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CHOP and Penn researchers have developed a proof-of-concept model for delivering gene editing tools to treat blood disorders directly within the body.
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In a short documentary by the New England Journal of Medicine, Dr. Alexis Thompson discusses the early successes of gene therapy, as well as its risks and benefits.
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A leading study site for nearly a decade, CHOP will be part of a network of treatment centers in the country offering the therapy to patients who require regular blood transfusions.
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A group of researchers that includes CHOP presented new data on CRISPR-edited gene therapy for blood disorders at an international meeting.
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CHOP researchers have developed a proof-of-concept treatment for blood disorders like sickle cell disease that elevates both fetal and adult hemoglobin.
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CHOP researchers were interviewed as a part of a comprehensive article about cell and gene therapy in Philadelphia Magazine.
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CHOP researchers have developed a gene therapy vector for sickle cell disease and other blood disorder that is potentially superior to competitors.
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The US Food and Drug Administration has approved two new breakthrough therapies for patients with sickle cell disease (SCD), and we believe both may enhance the treatment of our patients here at CHOP.
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The US Food and Drug Administration has granted accelerated approval to two novel drugs to treat sickle cell disease.
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Stefano Rivella, PhD, an expert in creating the next-generation lentiviral vectors for the cure of hemoglobinopathies including sickle cell disease (SCD), as well as a leader in the discovery of erythroid and iron disorders including beta-thalassemia, has led three new published studies that make significant contributions to the scientific body of literature regarding red blood cells, and how manipulating the production of red cells can lead to the development of new disease-modifying therapies.