MeSH tree: C16.320.070.875.150
Stem cell transplantation could be a rapid and effective way to restore hemoglobin production in individuals with the blood disorder β-thalassaemia. The treatment, presented in a phase 1 clinical trial, could reduce dependence ...
Apr 8, 2026
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Today's biomedical researchers are relentlessly searching for genes that drive disease, with the goal of creating therapies that target those genes to restore health.
Dec 10, 2025
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A study assessing the real-world commercial roll-out of gene therapies for sickle cell disease and beta thalassemia offers lessons learned to inform best practices as manufacturers and medical centers prepare to meet growing ...
Dec 6, 2025
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Preliminary results from two trials of the gene therapy exagamglogene autotemcel (exa-cel) suggest the therapy offers an effective cure for beta-thalassemia and sickle cell disease in children younger than 12. Researchers ...
Dec 6, 2025
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Treatment with exagamglogene autotemcel (exa-cel) led to robust and sustained improvements in quality of life for patients with severe sickle cell disease (SCD) or transfusion-dependent beta thalassemia, according to two ...
Aug 27, 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 Orkin, ...
Dec 18, 2024
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RCCS San Raffaele Scientific Institute in Milan researchers have discovered that hematopoietic stem cells (HSCs) adapt their lineage commitment during gene therapy based on the underlying genetic disease.
Harnessing the body's own cells to fight disease, long a medical dream, is finally a reality.
May 13, 2024
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In a landmark study, an international consortium led by researchers at Children's Hospital of Philadelphia (CHOP) published the final results of a key clinical trial of the gene therapy CASGEVY (exagamglogene autotemcel) ...
Apr 25, 2024
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After approving Casgevy (exagamglogene autotemcel) in December to treat sickle cell disease, the U.S. Food and Drug Administration announced Tuesday that the therapy has now been approved to treat patients older than 12 years ...
Jan 17, 2024
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