Gene Therapy Scientists Have Started Editing Genes Inside a Human Patient

Gene Therapy Scientists Have Started Editing Genes Inside a Human Patient

The procedure of gene editing was done for the first time this Monday, on a patient that suffers of the Hunter syndrome. Until now there hasn’t been any procedure that involves gene editing inside a human body.

Hopefully this intervention would help the patient to have his DNA changed in order to cure the disease.

Results Will Be Reported in Three Months

The experiment was conducted on Brian Madeux, through an IV that contained billions of copies of a gene that could correct his DNA. He stated that he wants to take this risk and he hopes that this procedure will not only help him, but also other people.

Results will be seen in about three months and the first signs of showing if it’s working or not will appear in a month.

If it turns out to have positive results, it would definitely be a boost in gene therapy. There are already gene therapies that involve first editing the genes in labs and afterward returning them to patients. Other gene therapies don’t require editing DNA.

But these other therapies that have been used until now can only tackle certain diseases, and some have results that don’t last. Others cannot control the place where the gene is introduced into the DNA. This last issue might lead to several types of cancer.

Gene Editing is Irreversible

But now the intervention happens in a more precise way. The president of Sangamo Therapeutics, Dr. Sandy Macrae explains that they ‘cut your DNA, open it up, insert a gene, stitch it back up. Invisible mending. (…) It becomes part of your DNA and is there for the rest of your life.’ They are also trying to find a way to see how to fight with hemophilia and two metabolic diseases.

But editing something means that there is no way of going back, so it should be no room for mistakes. However, studies must go on to fight incurable diseases.

Dr. Howard Kaufman, a scientist from Boston stated that ‘Now is not the time to get scared’ and that we cannot ignore the greatness of gene editing.

Brian Madeux hopes that this treatment will prevent any future complications made by the illness: ‘I’ve been waiting for this my whole life, something that can potentially cure me.’

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