Scientists use gene editing for the first time in attempt to cure metabolic disease

Shane McGlaun - Nov 17, 2017
Scientists use gene editing for the first time in attempt to cure metabolic disease

A team of scientists has for the first time tried to cure a disease by editing the genes inside the person’s body. The first use of this procedure happened this week in California on a man called Brian Madeux. In the procedure, Madeux was connected to an IV and through that, he was given billions of copies of a corrective gene and a genetic tool to cut his DNA in a very precise spot.

Madeux suffers from a metabolic disease called Hunter syndrome. The scientists involved with the procedure say that signs of if the therapy is working will show for certain within three months. This sort of gene editing treatment can only be used to treat a few types of diseases.

Some therapies of this sort provide results, but those results might fade over time. The risk with therapies of this sort is inserting the new gene in the incorrect part of the DNA causing a new issue such as cancer. The team says that in the treatment that Madeux is receiving, the new gene will be placed in precisely the right location and they liken it to sending a mini surgeon along.

Unsettlingly, there is no way to go back to how the DNA inside the person was before treatment. This sort of fix is permanent. Before the procedure was tested on a human, it was tested in animals and said to have promising results. The scientists say that so far there has been no evidence that this sort of genetic manipulation will be dangerous.

This treatment won’t reverse any of the damage that Madeux has already suffered, but it might eliminate the need for weekly enzyme treatments said to cost between $100,000 and $400,000 per year. The initial studies will use 30 adults in tests, but eventually, the scientists want to use the treatment on very young children before any damage is suffered to their body.


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