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A new milestone in the history of science has been created when the scientists edited the DNA inside the body of a living patient.
In a quest to cure the genetic disorder, a researcher group in Oakland from the UCSF Benioff Children’s Hospital tried out new treatment where they modified the human genome of one of the patients.
This is the first time ever in the history of science that someone has tried this gene therapy on a living patient. Earlier, they used to modify the gene in a laboratory and then re-incorporate them into the patient’s body.
Now, the researchers have taken the world by storm because every bit of modification that has been done to the DNA has happened right inside the body successfully.
Till date, there were some genetic disorders that were incurable, but this path-breaking success in the modification of DNA inside the body has opened new avenues and is believed to be the answer to many disorders related to human genes.
Is it for real?
One name that will go down the medical history is Brian Maddeux, 44 because he is the first patient on whom this trial was tried out. He received intravenous drug containing corrective genes that help in the modification of DNA when it gets linked to the genetic tool.
Zinc Finger Nucleases is the name of the tool that has succeeded the CRISPR. So, the basic work of this tool is to incise the DNA in the correct place and then replace the gene that has caused the disorder with the corrective gene. It is a complex procedure and the instructions for this work are coded into the virus and that virus targeted the liver of the patient.
Even when some enzyme goes missing or stops functioning, patients suffer from genetic disorders and this is exactly what happened to the patient. He was suffering from Hunter’s syndrome, a genetic disorder that is rare.
This rare genetic disorder is the one where the body is unable to break down the carbohydrates. It starts accumulating it, causing major damage like one’s liver size gets increased or they can suffer from hearing loss, the stiffness of joint, mental problem and breathing problem.
Scientists didn’t have any solution to this problem and the people suffering from this Hunter’s syndrome dies within 10 – 20 years. But, Maddeux was an exception. He lived beyond the time frame and when he was told about the treatment, he took the chance knowing that things might get wrong.
Maddeux was quoted saying that he wanted to take the chance because if it turned out to be successful, then it would be a great invention, especially for the kids who are suffering from this Hunter’s syndrome. Looking at the positive side, he could actually forego the risk that was involved in it and it was due to his willingness to carry out the experiment, the world has a new treatment for incurable disease too.
The Waiting game
Now that the corrective gene has been induced in the body, it is time to wait and see how the body reacts to the changes and things happening inside. This waiting game will take a minimum of three months to come out with any kind of result whether positive or negative.
If the procedure of replacing the damaged gene is not done properly, then there can be collateral damage done to the body. A minimum of three months of waiting is a must to see whether it was a success or a failure.
After three months, if the treatment proves to be a successful one, then it will be tried on several other adult patients suffering from the same disease before trying it on the children.