Researchers at Massachusetts Institute of Technology (MIT) have found a way to fine-tune the ability of biodegradable polymers to deliver genes, which may lead to safe and effective techniques for gene therapy.
Gene therapy involves inserting new genes into patients' cells to fight diseases like cancer. It holds great promise but has yet to realize its full potential, in part because of safety concerns over the conventional technique of using viruses to carry the genes.
There are risks associated with using viruses. As a result, many researchers have been working on developing non-viral methods to deliver therapeutic genes.
The new MIT work, published this week in Advanced Materials, focuses on creating gene carriers from synthetic, non-viral materials.
The MIT scientists focused on three poly (beta-amino esters), or chains of alternating amine and diacrylate groups, which had shown potential as gene carriers. They hoped to make the polymers even more efficient by modifying the very ends of the chains.
When mixed together, these polymers can spontaneously assemble with DNA to form nanoparticles. The polymer-DNA nanoparticle can act in some ways like an artificial virus and deliver functional DNA when injected into or near the targeted tissue.
The researchers developed methods to rapidly optimize and test new polymers for their ability to form DNA nanoparticles and deliver DNA. They then chemically modified the very ends of the degradable polymer chains, using a library of different small molecules.
"These minor alterations in polymer composition significantly increase the polymers' ability to deliver DNA, and these new materials are now the best non-viral DNA delivery systems we've tested," said the researchers.
The polymers have already been shown to be safe in mice, and the researchers hope to ultimately run clinical trials with their modified polymers.
Source: Xinhua
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