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Carnegie Mellon researchers develop safe economical gene delivery system

Carnegie Mellon Universtity : 21 December, 2003  (Technical Article)
Researchers from Carnegie Mellon and the University of Pittsburgh will receive $1.02 million over the next four years from the National Institutes of Health to create safer methods for using gene therapy in treating patients.
Specifically, the researchers are developing nanostructure ceramic carriers called NanoCaps for delivering DNA into cells to help fight diseases or for regenerative medicine applications.

Prashant Kumta, a professor of materials science and biomedical engineering at Carnegie Mellon, is working with Charles Sfeir, an assistant professor of oral medicine and pathology at the University of Pittsburgh, to conduct gene delivery without the use of viruses.

'Nonviral methods represent only a fraction of the gene delivery field, but they are catching up with viral vectors,' Kumta said.

For more than a decade, researchers have been working to alleviate disease through gene therapy. In this type of treatment, a gene is delivered to cells, allowing them to produce their own therapeutic proteins.

'Traditionally, gene therapy involves transferring genes by using viruses that could infect cells, release the DNA and take over the cells' machinery to produce the desirable proteins to fight a variety of diseases or for tissue-engineering purposes,' Kumta said. 'Tissue engineering is an emerging field to regenerate tissues lost by trauma, disease or congenital abnormalities. The general approach taken by tissue engineering researchers is to incorporate a protein or modify the surface with molecules that will help cells obtain the necessary signals to form a specific tissue on an engineered scaffold in the body,' he said.

'However, we now have the capability to deliver the plasmid DNA directly to cells in an effective manner,' said Sfeir.

The newly developed ceramic NanoCap particles, containing the condensed DNA, are 10,000 times smaller than a single human hair. These nano-sized calcium phosphate carriers enter the cell and release the DNA molecules, which code and direct the cell's living pattern and generate disease-fighting proteins. The approach can also be useful in engineering new tissue at certain specific sights in the body, according to researchers.

In the past, these nonviral methods of gene delivery were considered less efficient than using a live virus, researchers said. But Kumta's team is finding that non-viral delivery systems can carry larger DNA than live viruses and are less toxic. In addition, the nonviral systems can deliver DNA in a more economical fashion.

'Therefore, these newly developed materials have great potential, and will provide exciting new approaches to deliver DNA in a more controlled environment. In addition, the new materials and approaches will help manufacture smart scaffolds,' Sfeir said.
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