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.
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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.