Researchers at MIT have taken a big step toward developing safe and effective methods for gene therapy. They have found a way to refine the ability of biodegradable polymersto deliver the genes
Gene therapy involves the inserting of new genes into the cells of patients. It is used to combat diseases like cancer and it great promise, but in order to reach its full potential the researchers need to be able to set aside the concerns of the safety level of using viruses to transport the genes.
This research focused on creating a new type of carrier for the gene from synthetic, non-viral material.
Their aim was to take a safe biocompatible, degradable polymer and make it more effective instead of starting out with a virus and trying to make it safer.
Even thought gene therapy had been researched for close to 20 years, and there have been more than 1,000 clinical trials there still are no FDA approved gene therapies. In most of the trials viruses are used as carriers-vectors- to deliver the genes. But there are risks that are associated with using the viruses. Researchers have been working on the development of non viral delivery methods.
In this particular study the scientists at MIT focused their attention on three chains of alternating amine- an organic compound- and diacrylate- crosslinking agent between the molecular chains of polymers- groups. These groups had previously shown potential to be gene carriers. They hope to make them even more efficient by modifying them at the very ends of the chains.
When these polymer are mixed together, they can spontaneously mix with DNA to make nanoparticles. These can act in certain ways like an artificial virus and thereby deliver functional DNA when it is injected either right into or near the target. They have already been shown safe in mice and the next step will be to run clinical trials.
No viral vectors like these could turn out to be not only safer than the viruses are but they may be able to avoid the immune system , which would allow for multiple applications.
One of the specific line of research that MIT is pursuing along with Janet Sawicki at the Lankenau Institute for Medical Research involves ovarian cancer. With this, they have found that these polymer-DNA nanoparticles are able deliver DNA at high levels to ovarian tumors without harming healthy tissue.
The team is led by Daniel Anderson, research associate in MIT’s Center for Cancer Research. First co author is Jordan Green, a graduate student in biological engineering as is Gregory Zugates, a former graduate student in chemical engineering now at WMR Biomedical, Inc. The rest of the team are Nathan Tedford, a former graduate student in biological engineering now at Epitome Biosystems; Linda Griffith, professor of biological engineering; Douglas Lauffenberger, head of biological engineering, and Institute Professor Robert Langer. Sawicki and Yu-Hung Huang of the Lankenau Institute.
The research was funded by the National Institutes of Health, the Department of Defense and the National Science Foundation.
Sosurce: MIT http://web.mit.edu/
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