• June 14, 2023
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‘Gene therapy potential cure for sickle cell disease’

People diagnosed with sickle cell disease can now rejoice, as a recent study has discovered a potential cure for the painful disease. Researchers have discovered that a novel gene therapy has been successful and safe for four patients.

Doctors at Cleveland Clinic Children’s in Ohio, where two of the patients received treatment, are optimistic that their promising findings will be confirmed by further studies, as UPI.com reported.

“New treatments like this are critical for people who have sickle cell disease,” said researcher Dr Rabi Hanna, director of the paediatric blood and bone marrow transplant programme at Cleveland Clinic Children’s.

“These initial results provide hope that this new technology will continue to show progress as we work towards creating a possible functional cure for this devastating and life-threatening disease,” Hanna added in a hospital news release.

Sickle cell disease is a painful and disabling genetic blood disorder. The illness, which affects about 100,000 Americans, is more prevalent in black people. Blood cells cannot transport as much oxygen because they are shaped like sickles rather than being round.

Additionally, sickle cells have a propensity to cluster together and block tiny blood vessels. It may result in heart failure, liver fibrosis, liver failure, stroke, cardiomyopathy, and heart failure.

The multicenter Ruby Trial, as it is known, aims to enrol 40 patients with severe sickle cell disease.

Based on the results of the study, a patient’s own blood-forming stem cells are altered by this experimental, one-time gene-editing therapy. The goal is to fix the mutation that causes sickle cell anaemia.

The four patients underwent chemotherapy to remove any remaining bone marrow before having their stem cells harvested for gene editing. They were subsequently given their bodies’ repaired cells back.

CRISPR/CA12 is the name of the CRISPR gene-editing method that is employed. Blood stem cell genome modification allows the body to create strong, healthy blood cells with a high degree of precision.

All four patients were found to have new white blood cells by the time the study was finished, and there were no serious side effects.

The patients’ haemoglobin levels also returned to normal, according to the researchers. Since red blood cells carry oxygen throughout the body, this is by far their most crucial component.

The trial results presented at a meeting of the European Haematology Association were preliminary until published in a peer-reviewed journal. Medications can help with symptoms but not cure sickle cell disease, which has an average lifespan of the mid-40s.

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