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
0
2
Hemic and Lymphatic Diseases: News and Research on beta-Thalassemia
MeSH tree: C15.378.050.141.150.875.150
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
0
0
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
0
0
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
0
0
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
0
27
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.
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
0
0
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
0
71
FDA approves exagamglogene autotemcel to treat beta-thalassemia
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
0
0
Revolutionary gene therapy may offer new life for patients battling chronic sickle cell disease
Lyric Porter wanted her life back. Born with sickle cell disease, she had a fever at age 5 that scarred her lungs and required her to roll an oxygen tank to school. When she was growing up, clogged blood vessels racked her ...
Jan 3, 2024
0
0
