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.

Once the child is born, it is switched off. "In people with sickle cell disease or beta-thalassemia, it's the adult globin gene—the main engine that powers red blood cells—that is broken. But fetal ...

Distribution of different genotypes with α-globin gene triplication in 73,967 subjects. (A) Gender distribution. (B) The proportion of different genotypes among 1,443 α-globin gene triplication ...

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 ...

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, ...

BHUBANESWAR: The New Year seems to have ushered in a major breakthrough for thalassaemia patients with the US Food and Drug ...

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.