New gene editing success with leukaemia seems promising for thalassaemia and haemophillia as well
For almost every warning fired at gene-editing, there emerges one spectacular success story. So, while Chinese scientists’ efforts to create a “designer” human embryo triggered a flood of letters from scientists elsewhere urging them to desist, on both scientific and ethical grounds, significant success in controlling HIV/AIDS with the help of gene-reporting was reported in 2014. And, now, it has helped a 1-year-old leukaemia patient beat the disease.
Nature reports Layla,being treated in a London hospital, went into remission after she received modified immune cells from another person. The toddler had not responded to known methods of treatment. Even as companies get ready for trials that will inject DNA coding gene-editing enzymes directly into the subject’s body, in Layla’s case, the researchers extracted T-lymphocytes (immune cells maturing in the thymus ), that is responsible for cell-mediated immunity, and exposed it to a DNA-cutting enzyme called TALEN. The enzyme deactivated the immune genes which otherwise would have attacked Layla’s cells, and modified genes to protect the new cells from anti-cancer drugs. The toddler then underwent treatment that destroyed her own immune system—where leukaemia originates—and replaced it with the new immune cells. However, the treatment is only a “bridge” that keeps the person alive until a donor with matched T-cells can be found. That aside, the success of ex-vivo gene editing in this case promises a new wave of treatment for diseases such as haemophilia and thalassaemia.