WHEN it comes to gene therapy, it can鈥檛 get much simpler. Rather than using viruses, spheres of fat or nanoparticles to deliver DNA to cells, Jon Wolff鈥檚 team at the University of Wisconsin-Madison has shown that a high-pressure injection of 鈥渘aked鈥� DNA straight into a vein is enough to get it into up to half of cells in the targeted muscles of rats, dogs and monkeys. 鈥淚 admit I first thought it might blow up the vein,鈥� he says. 鈥淏ut it worked and that changes everything.鈥�
Wolff, who presented his results to the American Society of Gene Therapy last week, plans to use the method to treat Duchenne鈥檚 muscular dystrophy, and hopes a human trial will begin within two years.