Gene therapy has shown some benefit against a new rare disease target, chronic granulomatous disorder (CGD), which prevents normal immune responses against bacterial and fungal infections.
Great Ormond Street Hospital in London has reported that a teenager with CGD has shown a good response to gene therapy that introduced a functional gene to blood stem cells that were used to repopulate his own blood. Unlike earlier gene therapy successes with genetic immune disorders (see previous news), the treatment reportedly does not have such a long-term effect, but without it the patient was not expected to survive.
The latest result was announced on 29th February – the rare date selected as international National Rare Disease Day. The majority of rare diseases are genetic in origin and many are potential gene therapy targets, although the potential of the technique to treat more common forms of disease such as cancer or blindness is also being widely investigated.
Great Ormond Street is currently fundraising for a special Centre for Children's Rare Disease Research.