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 worldwide – mostly blacks.
Doctors hope that a one-time treatment that permanently changes the DNA in blood cells using a tool called CRISPR can treat and potentially cure sickle cell disease and beta thalassemia.
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Some results were published by the New England Journal of Medicine and partial results were presented at a conference of the American Society of Hematology on Saturday.
Doctors explained the progress of 10 patients who were several months removed from their treatment. 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 been in severe pain for a long time, which took her to the hospital.
Read more: Life as a Sickle Cell Warrior: How Lawyers Are Changing the Face of Disease
« I’ve had aching pains, sharp pains, burning pains, as you call it. That’s all I’ve known all my life, ”said Gray, 35. “I hurt everywhere my blood flowed. ”
Gray was treated for a year ago and has since weaned herself from pain medication, which she relied on to relieve her symptoms.
Sickle cells affect millions and most are black, while beta thalassemia affects around one in 100. 000 people affected.
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The treatment studied attacks the problem at its genetic roots. In the womb, fetuses produce a special type of hemoglobin. When babies breathe on their own after birth, a gene is activated that instructs cells to switch and make an adult form of hemoglobin instead. The adult hemoglobin is defective in people with one of these diseases. The CRISPR machining aims to cut out the Schaltgen.
The treatment for these diseases involves removing stem cells from the patient’s blood and then using CRISPR in a laboratory to turn off the switching gene. Patients are given powerful drugs to kill their other, faulty blood-producing cells. Then they get their own laboratory-modified stem cells back.
Saturday results include detailed information on the first ten patients, seven with beta thalassemia and three with sickle cells.
Sickle Cell Disease, Cell, Beta Thalassemia, Gene Therapy, Medicine, Hematological Disease, Hematology
World News – AU – Doctors Hope For Sickle Cell Gene Editing Treatment – TheGrio
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