Gene Editing May Hold the Key to Curing Sickle Cell Disease
March 21, 2023
Source: drugdu
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Researchers from the University of California, Berkeley have used CRISPR gene-editing technology to cure sickle cell disease in mice. Sickle cell disease is a genetic blood disorder that affects millions of people worldwide. The disease is caused by a mutation in the HBB gene, which produces an abnormal form of hemoglobin that causes red blood cells to become misshapen and break down easily.
Using CRISPR, the researchers were able to correct the HBB gene in mice with sickle cell disease. The corrected gene produced normal hemoglobin, which prevented the misshapen red blood cells from forming. The mice were cured of the disease and showed no signs of sickle cell-related symptoms.
The researchers say that the same approach could be used to cure sickle cell disease in humans. While more research is needed before clinical trials can begin, this is a promising development in the field of gene therapy.
Reference: https://news.harvard.edu/gazette/story/2021/06/gene-editing-shows-promise-as-sickle-cell-therapy/
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