MeSH tree: D12.776.124.400.303
Scientists from Johns Hopkins Medicine and eight other institutions in the United States, Africa and Europe say they have identified a potential new gene target that could be edited to treat sickle cell disease, an inherited ...
Mar 24, 2025
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The new gene therapies for sickle cell disease—including the gene-editing treatment Casgevy, based on research at Boston Children's Hospital—have been game-changing for the patients who have received them. But Stuart ...
Dec 18, 2024
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Gautam Dongre's two children in India and Pascazia Mazeze's son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain.
Dec 14, 2023
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A type of gene therapy that precisely "edits" a key bit of DNA might offer a new way to treat sickle cell disease—a painful inherited condition that largely strikes Black children and adults.
Sep 3, 2023
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New research published in the New England Journal of Medicine indicates that stem cell gene therapy may offer a promising, curative treatment for the painful, inherited blood disorder sickle cell disease (SCD).
Aug 30, 2023
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Gene therapy that alters hemoglobin genes may be an answer to curing sickle cell disease (SCD) and beta thalassemia. These two common life-threatening anemias afflict millions of individuals across the globe.
Jun 29, 2023
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Scientists at St. Jude Children's Research Hospital have shown how a protein responsible for adapting to low oxygen conditions (hypoxia), causes increased expression of fetal hemoglobin (HbF) in adults. The finding has implications ...
Oct 12, 2022
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Researchers at Children's Hospital of Philadelphia (CHOP) have discovered a new part of the regulatory cascade responsible for silencing fetal hemoglobin in adult cells. The findings, published today in Nature Genetics, refine ...
Aug 8, 2022
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