Researchers at University of Health Sciences (UHS) have reported progress in developing a new gene-editing approach for the treatment of beta-thalassemia, an inherited blood disorder affecting thousands of patients in Pakistan. The research is being conducted under the supervision of Dr Mahmood Saba at the university’s Human Genetics and Molecular Biology Department. According to UHS, the team is working on an advanced gene-editing technique known as multiplex prime editing, which allows highly precise modifications in DNA.

The research is being conducted under the supervision of Dr Mahmood Saba at the university’s Human Genetics and Molecular Biology Department. According to UHS, the team is working on an advanced gene-editing technique known as multiplex prime editing, which allows highly precise modifications in DNA.

Researchers have developed recombinant genetic constructs targeting the gamma-globin gene, which controls fetal hemoglobin production. Scientists believe reactivating fetal hemoglobin in beta-thalassemia patients may help compensate for defective adult hemoglobin and reduce the severity of the disease.

The university said the development marks an important early step toward gene-based treatment options that could eventually reduce patients’ dependence on lifelong blood transfusions.

Beta-thalassemia is caused by defects in the beta-globin gene, leading to severe anemia and other health complications. Many patients require regular blood transfusions, iron-removal therapy, and long-term medical care.

According to the data shared by UHS, nearly 100,000 people in Pakistan are living with thalassemia major, while an estimated 5,000 to 9,000 affected children are born annually.

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