The “Mad” Science Making Mad Progress Toward a CGD Cure

When most people hear about scientists messing around with genetic manipulation, they think of the antagonist of a comic book trying to make super soldiers.

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You know, just your average everyday Marvel super soldiers. Source:

But, that’s not what geneticists do – I think it’s safe to say most geneticists don’t dabble in the creation of mutant armies. These scientists are working with genetic editing to find new ways to cure diseases, like Chronic Granulomatous Disease (CGD).

CGD is actually a genetic mutation in and of itself.

Because of a deficiency in the ability of certain genes to produce an enzyme that creates superoxide (sounds like something you add to your laundry to get whiter whites and brighter colors), the patient is unable to fight off some infections caused by fungi and bacteria.

Currently, doctors prescribe antibiotics to treat the symptoms of the infections rather than addressing the underlining disease. In efforts to attack the disease, the only weapons in the doctor’s arsenal are bone marrow or stem cell transplants.

The newest idea to treat patients with CGD is to use CRISPR-Cas9 technology. CRISPR-Cas9 is an innovative gene editing tool that allows scientists to repair abnormalities that were present from birth. Using this technology, geneticists have had some success in the laboratory making repairs to the NOX2 protein: NOX2 is responsible for boosting the immune system and eliminating pathogens that cause infections.

According to a study published in Science Translational Medicine, scientists used the CRISPR-Cas9 technology to repair the gene sequence in mice. The experiments showed the repair holding up over time without noticeable side effects.

This new treatment is in the early stages, but the results should give us all a ray of hope. Of course, more development will be needed before any clinical studies can be conceived.

The idea of genetic manipulation doesn’t sound so scary when it’s being used to help people with an immunodeficiency. It seems like these “mad” scientists aren’t trying to create supermen; just superoxide!

You can learn more about genetic modifications in medicine by clicking these links.

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