Sickle cell disease (SCD) is an autosomal recessive disorder in which a substitution of valine for glutamic acid in the β-chain hemoglobin molecule143 causes red blood cells to form sickle shapes and polymerize particularly when patients are exposed to hypoxemia or extremes of temperature or are dealing with infections.
All these can be summarised as a genetic disorder in which the shape of red blood cell is distorted resulting in an irregular shap instead of a disc shape.
Currently, sickle cell disease is most often treated with extended hospitalizations and constant medication. The only curative treatment currently available is a bone marrow transplant, which is an invasive treatment and requires a donor.
But due to numerous innovative idea, the breakthrough gene-editing technology CRISPR-Cas9, which was co-discovered by Jennifer Doudna, a UC Berkeley professor of biochemistry and molecular biology, may soon be applied to humans for the first time to treat sickle cell disease. Sickle cell disease primarily affects Black people, which makes finding test subjects for clinical trials more difficult.
CRISPR as a treatment for sickle cell disease is the first step in what could become a common way to treat genetic diseases. While there remains a great deal of skepticism about gene-editing technology, there also exists a great deal of hope where none previously existed. Diseases that have long been mitigated but never fully addressed could soon be curable using this technology.
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