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Press Release

Chriss Swaney

For immediate release:
March 31, 2003

Carnegie Mellon Researchers Develop New Delivery System for Gene Therapy

PITTSBURGH—Carnegie Mellon University researchers have developed a new system to improve the delivery of genes, which could have the potential cure for several genetically transmitted diseases.

Under the direction of Prashant Kumta, a professor of materials science, engineering and biomedical engineering, researchers are creating nano-particles capable of delivering DNA-based therapies for potential use in a variety of cancers and several genetic diseases.

"We have developed a new system that will help physicians deliver their genetic life-saving payloads into enough cells to do some good," said Kumta, who has applied for a patent on the non-viral gene delivery system.

The new non-viral gene delivery system is designed to circumvent the current challenges of using viruses in gene therapy treatment, according to Charles Sfeir, a member of Kumta's research team and a molecular biologist and assistant professor of oral medicine and pathology at the University of Pittsburgh.

The challenges facing the development of efficient and safe delivery systems for gene therapy remain daunting. Most early trials of gene delivery used retroviruses, replacing their harmful components with genes intended to help treat disease, such as muscular dystrophy, cystic fibrosis and even brain cancer.

But viral-based gene delivery systems, especially the ones using a retrovirus system, have to infect the cell membrane to introduce the genes that can help cure disease in cells. Those retroviruses lucky enough to make it through the tough membranes to infect the damaged cells do so in a very unpredictable manner. And in some cases, that unpredictability of the new gene interrupts an important healing sequence, harming the cell by potentially causing mutations leading to cancer, and even triggering a dormant oncogene - oncogene is a gene that helps cells undergo dramatic change - in the body resulting in an incurable life-threatening disease.

So, Carnegie Mellon researchers have developed a simple delivery system that uses nanoparticles to deliver DNA to cells making gene therapy safer for patients. These nanoparticles will help deliver life-saving DNA much more effectively than existing methods. The nanoparticle DNA complex can be delivered in several forms such as injectable fluids, skin patches, creams or lotions and aerosol inhalers.

The research is funded through a $1 million grant from the National Science Foundation with initial seed funding from the Pittsburgh Tissue Engineering Institute.


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