Navigation Links
MIT works toward safer gene therapy
Date:9/7/2007

CAMBRIDGE, MA (09/07/2007) -- In work that could lead to safe and effective techniques for gene therapy, MIT researchers have found a way to fine-tune the ability of biodegradable polymers to deliver genes.

Gene therapy, which involves inserting new genes into patients' cells to fight diseases like cancer, 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.

The new MIT work, published this week in Advanced Materials, focuses on creating gene carriers from synthetic, non-viral materials. The team is led by Daniel Anderson, research associate in MIT's Center for Cancer Research.

What we wanted to do is start with something that's very safe-a biocompatible, degradable polymer-and try to make it more effective, instead of starting with a virus and trying to make it safer, said Jordan Green, a graduate student in biological engineering and co-first author of the paper.

Gregory Zugates, a former graduate student in chemical engineering now at WMR Biomedical, Inc., is also a co-first author of the paper.

Gene therapy has been a field of intense research for nearly 20 years. More than 1,000 gene-therapy clinical trials have been conducted, but to date there are no FDA-approved gene therapies. Most trials use viruses as carriers, or vectors, to deliver genes.

However, 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 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.

Just by changing a couple of atoms at the end of a long polymer, one can dramatically change its performance, said Anderson. 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.

The polymers have already been shown to be safe in mice, and the researchers hope to ultimately run clinical trials with their modified polymers, said Anderson.

Non-viral vectors could prove not only safer than viruses but also more effective in some cases. The polymers can carry a larger DNA payload than viruses, and they may avoid the immune system, which could allow multiple therapeutic applications if needed, said Green.

One promising line of research involves ovarian cancer, where the MIT researchers, in conjunction with Janet Sawicki at the Lankenau Institute for Medical Research, have demonstrated that these polymer-DNA nanoparticles can deliver DNA at high levels to ovarian tumors without harming healthy tissue.


'/>"/>

Contact: Elizabeth Thomson
thomson@mit.edu
617-258-5402
Massachusetts Institute of Technology
Source:Eurekalert

Related biology news :

1. New Study from Affymetrix Laboratories Points to Changing View of How Genome Works
2. MSI releases moleculizer - a new approach to simulation of intracellular biochemical networks
3. VCU Researchers Identify Networks Of Genes Responding To Alcohol In The Brain
4. Computational Method Speeds Mapping of Cell Signaling Networks
5. FDA Works To Speed The Advent Of New, More Effective Personalized Medicines
6. Brain works more chaotically than previously thought
7. Researchers discover how tumor suppressor gene works
8. Brain networks change according to cognitive task
9. Hand sanitizer gel works
10. Gene therapy works in mice to prevent blindness that strikes boys
11. NIAID researchers show how promising TB drug works
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/22/2017)... 21, 2017 Vigilant Solutions , a ... enforcement agencies, announced today the appointment of retired FBI ... public safety business development. Mr. Sheridan brings ... including a focus on the aviation transportation sector, to ... position, Mr. Sheridan served as the Aviation Liaison Agent ...
(Date:3/9/2017)... Australia , March 9, 2017 ... the prestigious World Lung Imaging Workshop at the University ... , was invited to deliver the latest data to ... globally recognised event brings together leaders at the forefront ... developments in lung imaging. "The quality ...
(Date:3/2/2017)... 2, 2017 Summary This report provides ... its partnering interests and activities since 2010. ... Read the full report: ... since 2010 report provides an in-depth insight into the partnering ... On demand company reports are prepared upon purchase ...
Breaking Biology News(10 mins):
(Date:3/22/2017)... ... 22, 2017 , ... Researchers face a fundamental challenge as ... full-size tissues, bones, even whole organs to implant in people to treat disease ... into the developing tissue. , Current bioengineering techniques, including 3-D printing, can’t ...
(Date:3/22/2017)... Mass. , March 22, 2017 Good ... that it has eclipsed the 130 million covered lives ... Blue Shield of Texas . With ... the Company continues to enjoy strong payor acceptance based ... its clinical programs and genetic counseling, its industry-leading customer ...
(Date:3/22/2017)... , March 22, 2017 ... fragmented, states a research report by Transparency Market Research ... Inc., Amgen Inc., and AbbVie Inc., accounted for a ... The prominent players in this market are focusing aggressively ... product portfolio, which is likely to lead to market ...
(Date:3/22/2017)... , March 22, 2017   Boston Biomedical , ... therapeutics designed to target cancer stemness pathways, today announced ... Andrews as Chief Executive Officer, effective April 24, ... Chiang J. Li , M.D., FACP, who has led ... ago. Under his leadership, Boston Biomedical has grown from ...
Breaking Biology Technology: