Gene silencing technique gets patent

An important discovery in modern molecular biology is that double-stranded RNA can quash the activity of specific genes in plants, animals, and fungi. In 1997, Dr. Andrew Fire of the Carnegie Institution of Washington and Dr. Craig Mello of the University of Massachusetts Medical School, and their team found that by specially designing RNA with two strands they could silence targeted genes. Their discovery, called RNA interference (RNAi) was recently patented (US Patent 6,506,559 B1), and it has been widely licensed in the U.S., Europe, and Japan.

From the Carnegie Institution:
New milestone for gene silencing

Washington, DC, February 11, 2003. One of the most important breakthroughs in modern molecular biology is the discovery that double-stranded RNA can quash the activity of specific genes in plants, animals, and fungi. And now this method has been made widely available to researchers so they can better understand gene function and develop new ways to fight disease.

In 1997, Dr. Andrew Fire of the Carnegie Institution of Washington and Dr. Craig Mello of the University of Massachusetts Medical School, and their team found that by specially designing RNA with two strands they could silence targeted genes. Their discovery, called RNA interference (RNAi) was recently patented (US Patent 6,506,559 B1), and it has been widely licensed in the U.S., Europe, and Japan to address a broad range of research questions.

“I am extremely pleased that RNAi has been made freely available to researchers in universities and similar institutions. I look forward to astonishing discoveries that will result from its use,” remarked Fire.

“Dr. Fire’s work has excited every one of us here at the institution,” said Tom Urban, chairman of the Carnegie board. It is a vivid example of how Carnegie’s commitment to basic research in unique areas yields extraordinary results, which can benefit humankind.”

The singled-stranded RNA molecule is commonly known for its role shuttling the genetic code contained in DNA from the cell’s nucleus to ribosomes where proteins are made, and for making sure that the proteins are correctly constructed to perform their functions in living organisms. As Fire explains: “The first strand of our double-stranded RNA molecule has the ribonucleotide sequence that matches the nucleotide sequence in the targeted gene. The second strand of RNA has a complementary sequence to that in the target gene. When it’s introduced to an organism, it degrades a specific Messenger RNA (mRNA) molecule, which disrupts the message-carrying process and inactivates the gene.” This finding opens up the possibility of treating various types of diseases by either shutting down the disease-causing gene, or by directing researchers to appropriate pathways for effective drug development.

Paul Kokulis, counsel for Morgan Lewis, the firm involved in obtaining the patent stated: “This is the first of several U.S. patents that Carnegie and the University of Massachusetts expect to obtain on various aspects of this important invention. It has been extensively licensed and it is being widely used both as a research tool and for the development of products including drugs that will combat diseases such as cancer and HIV.”


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