Transposons: Non-Viral Alternative for Gene Therapy

Filed in archive Drugs, Vaccines and Therapeutics , Genomics, Proteomics and Bioinformatics on October 4, 2006

Scientists are exploring the potential of using the piggyBac transposon, a jumping gene first identified in a cabbage eating moth, and Sleeping Beauty, a transposon first found "asleep" in fish, as alternatives to viruses used in gene therapy.

"If we want to add a therapeutic gene, we can put it within the transposon and use it to deliver the gene into the cell." [snip] "You can use these wherever retroviruses have been used."

While Sleeping Beauty has been demonstrated to correct genetic diseases such as haemophilia in mouse models, piggyBac has shown superior potentials. Scientists say that if the integration targets of these transposons can be refined and made very specific, they may present a safer and viable alternative to using viruses.

The study has been published in PNAS. For an overview, see the news report from the Medical College of Georgia.