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Three New RNAi-Related Patent Applications Published By US Patent Office

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Title: Further Use of Protein Kinase N Beta. Number: 20040106569. Filed: Aug. 14, 2003. Lead Inventor: Anke Klippel-Giese, Atugen.

According to the patent application's abstract, the invention "is related to the use of protein kinase N beta or a fragment or derivative thereof as a downstream target of the PI-3 kinase pathway, preferably as a downstream drug target of the PI 3-kinase pathway."

The application specifically claims a method of treating a disease or pathological condition associated with the dysregulation of the PI-3 kinase pathway, in particular cancer or a precancerous growth, by administering a compound (including an siRNA) that inhibits the activity of protein kinase N beta.

The application also claims the use of a protein kinase N beta-inhibiting siRNA to treat any of the following conditions: Bannayan-Zonana syndrome, Lhermitte-Duklos' syndrome, hamartoma-macrocephaly diseases, a mucocutaneous lesion; macrocephaly, mental retardation, gastrointestinal hamiatoma, lipoma, thyroid adenomas, fibrocystic disease of the breast, and cerebellar dysplastic gangliocytoma.


Title: Intravascular Delivery of Non-Viral Nucleic Acid. Number: 20040106567. Filed: June 30, 2003. Lead Inventor: James Hagstrom, Mirus.

The invention comprises "designing a polynucleotide, such as an siRNA, for transfection," the patent application's abstract states.

"The polynucleotide is inserted into a mammalian vessel such as an artery," it adds. "Prior to insertion, subsequent to insertion, or concurrent with insertion, volume in the vessel is increased allowing polynucleotide delivery to the parenchymal cell."

The patent application also covers the delivery of antisense and morpholino polynucleotides.


Title: RNA-Splicing and Processing-Directed Gene Silencing and the Relative Applications Thereof. Number: 20040106566. Filed: May 15, 2003. Lead Inventor: Shi-Lung Lin, University of Southern California.

"The present invention relates to a method for generating a recombinant gene composition, which is able to elicit specific gene silencing effects through RNA splicing and/or processing mechanisms, and the relative utilization thereof," the patent application's abstract states. "The recombinant gene molecule so generated is useful not only for delivering desirable gene function into the transfected cells thereof but also for suppressing undesirable gene function in the transfected cells, respectively or simultaneously.

"Furthermore, the derivative products of this novel recombinant gene have multiple utilities in probing gene function, validating drug [targets], and treating as well as preventing gene-related diseases," it adds.

The patent application specifically claims a method of inducing posttranscriptional gene silencing effects comprising the construction of "a recombinant gene composition containing a functional RNA polymerase promoter and at least an intron flanked with a plurality of exons, wherein said intron can be cleaved out of the exons by RNA splicing and/or processing for gene silencing and said exons can be linked to form a gene with desired function;" the introduction of the gene composition into a cell or in vivo; the generation of RNA transcripts of the gene composition; and the release of "the metabolic products of said intron by RNA splicing/processing mechanisms, so as to provide gene silencing effects against the genes containing sequences homologous to said intron."

The application states that the intron can be a nucleic acid template encoding small-interfering RNAs, short-hairpin RNAs, microRNAs, and double-stranded RNAs.

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