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MicroRNA a Key Component of Gene Therapy Study

A team of scientists from the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) in Milan, Italy reported a potential breakthrough using a set of genes that is regulated by a molecule known as microRNA (tiny fragments of ribonucleic acid). The use of these molecules in gene therapy delivery is especially advantageous because they are able to "turn off" the identity of the therapeutic gene in cells, effectively "hiding" that gene from an immune system that would otherwise destroy it. A healthy immune system will often attack genetic material delivered into the body as a foreign substance, thereby thwarting its intended therapeutic functions.

The HSR-TIGET research team led by Luigi Naldini, MD, PhD, and Brian D. Brown, PhD, injected laboratory mice with a microRNA-regulated gene and detected no immune response. "The discovery of microRNAs has changed our understanding of biology," said Brown. "Almost every week a new study comes out implicating them in some cellular process or pathology. Now we can take advantage of this information for creating therapies."

The team at HSR-TIGET will continue to use microRNAs to aid in the development of viable gene therapies for diseases such as hemophilia and cancer.

Study results were reported at the 9th Annual Meeting of the American Society of Gene Therapy on June 3, 2006 in Baltimore.

Source: American Society of Gene Therapy press release dated June 3, 2006


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