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β-Thalassemia is caused in part by a mutated β-globin gene, so researchers developed a way to replace the entire mutated gene with a healthy version that restored and balanced protein production to a normal level.
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β-thalassemia is a disease that is caused in part by a mutated β-globin HBB gene and involves a dangerous reduction in hemoglobin production, resulting in anemia, muscle weakness and fatigue. It affects one in 100,000 people worldwide, making it one of the most common genetic diseases in the world. Researchers at Stanford University have developed a gene therapy that replaces the entire mutated β-globin gene with a healthy version. They took stem cells from patients, cut out one of the two HBA genes that produces α-globin and replaced it with an HBB transgene using CRISPR-Cas9 technology, transfected the modified cells into the bodies of mice. The patients thus become their own donor. This technique allowed the α-globin promoter to be used to restore and balance β-globin production to a normal level. Now, the question is whether the corrective effects in single cells and mouse models translate into curative effects for human patients
https://www.nature.com/articles/s41591-021-01284-y