ICYMI: The First Patient to Receive CRISPR for Sickle Cell Disease

Victoria Gray’s recent interview with NPR confirmed that her genetically modified blood cells have truly transformed her life. Victoria volunteered in the first-ever attempt in the U.S. to use CRISPR, the gene-editing technique in the treatment of a genetic disorder.

Victoria has struggled with sickle cell disease for 35 years. This is the first year she has not experienced sudden attacks of pain, fatigue, and rushing to the emergency room for blood transfusions and pain medication.

Will The CRISPR Technique Keep Working?

So far Victoria, who lives in Forest, Mississippi, reports that it has been working. She told NPR that she is “doing great.” She admitted to a cold recently that understandably made her a little nervous. With sickle cell, even a cold could bring about a severe attack.

In fact, Victoria’s recovery has been so successful that she has been released from the landmark study for which she had been selected.

Doctors removed cells from Victoria’s bone marrow and edited genes in the cells. They use a technique called CRISPR that enabled them to make precise changes to the patient’s DNA. It is believed that the procedure will revolutionize medicine.

Billions of these modified cells were infused into Victoria’s body to produce fetal hemoglobin, a protein designed to alleviate sickle cell symptoms.

The procedure has been successful not only for Victoria, but at this writing, it has been performed on a total of 45 patients, some of whom have a related disorder called beta thalassemia. Reports thus far indicate that almost half of the entire group is seeing positive results.

Looking Forward

Vertex Pharmaceuticals of Boston and CRISPR Therapeutics of Cambridge, are jointly seeking FDA approval for the procedure in the next year or so.

And although Victoria is continually aware that she must protect herself and her family from COVID, she has ventured out into the world by working full-time in a cashier’s job. She also has plans to return to her studies at a future date.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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