Genomic maps untangle the complex roots of disease
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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Hemic and Lymphatic Diseases: News and Research on Thalassemia
MeSH tree: C15.378.050.141.150.875
Study offers real-world data on commercial implementation of gene therapies for sickle cell disease, beta thalassemia
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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Exa-cel gene therapy may off effective cure for beta-thalassemia and sickle cell disease in children younger than 12
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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One-time gene therapy could end lifelong transfusions for rare blood disease
Thanks to in-utero blood transfusion technology, what was once a fatal diagnosis in the womb can now result in live births. However, this medical advancement created a new challenge: a growing population of children born ...
Sep 18, 2025
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Gene therapy leads to improved quality of life in patients with sickle cell disease and beta thalassemia
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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Could a pill replace gene therapy for sickle cell disease? Research uncovers potential path to small-molecule drug
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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Stem cells can tailor their role in gene therapy based on the underlying disease, study suggests
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.
Lentiviral gene transfer improves human alpha globin production for the treatment of alpha thalassemia
Researchers at Children's Hospital of Philadelphia (CHOP) and the University of Pennsylvania Perelman School of Medicine have pioneered a new model that offers a potential platform for developing novel therapies to treat ...
Jul 2, 2024
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Breakthrough therapies are saving lives, but can we afford them?
Harnessing the body's own cells to fight disease, long a medical dream, is finally a reality.
May 13, 2024
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Researchers publish final results of key clinical trial for gene therapy for sickle cell disease
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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