Promising results from the first clinical trials of globin gene transfer to treat beta-thalassemias-inherited forms of anemia-have eliminated the need for blood transfusions in some individuals.

Betibeglogene autotemcel gene therapy led to transfusion independence in 89% of patients with severe transfusion-dependent β-thalassemia. The trial involved 18 patients with specific genotypes, ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Thalassemia is a common genetic condition affecting red blood cells. Children with severe forms of thalassemia often develop symptoms early in life, but effective treatment can manage the condition.

Researchers described a promising new approach for using gene therapy to treat sickle cell disease in the peer-reviewed journal Human Gene Therapy. To improve the efficacy of gene therapy when using ...

Researchers have found a promising new method for gene therapy. They successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

Scientists successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate piece of DNA was cut out using CRISPR-Cas9 technology.