Gene therapy hybrid

Gene therapy delivery systems can either be viral or nonviral. Viruses, which offer strong specificity, high efficiency, and a natural predilection for delivering DNA into the cell and having it expressed, can also induce unintended and sometimes fatal consequences. Nonviral approaches, on the other hand, though less toxic, are relatively inefficient and nonspecific. Now, a group of Japanese researchers has blurred the lines to create a hybrid approach that they say offers the tissue specificity and efficiency of a viral approach without the harmful side effects.

The team, led by Shun'ichi Kuroda of Osaka University, reports in the June 29 advance online issue of Nature Biotechnology that they used yeast to produce vesicles called L particles, essentially nanometer-scale fat droplets studded with hepatitis B virus (HBV) envelope L protein. They then inserted DNA or drugs into the particles by electroporation, tested them both in vivo and ex vivo, and observed...