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Patent Awarded for RNA Process of Inhibiting Gene Expression

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The U.S. Patent and Trademark Office awarded a patent last week for the use of RNA interference to inhibit expression of a target gene in animal cells. Patent 8,283,329 was awarded on 9 October to eight inventors — including Andrew Fire and Craig Mello, winners of the 2006 Nobel Prize in Physiology and Medicine — and assigned to Carnegie Institution for Science and University of Massachusetts Medical School.

Ribonucleic acid (RNA) is genetic material related to DNA responsible for the transmission of genetic information. Those instructions give DNA the orders for building proteins to perform their functions in living organisms. RNA interference offers a way to shut down disease causing genes in animals, including humans, by inhibiting the genes from expressing, or producing the disease-causing proteins.

The patent covers the RNA interference process of using a double-stranded form of RNA, which when introduced into an organism degrades the message-carrying molecule in RNA — called messenger RNA. This messenger RNA has the instructions for expressing the disease-causing gene, but the RNA interference process in the patent disrupts delivery of the message and inactivates the gene. Inactivating the target gene thus stops the development or progression of the disease-causing agent, such as an invading viral infection or tumor growth.

The patent also covers methods of producing RNA, either derived from living organisms or synthesized in the lab. In addition, the patent specifies the methods of introducing RNA into the organism: external methods such as bathing, or given orally such as mixing with food, or direct injections in the cells or organisms.

The patent covers only the double-stranded RNA process and not other methods for using RNA to inhibit gene expression. The patent distinguishes the double-stranded process from techniques using an RNA strand with a complementary sequence to the target gene, called antisense, as well as methods based on triple helical nucleic acid structures and co-suppression of unlinked but similar genes.

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