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USPTO Publishes Seven RNAi-Related Patent Applications: Nov 6, 2008

Title: Compositions for Silencing the Expression of VDAC1 and Uses Thereof
Number: 20080267931
Filed: Oct. 15, 2006 PCT Filed: Oct. 15, 2006
Lead Inventor: Varda Shoshan-Barmatz, Ben Gurion University
The invention, the patent application’s abstract states, “relates generally to the down-regulation of [the expression of the] mitochondrial protein voltage-dependent anion channel … by RNAi or antisense therapy. In particular, the … invention is directed to VDAC1-silencing molecules useful in regulating cell proliferation and to pharmaceutical compositions comprising same useful in the treatment of diseases associated with aberrant cell proliferation.”

Title: Methods for Treating Cancer Using Agents that Inhibit Wnt16 Signaling
Number: 20080267951
Filed: July 11, 2005 PCT Filed: July 11, 2005
Lead Inventor: Liang You, University of California, San Francisco
“This invention relates to methods of inhibiting the growth of cells, in particular cancer cells that over express Wnt16 protein,” the patent application’s abstract states. “The methods comprise contacting the cell with an agent [that binds to Wnt16 mRNA or Wnt16 protein, interferes with Wnt16 signaling or inhibits binding of the Wnt16 protein to another protein, such as a Frizzled receptor.”
The application specifically claims the use of an siRNA that inhibits Wnt16 signaling.

Title: siRNA Targeting Forkhead Box P3
Number: 20080268457
Filed: June 6, 2008
Lead Inventor: Anastasia Khvorova, Dharmacon (Thermo Fisher Scientific)
“Efficient sequence-specific gene silencing is possible through the use of siRNA technology,” the patent application’s abstract states. “By selecting particular siRNAs by rational design, one can maximize the generation of an effective gene-silencing reagent, as well as methods for silencing genes. Methods, compositions, and kits generated through rational design of siRNAs are disclosed including those directed to nucleotide sequences for FOXP3.”

Title: RNA Interference-Mediating Small RNA Molecules
Number: 20080269147
Filed: Dec. 6, 2006
Lead Inventor: Thomas Tuschl, Max Planck Institute
According to the patent application’s abstract, “double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference. Using a Drosophila in vitro system, we demonstrate that [19 to 23 nucleotide] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA.
“Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA,” the abstract adds. “Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex.”

Title: Modified Small Interfering RNA Molecules and Methods of Use
Number: 20080269148
Filed: Sept. 30, 2005 PCT Filed: Sept. 30, 2005
Lead Inventor: Jang Han, Novartis
The invention, the patent application’s abstract states, “provides double-stranded RNA molecules that mediate RNA interference in target cells, preferably hepatic cells. The invention also provides double-stranded RNA molecules that are modified to be resistant to nuclease degradation, which inactivates a virus, and more specifically, hepatitis C virus. The invention also provides a method of using these modified RNA molecules to inactivate virus in mammalian cells and a method of making modified small interfering RNAs using human Dicer.”
The abstract also states that the invention “provides modified RNA molecules that are modified to include a dsRNA or siRNA wherein one or more of the pyrimidines in the RNA molecule are modified to include 2'-fluorine. The invention also provides dsRNA or siRNA in which all pyrimidines are modified to include a 2'-fluorine. The invention provides that the 2'-fluorine dsRNA or siRNA molecule is further modified to include a two base deoxynucleotide ‘TT’ sequence at the 3' end of the molecule.”

Title: Inhibitors of RTP801 and Their Use in Disease Treatment
Number: 20080269156
Filed: Feb. 26, 2008
Lead Inventor: Elena Feinstein, Quark Pharmaceuticals
The invention “provides novel molecules, compositions, methods, and uses for treating microvascular disorders, eye diseases, respiratory conditions, and hearing disorders based upon inhibition of the RTP801 gene and/or protein,” the patent application’s abstract states.

Title: Novel shRNA Molecules and Methods of Use Thereof
Number: 20080269474
Filed: Nov. 9, 2007
Inventor: Donald Rao, Murex Pharmaceuticals

The invention “relates to certain novel shRNA molecules and methods of use thereof” to reduce target gene expression, the patent application’s abstract states. “Such methods generally comprise providing a cell with one or more precursor nucleic acid sequences that encode two or more RNA molecules. A first RNA molecule comprises a double-stranded sequence, which includes a guide-strand sequence that is complementary to a portion of an mRNA transcript encoded by the target gene. In addition, a second RNA molecule comprises a second double-stranded sequence, which includes a second guide-strand sequence that is partially complementary to a portion of the mRNA transcript encoded by the target gene. Preferably, the second guide-strand sequence comprises one or more bases that are mismatched with a nucleic acid sequence of the mRNA transcript encoded by the target gene.”

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