According to a press release issued by the Swiss Federal Institute of Technology in Zurich (ETH), molecular biologist Mandy Boontanrart has developed a promising approach to the treatment of hereditary anemia, using the CRISPR/Cas9 technology known as gene scissors. Boontanrart, who is part of a group led by ETH Professor Jacob Corn, worked on a study that could prove groundbreaking in the treatment of hereditary beta-hemoglobinopathies. This term covers two types of anemia: beta thalassemia and sickle cell anemia, which are two of the world’s most common hereditary diseases.
There is currently no effective treatment for beta-hemoglobinopathies. In their new study, Boontanrart and other researchers show that the problem could be solved by promoting production of delta globin, which would replace the faulty beta globin.
They had the idea of changing the control sequence in order to increase delta globin production, using the CRISPR-Cas9 gene scissors to insert three additional DNA sections in progenitor blood cells ahead of the HBD gene, which contains the blueprint for delta globins. These insertions stimulate the cell machinery to produce more delta globin, explains the ETH press release. “We managed to significantly increase the proportion of delta globin, to the point where it could offer a therapeutic benefit,” Boontanrart says.
To translate her research findings into practice, Boontanrart launched the Ariya Bio project in 2021 during her ETH Pioneer Fellowship. The spin-off is based in ETH’s ieLab in Schlieren in the canton of Zurich. ce/gba
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