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 ...

Jun 19, 2025 Scientists discover new approach to gene therapy Scientists successfully restarted inactive genes by bringing them closer to genetic switches on the DNA called enhancers. The intermediate ...

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 ...

Background Deletions removing 100s–1000s kb of DNA, and variable numbers of poorly characterised genes, are often found in patients with a wide range of developmental abnormalities. In such cases, ...

The first gene therapy clinical trials were initiated more than two decades ago. In the early days, gene therapy shared the fate of many experimental medicine approaches and was impeded by the ...

CRISPR-Cas9 deletions of STAT5-bound elements and functional impact on Il2ra superenhancer activity and gene expression. (A) ChIP-Seq analysis of STAT5 binding in preactivated CD4 + T cells in WT, ...

BACKGROUND Angelman syndrome (AS) is a severe neurobehavioural disorder caused by defects in the maternally derived imprinted domain located on 15q11-q13. Most patients acquire AS by one of five ...