Philadelphia, Pennsylvania-(map)
Scientists made the first gene-Enn the medicine made for a single patient. Patient -A young baby in a sick due to one’s own problem DNA -It is now greatly improved and is expected to return home soon.
Immediately after his birth, doctors knew that KJ MULDOON was having a difficult time. According to the test, KJ’s DNA has made it difficult for the body to remove the dangerous chemicals of ammonia. Too many ammonia can damage the brain and other organs. About half of the children born with this problem still die while having a baby.
(source: chop.))
When the human body digests protein, ammonia is created. The liver of a healthy person helps to remove ammonia. But because of his problem geneKJ’s liver could not remove ammonia.
KJ was so young and small transplantationWaiting for transplants would have been very dangerous. The doctors of the Children’s Hospital in Philadelphia asked KJ’s parents if they wanted to try a new method, a special medicine made for KJ. His parents said so.
DNA editing
DNA is a collection of information and guidelines stored in small chemical forms. DNA tells us what to do with cells and body building blocks. All living things have a DNA that grows in certain ways.
A small DNA, called the “gene,” helps control other parts of the way the creature grows. For example, some genes can control the color of the eye. Some genes work themselves and other genes work together. Some people can help control more than one.
In recent years, scientists have greatly advanced their ability to “edit” DNA. CRISPR is a tool for scientists to cut or change DNA pieces. DNA editing is tricky and complicated.
(Source: SMEDLIB, At Wikimedia Commons.))
Scientists at Penn Medicine were studying the idea of making medicines to fix the genes of a single patient. Now they had a chance to try it. But because I didn’t have much time to help KJ, the pressure was applied.
Researchers studied KJ’s DNA. The problem was that 3 billion letters were fixed with a long word. Many scientists have developed CRISPR tools to edit KJ’s DNA, as well as helping many scientists to pitch and work for a long time.
(source: chop.))
Next, they tested new drugs of mice and monkeys. The result was positive -new treatment helped animals and looked safe. New medicine in 6 months FDA.
During this period, KJ was receiving special treatment and special treatment to help lower the level of ammonia. KJ is six months when the first minority of the new drug is gained, and it is designed to reach the liver and make a small change in DNA.
(source: chop.))
Immediately after the first treatment, KJ was able to eat protein without raising the ammonia level of the body. As a result, doctors gave KJ two more doses. KJ has a weight gain and is now much healthier. Soon the hospital expects him to return home for the first time in his life.
KJ is not completely treated. He may have to continue taking medicine and one day he needs liver transplantation. But he looks much better when he is born.
(source: chop.))
Scientists and doctors are very excited about the results. In just six months, the researchers and the company they worked together were able to create a special DNA editor for a single patient. In the past, it would have been more than 10 years. This project is not just KJ’s success. It also brings hope to millions of others with conditions that can be treated in a similar way.
Do you know… ?
KJ treatment is possible Decades A study paid by the US government. One of the relevant scientists, Fyodor Urnov, said, “I don’t think this can happen in other countries other than the United States.” Some of the related companies helped me for free.
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