Methods include gene silencing using short RNA molecules, or DNA constructs encoding short RNA molecules, in a wide range of organisms, including in plants and humans.
Silencing is a natural mechanism for down-regulating gene expression that is found in most complex organisms and it is the focus of tremendous activity in the life science industry.
It has been widely exploited in research for gene discovery, and for characterization of gene function.
It holds great promise as a therapeutic tool, and currently “gene therapy” applications are being developed for ailments as diverse as cancer, viral diseases and obesity. The technology is also referred to as “RNAi”, short for RNA interference.
The award of this new patent (8,097,710, issued 17th January 2012) comes as further recognition by the USPTO of the pioneering contributions of Professor Sir David Baulcombe and Dr Andrew Hamilton to the field of silencing.
The original patent application based on this work was filed by PBL in 1999, following Baulcombe and Hamilton’s ground-breaking research at The Sainsbury Laboratory in Norwich, UK and published in Science (“A Species of Small Antisense RNA in Posttranscriptional Gene Silencing in Plants”, (1999), 286, pp. 950-952).
This paper provided the first identification that short RNA molecules are the active agents of silencing, and the patent describes methods and compositions for use of such molecules for inducing silencing in living organisms.
The first of PBL’s patents to be granted by the USPTO, based on Hamilton and Baulcombe’s work, was US Patent No. 6,753,139, which issued in 2004, for methods of detecting gene silencing in plants.
In April 2010, patent number 7,704,688 issued with claims directed to detection of gene silencing in mammals.
Now, issuance of this most recent patent acknowledges the role of short RNA molecules as the common mediators of gene silencing in different species and organisms, and protects the use of short RNAs for the purpose of inducing the silencing of a target gene in a cell.
The importance of Silencing as a scientific discovery was underlined both by the award of a Nobel Prize in 2006 to Andrew Fire and Craig Mello, in recognition of their seminal publication in 1998 on the use of long dsRNA to induce silencing in nematodes, and the Lasker Foundation awarding the 2008 Albert Lasker Basic Research Award jointly to David Baulcombe (whose work demonstrated that short RNA molecules have a broad applicability as markers and inducers of gene silencing in living organisms), jointly with Gary Ruvkun and Victor Ambrose (for their combined effort in identifying the first miRNA in nematodes).
On issuing the award, the Lasker Foundation noted David Baulcombe’s contributions thus: “For discoveries that revealed an unanticipated world of tiny RNAs that regulate gene function in plants and animals”. In addition, in 2009, Professor Baulcombe was awarded a knighthood “for services to Plant Science”.
“PBL would like to record our appreciation of the excellent guidance and support provided by Kate Murashige of Morrison & Foerster, San Diego, in the complex prosecution of this portfolio of patent applications to patent issuance”, stated Gerard Bencen, PBL’s Patent Manager, who has worked closely with Kate Murashige in the prosecution of this portfolio.
PBL’s Managing Director, Dr Jan Chojecki, states “We are very pleased that our efforts in working with the US Patent Office have resulted in issuance of this patent. It is an excellent example of how innovations in a specialized field can, once recognized, have impact across many other areas of research, discovery and beneficial application. It is also a tribute to the quality of fundamental research carried out in public research laboratories in the United Kingdom. We look forward to answering any licence inquiries in connection with the issuance of this patent.”