Dr Sung-Yun Pai presents findings from a recent study at the 2013 ASH Annual Meetings.

Previous studies have investigated the potential for gene therapy using a retroviral vector to treat children with the fatal inherited disease, X-linked severe combined immunodeficiency (SCID-X1, or “bubble boy disease”). The vector works by latching to the surface of the T cell and injecting genetic material that helps “train” the cells to properly produce their own immune cells. While successful in earlier studies, in some cases the children developed leukaemia when new corrective genetic material was inserted near a trigger in the children’s DNA, predisposing T cells to turn into cancer cells.