Researchers at Children’s Hospital of Philadelphia (CHOP) have developed a potentially safer and more effective gene therapy vector for blood disorders.
The newly-developed gene therapy vector will focus on blood disorders like sickle cell disease and beta-thalassemia.
It is being considered potentially safer and effective than those currently used in gene therapy trials for these conditions.
The findings of the study were published today in the journal ‘Molecular Therapy’. 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, minimising the risk of toxic side effects.
“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,” added Breda.
Sickle cell disease and beta-thalassemia are genetic blood disorders caused by errors in the genes for hemoglobin, a protein consisting of globin and iron-containing subunits that are found in red blood cells and carries oxygen from the lungs to tissues throughout the body.
The disorders, sometimes referred to as beta-globinopathies due to mutations in the beta-globin gene, lead to serious health complications, ranging from delayed growth and jaundice to pain crises, pulmonary hypertension, and stroke.
Because children with beta-globinopathies have two abnormal copies of the hemoglobin gene – one from each parent – researchers have explored gene therapy as a potential breakthrough treatment.
Using an engineered carrier called a vector to introduce a functional copy of the beta-globin gene, this therapy has the potential to restore normal hemoglobin production in patients with beta-globinopathies.
However, there are challenges to this approach, including limited dose-response, dose-related toxicities, and the need in many cases for myeloablation, a procedure in which the bone marrow is suppressed via chemotherapy or radiation before gene therapy can begin. (ANI)
