The Associated Press reported that scientists are seeing promising results from the first studies testing gene editing for a painful, inherited blood disorder that affects millions globally — especially Black people.
Doctors are hopeful that a one-time treatment, which involves permanently altering DNA in blood cells with a tool called CRISPR, will treat and possibly cure sickle cell disease and beta thalassemia.
Some results were published by the New England Journal of Medicine and partial results were presented Saturday at an American Society of Hematology conference.
Doctors detailed the progress of 10 patients who are several months removed from their treatment. The patients are now free from the pain crises that plagued their lives prior to the new treatment, and no longer require regular blood transfusions.
The first patient in the sickle cell study, Victoria Gray, had long suffered severe pain that sent her to the hospital.
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“I had aching pains, sharp pains, burning pains, you name it. That’s all I’ve known my entire life,” said Gray, 35. “I was hurting everywhere my blood flowed.”
Gray received her treatment a year ago, and since then has weaned herself off of pain medications she relied on to manage her symptoms.
Sickle cell affects millions and most are Black people, while Beta thalassemia strikes about one in 100,000 people.
The treatment that was studied attacks the problem at its genetic roots. In the womb, fetuses make a special type of hemoglobin. After birth, when babies breathe on their own, a gene is activated that instructs cells to switch and make an adult form of hemoglobin instead. The adult hemoglobin is what’s defective in people with one of these disease. The CRISPR editing aims to cut out the switching gene.
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The treatment for these diseases involves removing stem cells from the patient’s blood, then using CRISPR in a lab to knock out the switching gene. Patients are given strong medicines to kill off their other, flawed blood-producing cells. Then they are given back their own lab-altered stem cells.
Saturdays results details information on the first ten patients, seven with beta thalassemia and three with sickle cell.
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