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.

Moreover, this treatment modifies a globin repressor gene, which indeed causes reactivation of fetal globin, but may well have effects on other genes as well, with unknown consequences for the patient ...

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 ...

STAT5-mediated chromatin interactions in superenhancers activate IL-2 highly inducible genes: Functional dissection of the Il2ra gene locus ...

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 ...