Recipient of Gene-Editing Sickle Cell Treatment Continues to Thrive

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Courtesy of Gift Habeshaw (Unsplash CC0)

In early 2019, a 34-year-old woman from Mississippi underwent a gene-editing experiment to treat the sickle cell disease that had plagued her since she was three months old. Victoria Gray volunteered to undergo treatment with the “revolutionary gene-editing technique known as CRISPR.”

It has been over a year, and her life has been transformed. She no longer has sudden attacks of horrible pain and overwhelming fatigue. Moreover, the nightmarish emergency room visits to get blood transfusions and powerful pain medication are gone.

The experimental treatment that genetically modified Gray’s blood cells has left her free from the complications of sickle cell disease. Gray answered: “I’m doing great,” when asked how she was. “I haven’t had any problems with sickle cell at all.” However, when she nervously mentioned that she recently had a cold, Gray revealed how traumatized she was by the disease that getting a cold still frightens her.

Gray likened ger fear to post-traumatic stress syndrome (PTSD). She worried if the cold would trigger a sickle cell crisis and that she would end up in the hospital again. But, the gene-editing recipient and her family are excited that she continues to thrive. Gray said:

This was major for me and my family. Two years without me being in the hospital? Wow. We can’t believe it. But we’re so grateful.

Understanding Sickle Cell Disease

sickle cell
Courtesy of National Human Genome Research Institute (Flickr CC0)

Sickle cell disease, also called sickle cell anemia, is a group of inherited red blood cell disorders that affects the hemoglobin, that protein that carries oxygen through the body. People with this disease inherit an abnormal hemoglobin gene from each parent. When a person has this disease, their red blood cells are crescent or sickle-shaped instead of disc-shaped and flexible, which helps the cells move through the blood vessels. However, crescent-shaped cells do not bend; they can block the blood flow to the body.

Serious problems can occur when blocked blood flow through the body includes stroke, eye problems, infections, and episodes of pain, called pain crises. One of the worst things about sickle cell disease; people regularly suffer sudden, excruciating bouts of pain.

Gray’s first sickle cell crisis happened when she was three months old. Her grandmother was bathing her, and she was crying. Believing something was wrong, she was taken to an emergency room where she was diagnosed with this genetic blood disorder.

sickle cell
Courtesy of COD News Room (Flickr CC0)

Sickle cell affects millions of people throughout the world. It is common among those whose ancestry originated from sub-Saharan Africa; Spanish-speaking regions in the Western Hemisphere — South America, the Caribbean, and Central America; Saudi Arabia; India; and Mediterranean countries like Turkey, Greece, and Italy.

Currently, the Centers for Disease Prevention and Control (CDC) reports the exact number of people living with sickle cell is unknown. Nonetheless, the agency estimates the disease affects about 100,000 Americans. The CDC is working with partners to learn more about those living with the condition to understand better how it impacts their health.

In the United States, sickle cell occurs in about one of 365 Black or African-American births, and about one out of every 16,300 Hispanic-American babies are born with the disease.

Scientists are watching the gene-editing experiment Gray and at least 45 others who underwent the treatment of their sickle cell disease. Vertex Pharmaceuticals and CRISPR Therapeutics are the potential cure’s developers. Vertex hopes to seek approval for the treatment in the next 18 months to two years from the Food and Drug Administration (FDA).

Written by Cathy Milne-Ware

Sources:

NPR: A Young Mississippi Woman’s Journey Through A Pioneering Gene-Editing Experiment; by Rob Stein
NPR: First sickle cell patient treated with CRISPR gene-editing still thriving; by Rob Stein
NIH: Sickle Cell Disease
CDC: Data & Statistics on Sickle Cell Disease

Featured and Top Image by Gift Habeshaw Courtesy of Unsplash – Creative Commons License
First Inset Image by Darryl Leja Courtesy of National Human Genome Research Institute’s Flickr Page – Creative Commons License
Second Inset Image by Keith Wells Courtesy of COD Newsroom’s Flickr Page – Creative Commons License

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