Scientists in Australia have developed a new gene therapy vector that uses the same machinery as viruses to transport their cargo into cells.
David Jans, from the Nuclear Signaling Laboratory at Monash University in Victoria, says that this achievement has raised the hope that one day therapeutic DNA will begin to be transferred to a cell's nucleus far more efficiently than in the past, and thus there will be more effective treatments for several genetic disorders and some types of cancers.
"Through the use of proteins that mimic key functions of viruses for the packaging and transport of therapeutic DNA, we hope to improve the efficiency, and above all, the specificity of human gene therapy," he said.
"Following the creation of efficient, specific and safe DNA delivery vectors, the challenges in human gene therapy will be able to move on from questions of delivery to actual clinical application," he added.
A gene therapy vector is used to deliver a therapeutic gene or a portion of DNA into a cell nucleus similar to how a syringe is used to inject medicines.
To create the new gene therapy vector, Jans and his colleagues used pieces of different genes to create a protein called a "modular DNA carrier", which can be produced by bacteria.
Writing about their work in The FASEB Journal, the researchers have revealed that this protein carries and delivers therapeutic DNA to a cell's nucleus, where it reprograms a cell to function properly.
While experimenting in their laboratory, the research team combined these carrier proteins with therapeutic DNA, and attached them to cell membrane receptors and the nuclear import machinery of target cells.
In turn, according to the researchers, the packaged DNA moved into the cell through the cytoplasm and into the nucleus.
"Effective gene therapy is clearly the best way to treat heritable diseases. It's also an approach to other diseases where the environment or infection messes up our genes." said Dr. Gerald Weissmann, the Editor-in-Chief of The FASEB Journal.
"The Australians have worked out how viruses identify our nuclear ZIP-code, and have delivered therapeutic genes to the same address. This work opens up a new era of pharmaceutical development," Dr. Weissmann added.