Jun 22, 2021
Cleveland Clinic Trial to Test Gene Therapy as Treatment of Sickle Cell Disease
Posted by Quinn Sena in categories: bioengineering, biotech/medical, genetics
Novel study designed to correct genetic abnormalities of red blood cells.
Cleveland Clinic researchers are enrolling patients in a clinical trial that aims to work toward a cure for sickle cell disease, by changing the patient’s genetics. Sickle cell disease, a genetic blood disorder, is a painful and debilitating condition for which there are few approved therapies.
The multicenter study will evaluate 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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