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2022 Canada Gairdner Award given to Stuart H. Orkin, MD, researcher at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center
His remarkable body of work has not only revolutionized our understanding of how these illnesses occur but has also led to promising new gene-based therapies for thalassemia and sickle cell disease, two inherited blood disorders that affect millions of people around the world.
The Doris Duke Charitable Foundation announced the six projects receiving approximately $5 million through the second Sickle Cell Disease/Advancing Cures grant competition.
Researchers at Dana-Farber/Boston Children's Cancer and Blood Disorders Center and the University of Massachusetts Medical School have developed a strategy to treat two of the most common inherited blood diseases, sickle cell disease and beta thalassemia, applying CRISPR-Cas9 gene editing to patients' own blood stem cells.
The research will focus on adapting gene therapy for use in low-resource areas with high rates of the disease.
A patient with a severe form of sickle cell disease is symptom-free after gene therapy knocks down BCL11A and restores fetal hemoglobin production.
In this Vector report, researchers at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center reveal how the protein, BCL11A, controls the switch in the body’s production of fetal hemoglobin to adult hemoglobin.
David Williams, MD, discusses Dana-Farber/Boston Children's new gene therapy clinical trial for sickle cell disease in a live webchat.