Friday, October 30, 2009

Nature Editorial on Gene Therapy

The latest issue of Nature has this insightful, and refreshing, editorial on renewing our optimism for gene therapy. Here is a sample:

In the early 1990s, when the first human trials got under way, it seemed to many that the era of gene therapy was at hand: the techniques of modern molecular biotechnology would make it possible to repair genetic defects by inserting healthy DNA directly into a patient's cells. The excitement was short-lived. Lasting effects proved difficult to obtain in early trials, and the community quickly grew sceptical. Then, in 2003, when it was announced that several gene-therapy patients in a Paris-based clinical trial had developed leukaemia, and that one of them had died, the mood became bleak. Subsequent reports of successful and effective gene-therapy trials have done little to lift the prevailing sense of doom. For most researchers, gene therapy now seems like a dead end.

....To reverse this trend, it is time for researchers and industry to refresh their perspective on gene therapy and to consider its successes with as much intensity as its setbacks. The focus on adverse events has had positive consequences: researchers dissected the exact molecular mechanisms that led to cancer, designed better vectors, devised animal models to test these vectors and developed sophisticated assays for monitoring patients. As a result, both scientists and clinicians now have a battery of extraordinarily refined tools for preclinical and clinical studies of gene therapy. The field is ripe for further successes.