Gene Therapy Gets a Big Boost

January 25, 2014 — Leave a comment

A medicine that treats illness on the genetic level is much closer to reality, thanks to a novel chemical compound developed by a team of European researchers with the EUREKA consortium. The breakthrough marks the debut of a new type of synthetic DNA-carrying agent.

Gene therapy involves transferring new genetic information into the nucleus of damaged or diseased cells to reprogram the cells, thus repairing them. Three avenues exist to do this: viral gene transfer (where the DNA is carried in a virus), retroviral, and nonviral. Viral gene transfer is done through “infection.” It’s the most effective method for getting new genetic data into a cell but carries significant downsides. The body, after all, is trained to fight infection, and so the risks for the body rejecting the drug are high.

The opposite is true for nonviral or synthetic agents, which are far less effective at getting new DNA into cells but more likely to be accepted by the body. Synthetic agents are also hard to produce in sufficient volume to be practical as a therapy.

The EUREKA project team’s new chemical agents deliver DNA into cell nuclei more efficiently than other man-made carriers and can be reproduced more easily than most synthetic agents.

“When these compounds are in a solution and DNA is added, they bind together,” explains project member Arto Urtti of Helsinki University. “The large, loose DNA molecule collapses and tiny particles of about 10-50 nanometers in diameter are formed, composed of both DNA and carrier. When you present this to the cells, the nanoparticles bind to the cell surface, which folds inwards to form a vesicle [bladder] within the cell. The particles then escape from the vesicle, releasing the DNA.”

The breakthrough is a promising one in a field that has seen decades’ worth of false starts and disappointments. While the mechanics of DNA transfer have been understood since 1959, and scientists are hopeful that gene therapy could be effective in treating cancer, AIDS, or even cardiovascular disease, it has yet to actually cure anything. There are, however, isolated examples of the technique playing a productive role as part of other therapies.

“Genome synthesis and assembly technologies are advancing exponentially. In a decade, we could see 103-105 improvements, driving synthetic genomic advances across the board, including gene therapies,” says Andrew Hessel, founder of the Pink Army Cooperative, an open-source genetics research project.

Hessel still sees considerable obstacles to further breakthroughs, but these are, in part, political and regulatory.

“The current tools for doing gene therapy are crude, as is the proprietary business climate used for their development. This means their development and application will be ad hoc at best and limited to severe conditions,” he says. “Given the Western regulatory architecture, the advancements are likely to come from smaller groups operating in permissive locales.”

-Patrick Tucker

Sources: Eureka Network, Personal interview with Andrew Hessel, founder of the Pink Army Cooperative.

Originally published in THE FUTURIST, November-December 2010


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