Gene Editing Therapy: A Cure for Sickle-Cell Disease


People of African descent have long been suffering from the effects of the sickle-cell disease which is caused by a single mutation in an errant gene. Progress to find a cure has been slow until the recent emergence of gene editing therapy.

Clinical trials have shown that patients who used to suffer from the agonizing pain brought by sickle cell disease and treated at the genetic level no longer showed signs of the illness. However, it might still take a few years before the cure is approved. At present, a rarely-used dangerous and expensive bone marrow transplant is the only treatment for the disease. Gene therapy could change all these.

A welcome cure

According to Dr. Edward Benz, a professor of medicine at Harvard Medical School, it would be the first genetic cure if a common genetic disease which would mark the beginning of change for patients in less affluent minority communities inflicted with the illness.

Sickle-cell disease is most common in sub-Saharan Africa, where an estimated 70 percent of children suffering from it die before adulthood. Records also show that 300,000 infants are born with sickle-cell disease worldwide and the figure is still increasing. In the United States, 100,000 people are recorded to be suffering from the illness. 

What is sickle-cell disease?

People with sickle-cell disease have blood cells that are stuffed with hemoglobin distorted into sickle shapes which can get stuck in blood vessels and cause strokes, organ damage, and episodes of agonizing pain. These episodes are known as crises, where the muscles are starved of oxygen. For children, it is easier to return to normal between crises, but for teenagers and adults, the periods in between may cause chronic pain. The misshapen cells survive roughly 10 to 20 days in the blood compared to the usual 120 days for normal red blood cells. Patients usually suffer from anemia and are prone to infections.

Life can be a challenge

According to Dr. John Tisdale, a senior investigator at the National Institutes of Health, a lot of adults with sickle-cell disease have no health insurance, especially in states that did not expand Medicaid. Getting employed can also be difficult since the disease is debilitating.  Those who apply for Social Security disability are denied and end them up in emergency rooms when they are in crisis. For those who are capable of spending, treating the disease costs roughly $10,000 a year for children and $30,000 for adults annually.

Top of the gene editing therapy list

When gene therapy came about in the 1980s, sickle-cell disease was placed on the priority list since a small change in a single gene could relieve the pain of those who suffer from it for a lifetime. One of the three methods scientists are looking into to cure the disease is with gene editing using Crispr. This tool can let scientists snip out parts of genes and paste-in new sections. 

Bluebird Bio, a biotech company in Cambridge, Mass. has conducted gene therapy trials beginning with nine patients and is now planning a larger study involving 41 patients. This is under the consultation with the Food and Drug Administration and once they finish the study, they will proceed to get approval for the treatment in 2022.

With the pioneers of the gene therapy study showing remarkable results, we are well on our way to achieving success in curing sickle-cell disease.

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