New gene therapy proving safe and successful in sickle cell patients' treatment
Written By : Isra Zaman
Medically Reviewed By : Dr. Kamal Kant Kohli
Published On 2023-06-10 04:00 GMT | Update On 2023-06-10 09:47 GMT
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Researchers presenting preliminary data from a clinical trial aimed at discovering a cure for sickle cell disease reveal positive results among its first patients.Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.
Researchers involved in the multicenter Ruby Trial presented an update on the safety and effectiveness of a single dose of EDIT-301, an experimental one-time gene editing cell therapy that modifies a patient’s own blood-forming stem cells to correct the mutation responsible for sickle cell disease.
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