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CRISPR Gene Editing To Correct Mutations In Patients With Immunodeficiency

Update Date: Jan 14, 2017 09:00 AM EST
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A team of scientists is using the CRISPR-Cas9 gene editing technology to correct mutations in the blood stem cells of patients suffering from X-linked chronic granulomatous disease, a rare immunodeficiency disorder.

The study, published in Science Translational Medicine, is one of the many promising discoveries from the powerful CRISPR-Cas9 technology. This could pave the way to the discovery of potential therapeutic applications for hereditary diseases. The gene editing tool also shows promise in the process called ex vivo gene therapy, wherein disease-causing mutations in the cells of patients are edited or tweaked at the laboratory and implanted back into the body.

One of the diseases the scientists are hoping would be cured by the gene editing tool is chronic granulomatous disease (CGD), a hereditary disorder with limited treatment options today. This disease is associated with weakened immune systems leading to life-threatening infections.

What Is Chronic Granulomatous Disease (CGD)?

According to Medscape, chronic granulomatous disease (CGD) is a rare disease caused by mutations in any one of the five components of the nicotinamide adenine dinucleotide phosphate (NADPH) oxidase in phagocytes. This important enzyme creates superoxide that is important for intracellular killing of pathogens by phagocytes.

When a person has this disease, the phagocytes are unable to kill bacteria and fungi. Hence, this condition makes the patient prone to infections, which is termed as immunodeficiency. It may lead to recurrent or persistent intracellular bacterial and fungal infections.

Today, the only treatment given to patients are antibiotics to treat the disease and prevent infections. The only promising cure is bone marrow or stem cell transplant. That's why researchers are looking for ways to use gene editing in the hopes to create better therapies for this rare disease.

The Promise Of CRISPR Technology

The team of scientists applied an ex vivo gene editing approach using the CRISPR-Cas9 platform to CGD. Since CGD is caused by defects in the NOX2 protein, a key molecule that helps the immune system to kill pathogens, they tried to repair the NOX2 mutation in stem cells from CGD patients.

When planted into mice, the pool of altered immune cells maintained their gene edits long-term, with no signs of side effects. If the process will be further developed, the gene editing tool may offer a new clinical strategy for the treatment of CGD and possibly, other blood disorders, Science Daily reports.

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