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Jumping gene could provide non-viral alternative for gene therapy

into cells.

Although piggyBac is not as successful as the virus at integrating into DNA, "we could potentially make a hyperactive version of piggyBac, like they did for Sleeping Beauty, which might be as good or better than retroviruses," Dr. Kaminski says. "I think we'll do it or somebody will. I think it's a safer method."

"At the moment, unless something new comes out, it's the only way to go because viruses have been killing people," says Dr. Moisyadi, who has avoided viruses in his transgenesis studies.

"One of our next goals is to use transposons to deliver a radio-protective gene, called manganese superoxide dismutase, to potentially protect normal tissue from radiation damage," Dr. Kaminski says.

In cancer, he suspects gene therapy will focus on this type of modification of normal tissue for protective purposes as well as manipulating the immune response. However, it has broad applications for correcting single gene disorders, such as hemophilia, sickle cell disease and muscular dystrophy.


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Source:Medical College of Georgia


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