Can Gene Editing Cure Sickle Cell?

01/01/2024
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    This electron microscope image shows a blood cell altered by sickle cell disease, top. (National Center for Advancing Translational Sciences, National Institutes of Health via AP)
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    Victoria Gray takes part in a gene editing trial for sickle cell disease. Gray was the first patient to test the treatment. She described feeling like she “was being reborn” the day she got the gene therapy. (Anthem Pictures/Sarah Cannon Research Institute via AP)
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    Crescent-shaped red blood cells from a sickle cell disease patient (Dr. F. Gilbert/CDC via AP)
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    Jalen Matthews was diagnosed with sickle cell at birth. When she was 12, the disease led to a spinal cord stroke that left her with some paralysis in her left arm and leg. She had to “basically learn how to do everything all over again,” says Matthews, now 26. (AP/Timothy D. Easley)
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    The sunset reflects off the Vertex Pharmaceuticals headquarters building in Boston, Massachusetts. (AP/Bill Sikes)
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The only known cure for painful sickle cell disease has long been a bone marrow transplant. But another option has just broken the horizon. Scientists say a new treatment attacks the disorder—by altering a person’s genes.

Sickle Cell Disease, or SCD, is a group of inherited red blood cell disorders. Healthy round red blood cells carry oxygen through the body’s blood vessels. But C-shaped sickle cells aren’t healthy. They’re stiff and gummy. Sickle cells can block blood flow in small blood vessels. This causes pain and other serious problems such as anemia, infection, organ damage, even stroke.

Sickle cells live only about a tenth of the time a normal blood cell lives. That means someone with SCD nearly always lacks enough red blood cells—and gets less oxygen because of that.

Millions of people around the world have SCD. Anyone can be afflicted or carry the gene for the disease. However, SCD occurs most often among black persons and people from places where malaria is or was common, such as Africa and India.

Treatments for SCD include medicines and blood transfusions. The only permanent solution has been a bone marrow transplant. That requires a matching donor. And transplant carries rejection risks.

Vertex Pharmaceuticals researchers have developed a new therapy using a gene-editing tool called CRISPR.

The one-time therapy involves permanently changing DNA in a patient’s blood cells. The goal is for the body to return to producing a healthy form of its own blood protein (hemoglobin).

First, doctors remove stem cells from the blood. They use CRISPR to knock out the gene that makes diseased blood. Then they return the altered cells to the patient.

Dr. Allison King cares for young people with SCD. “Anything that can help relieve somebody with this condition of the pain and the multiple health complications is amazing,” says King. “It’s horribly painful. Some people will say it’s like being stabbed all over.”

God formed every detail of every human’s body. (Psalm 139:13) Illness and pain are part of physical existence ever since the fall into sin. When suffering comes, it’s helpful to remember that even hardship comes sifted first through God’s good hands. (Job 2:10) And God is compassionate. He gives humans the ability to seek ways to reduce suffering. Jesus Himself entered into our suffering. (Isaiah 53)

In December, the FDA approved two gene therapies for patients 12 and older with severe forms of SCD. One uses the CRISPR tool.

As exciting as the new therapies sound, genetics experts are still unsure whether such treatment could cause unexpected, unwanted changes to a person’s genetic material. But early results look promising.

Dr. Monica Bhatia treats children with SCD. She says someday, gene therapy “could be transformative and really change the landscape of sickle cell disease.”

Why? Medical advances like gene editing can bring merciful relief to the suffering. But it’s important to consider possible unintended consequences of new methods and treatments.

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