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USPTO Publishes Four RNAi-Related Patent Applications: May 29, 2008

Title: Double-Strand Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
Number: 20080119427
Filed: June 2, 2005
Lead Inventor: Balkrishen Bhat, Isis Pharmaceuticals
The invention “provides double-stranded compositions wherein each strand is modified to have a motif defined by positioning of beta-D-ribonucleosides and sugar modified nucleosides,” the patent application’s abstract states. “More particularly, the … compositions comprise one strand having a gapped motif and another strand having a gapped motif, a hemimer motif, a blockmer motif, a fully modified motif, a positionally modified motif, or an alternating motif. At least one of the strands has complementarity to a nucleic acid target.
“The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes,” the abstract adds. “In some embodiments, the compositions of the … invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The present invention also provides methods for modulating gene expression.”

Title: Compositions and Methods for Angiogenesis-Related Molecules and Treatments
Number: 20080119430
Filed: Aug. 9, 2005 PCT Filed: Aug. 9, 2005
Lead Inventor: Gabriel Kremmidiotis, Bionomics
The patent application, its abstract states, claims “short interfering RNA molecules [that] modulate the expression of an angiogenesis-related gene by RNA interference,” as well as “short hairpin RNA molecules comprising said siRNA molecules.
“These molecules can target all, or specific, isoforms of the gene,” it adds, and the application further claims “the use of these molecules and of isoforms of the gene for the treatment and diagnosis of angiogenesis-related disorders.”

Title: Regulation of Oncogenes by microRNAs
Number: 20080119436
Filed: Oct. 22, 2007
Lead Inventor: Frank Slack, Yale University (Asuragen)
According to the patent application’s abstract, the invention comprises “naturally occurring miRNAs that regulate human oncogenes and methods of use thereof. … Suitable nucleic acids for use in the methods and compositions … include, but are not limited to, pri-miRNA, pre-miRNA, mature miRNA, or fragments of variants thereof that retain the biological activity of the mature miRNA and DNA encoding a pri-miRNA, pre-miRNA, mature miRNA, fragments, or variants thereof, or regulatory elements of the miRNA. The compositions are administered to a subject prior to administration of a cytotoxic therapy in an amount effective to sensitize cells or tissues to be treated to the effects of the cytotoxic therapy.”

Title: RNAi-Based Method for Selecting Transfected Eukaryotic Cells
Number: 20080120733
Filed: June 16, 2005 PCT Filed: June 16, 2005
Lead Inventor: Martin Hafner, Helmholtz Centre for Infection Research
“The invention relates to a method for the production of a eukaryotic cell selectable by inactivation or reduction of an endogenous gene function [by introducing] one or more vectors into the cell and [expressing] a siRNA and preferably shRNA coded by the one or more vectors,” the patent application’s abstract states. These vectors are directed against, and inactivate, an endogenous selectable gene and inactivating same, while the siRNA or shRNA are the transcription product of an RNAi selection cassette [comprised of] a section of at least 19 nucleotides of the transcribed region of the gene [that is] operatively linked to a promoter and a transcription termination signal, it adds.
“Furthermore, the invention relates to a eukaryotic cell comprising an RNAi-selection cassette [that] is directed against an endogenous, selectable gene and inactivates the function of said gene,” the abstract states. “Moreover, the invention relates to methods for the production of a transgenic mammal, comprising the steps [of injecting] the embryonic stem cell [or] an embryonic stem cell … in blastocysts of a mammal, [transferring] the blastocysts into the uterus of a mammal, and carrying the transgenic mammal to full term. Finally, the invention relates to a method for the production of a transgenic plant.”

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