There's more good news in the battle against sickle cell disease, with another trial finding CRISPR gene-editing therapy delivering impressive results for patients.
“It's encouraging that this gene-editing treatment continues to show promising efficacy for sickle cell patients,” said study lead investigator Dr. Rabi Hanna. He's chair of the division of pediatric hematologyRecent advances in what's known as CRISPR gene-editing technology helps correct the disorder by tweaking the underlying gene abnormality behind it.
The Cleveland Clinic CRISPR approach is called renizgamglogene autogedtemcel . It's am experimental one-time treatment that uses the patient's ownare harvested and then sent for gene editing in the lab. Patients also underwent chemotherapy to make room for the new stem cells to be infused into their bone marrow.
"Following treatment, all patients successfully regained their white blood cells and platelets," according to a clinic news release. "Importantly, all patients have remained free of painful events since treatment, and those followed for five months or greater have seen their"These latest results offer hope that this new experimental treatment will continue to show progress and get us closer to a functional cure for this devastating disease," he said.
The findings were presented Thursday at the European Hematology Association 2024 Hybrid Congress in Madrid. Such findings should be considered preliminary until published in a peer-reviewed journal.Find out more about sickle cell disease at the Sickle Cell Disease Association of America.Anemia Symptoms and Signs, Types, Treatment and Causes
Anemia is a disease marked by low numbers of red blood cells. Low iron or underlying disease, like cancer, may be to blame. Treatment can resolve anemia.
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