A safer, more effective vector could offer a new treatment option for patients with devastating blood disorders
Blood disorders like sickle cell disease and beta-thalassemia wreak havoc on a child’s body, causing anemia and excruciating pain and increasing the risk of life-threatening infections. Treatment options are limited, and existing gene therapy can come with toxic side effects.
Through donation-supported research, Stefano Rivella, PhD, and Laura Breda, PhD, have engineered a superior vector that does more with less. By requiring less vector to deliver healthy genes to a patient’s cells, this new technology reduces the potential of bone marrow defects and cancer. This stronger vector could also lead to modifications that reduce the side effects associated with the myeloablation required to engraft genetically corrected bone marrow cells. Further development of this technology could lead to in vivo gene delivery, thus eliminating the need for toxic myeloablation altogether.
This new vector, soon moving into clinical trials, could provide a safer, more effective treatment for patients with sickle cell disease.
Professor of Pediatrics, Division of Hematology & Scientific Director of the Sickle Cell and Red Cell Disorders Curative Therapy Center (CuRED)
Research Assistant Professor, Division of Hematology
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