BEACON TRANSCRIPT – Gene editing is not a novel technique, as many researchers have been trying it in plenty of cases. However, they have recently made a unique attempt at editing the DNA of a man directly inside his body. Between one and three months afterwards, they will check if they managed to alter the man’s genetic code, thus finding out if the solution can be used as an effective treatment against diseases.
The gene editing took place inside the man’s body
The patient in question is Brian Madeux, 44, who is suffering from Hunter syndrome. This disease is characterized by one missing gene in those cells in the liver which break down molecules. As a result, he might easily suffer infections and other complications, such as heart problems.
Researchers from Sangamo Therapeutics prepared a concoction which Madeux had to drink, and then its compounds could edit his DNA and eventually add the missing gene. If the process is successful and the gene manages to be inserted in the DNA, then it should remain there for the rest of the man’s life.
However, researchers need to be careful. As mentioned above, once the gene editing is performed, the process is permanent. Therefore, if they make a mistake, it will stay there forever. Even so, Madeux agreed to undergo the procedure. After 26 surgeries, he thought the risk was worth taking.
The process makes use of a technique similar to CRISPR
Doctors and researchers have tried gene editing on people even before. However, during other attempts, they took cells from a patient’s body, using the gene-editing technique CRISPR to modify them outside the body, and then put them back in. In this case, the researchers have edited the cells directly inside the patient’s body.
Instead of CRISPR, the gene editing is performed by zinc finger nucleases. The process works just like the other one, meaning that it cuts DNA and inserts the necessary gene where it’s needed. This procedure is indeed risky but, if it works, it can cure diseases when other types of therapy were not effective.
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