Gene therapy for beta-thalassaemia

The list of life-limiting inherited conditions for which radically new gene-based treatments are appearing continues to grow. The latest is β-thalassaemia, which affects over 1/4 million people worldwide. The majority of sufferers have a lifelong continuing blood transfusion requirement, with all the complications that come with it, such as iron overload. Those fortunate enough to have a compatible relative can sometimes be ‘cured’ by a bone marrow transplant, but that won’t work for many.

Virus vectors have been developed as a means of introducing modified DNA into deficient cells. Researchers in the US and France successfully trialled using a lentiviral vector to transduce an extended β-globin gene structure into thalassaemic haemopoietic stem cells in the lab (Thompson A et al. NEJM 2018. doi: 10.1056/NEJMoa1705342). They then went on to carry out a multicentre phase 1–2 clinical trial, in 22 transfusion-dependent patients aged 12 to 35 (mean 19) years. Nine...

from Archives of Disease in Childhood current issue https://ift.tt/2JNHhaS