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Philadelphia, January 29, 2021--Researchers at Children's Hospital of Philadelphia (CHOP) have developed a gene therapy vector for blood disorders like sickle cell disease and beta-thalassemia that is potentially safer and more effective than those currently used in gene therapy trials for those conditions. The vector, an engineered vehicle for delivering functional copies of the hemoglobin gene to correct a genetic abnormality, leads to the production of more hemoglobin with a lower dose, minimizing the risk of toxic side effects.
The findings were published today in
Molecular Therapy.
"These results have many potential benefits for the successful treatment of patients affected by beta-globinopathies like sickle cell disease and beta-thalassemia, including a better dose response, a minimized chance of clonal expansion and tumorigenesis, a reduced cost of therapy, and a potentially reduced need for chemotherapy or radiation before beginning gene therapy," said Laura Breda, PhD, research assistant professor at CHOP and first author of the paper. "All of us in the CuRED Frontier Program at CHOP are dedicated to finding new and improved curative therapies for blood disorders, and we look forward to taking steps to move this vector into clinical trials."