Genetic editing has emerged as another treatment option for genetically inherited human illnesses, including sickle cell disease.
Scientists published partial results New England Journal of Medicine This showed that gene editing was successfully used to treat the disease, with recipients showing positive results. Since receiving treatment a few months ago, the 10 participants in the study have not required hospitalization or blood transfusions and are not at risk of pain while the side effects are resolved.
Sickle cell disease, also known as sickle cell anemia, is a hereditary red blood cell disorder that affects more than 12 million people, mainly in sub-Saharan Africa, African Americans, and Indians. It is the cause. Saudi Arabia and Mediterranean countries.
Patients with sickle cell disease can suffer from intermittent extreme physical pain, commonly referred to as a “crisis,” due to the “illness” of some red blood cells. Currently, there is no cure for the disease and the best results from the disorder can only be obtained through management, so countermeasures prioritize preventative strategies, including increased access to testing.
The approach to sickle cell disease management adopted in this study is a clinical application of the latest advances in gene editing that enable the correction of diseases caused by genetic errors.
In November, the Royal Swedish Academy of Sciences awarded Emmanuelle Charpentier and Jennifer Doudna the Nobel Prize in Chemistry, which they described as “a tool for rewriting the code of life,” for the development of CRISPR / Cas9 gene scissors.
“This technology is revolutionizing life sciences, contributing to new cancer treatments and may realize our dream of treating hereditary diseases,” said the Academy.
The results of the sickle cell test showed that it was effective in raising fetal hemoglobin levels while lowering sickle cell hemoglobin levels.
A research team led by pediatric hematologist Haydar Frangoul admitted that it was previously unthinkable that clinical trials could be achieved.
“Our vision for this approach is to use the patients’ own stem cells to provide transformative therapies. With these data from 10 patients, we can see the potential to achieve this vision. Increasingly, with more data and longer follow-up periods, we hope to see that there are durable treatments that can change the lives of many patients, “Frangoul said in a statement.
However, the widespread and expensive elaborate technology needed may be out of the reach of patients with sickle cell disease in Africa, where the highest disease burden exists.
Ayodele Akinyemi, a genetics researcher at the University of Ibadan in Nigeria, said the current approach is still cumbersome and patient-specific, so it may take some time before it is cheap and easy to mass produce. “It’s no longer impossible to end sickle cell disease,” he says. “The tools seem to be available now, but what we have to do now is to make them very cheap and easy to deploy anywhere.”
The treatment is still , but CRISPR Therapeutics and Vertex Pharmaceuticals, who jointly developed the treatment, Recently revealed They have already secured regulatory approvals, including the United States and Europe.
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