Story Summary: Sawicki was working on treating ovarian cancer by delivering through viruses the gene for the diphtheria toxin, which kills tumor cells. I had been working with adenoviruses to deliver DNA, and I was running into some problems with using them, says Sawicki. This summer, the two laboratories reported that the nanoparticle-delivered gene therapy successfully suppressed ovarian tumor growth in mice. Though Anderson found his thesis topic scientifically interesting, it didnt have a sense of immediate impact for me. I wanted to see if I could get closer to medicine. Anderson and chemist David Lynn, then a postdoctoral fellow in Langers lab and now a professor at the University of Wisconsin, developed a large collection of different biodegradable polymers (large molecules composed of repeating subunits) known as poly(beta-amino esters). When these synthetic polymers are mixed with DNA, they spontaneously assemble to form nanoparticles. In some ways, these polymer-DNA nanoparticles can act like an artificial virus, delivering functional DNA when injected into or near the targeted tissue. If you can try one or two things every six months, it might take a while to find something that works. But if you can try tens of thousands of things, your chances of success are much greater, and thats true for any venue, says Langer. Non-viral vectors are now comparable to viral vectors, in some cases, says Huang, whose research focuses on delivering genes surrounded by a fatty membrane. Both viral and non-viral methods could eventually prove useful and safe, says gene therapy researcher Katherine High, who is part of a team that recently used viral gene therapy to restore some sight to children suffering from a congenital retinal disease. Its been a slow road, says High, a professor at the University of Pennsylvania Medical School, but over the past 20 years scientists have made much progress in managing the safety issues posed by viral vectors….Read the Full Story

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