is now on low nitrogen drugs. With more protein in its diet, move from 9Y In percentage to 35Y Or 40Y Celsius. He is about 9 and a half months old, and his doctors are preparing to allow him to return home from the hospital for the first time. Although he will have to be closely monitored and still at some point need to be a liver transplant, his family and doctors celebrate improvements so far.
