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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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BigField GEG Tech's insight:
The authors used CRISPR/Cas9 to correct β-Thal iPSCs, gene-corrected cells exhibit normal karyotypes and fully pluripotency as hES, showed no off-targeting effects. Their results shown that the gene-corrected β-Thal iPS cell lines restored HBB expression and reduced reactive oxygen species (ROS) production. www;geg-tech.com
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BigField GEG Tech's insight:
This review summarizes the most significant developments in β-thalassemia gene therapy over the last decade, with a strong emphasis on the most recent findings, for β-thalassemia model systems; for β-, γ-, and anti-sickling β-globin gene addition and combinatorial approaches including the latest results of clinical trials; and for novel approaches, such as transgene-mediated activation of γ-globin and genome editing using designer nucleases.
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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