Scientists find new pulse on gene therapy delivery

While the researchers' new technique hasn't advanced enough to deliver drugs, a team at Ohio State University has invented a system that used electric pulses to send pieces of DNA into cells in a lab experiment. This approach could eventually find gene-therapy applications, using it as a way of getting precise amounts of genetic material into cells that can then be injected into patients to treat cancer and other diseases, according to the university's press release.

The university says that the technology offers the first way to deliver a "precise dose of a gene therapy agent directly into a living cell without a needle." The scale of the system now enables them to inject only a small number of cells with genetic material, making that prototype impractical as a tool for delivering gene therapies. In a lab experiment, the OSU group led by professor L. James Lee showed that their system could move anti-cancer genes into leukemia cells, causing the cells to perish. Lee and his group are now working on a version that could be used to inject as many as 100,000 cells with agents at once, making therapeutic and diagnostic applications of the technology feasible, according to the university.

Gene therapy has been making a comeback in recent years as research teams make progress with the experimental treatments, yet there's always a question of how to safety administer the genes into patients' cells. The OSU group's technique, called nanochannel electroporation (NEP), involves the placement of cells inside an electronic device, which also holds a therapeutic agent in separate compartment. Pulses of electricity are used to advance the agents through a tiny channel, then into small openings in the cell membrane. The dose size depends on the size of the channel and the number of electrical pulses. The system can move DNA fragments into cells in a matter of milliseconds.

"We hope that NEP could eventually become a tool for early cancer detection and treatment--for instance, inserting precise amounts of genes or proteins into stem cells or immune cells to guide their differentiation and changes--without the safety concerns caused by overdosing, and then placing the cells back in the body for cell-based therapy," Lee said in a statement.

His group's work has been published in online in the journal Nature Nanotechnology.

- here's the release