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Seven RNAi-Related Patent Applications Published by the USPTO: Sep 9, 2005

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Title: Antisense Oligonucleotides and RNA Interfering Molecules Targeting PAK4

Number: 20050191672

Filed: Jan. 27, 2005

Lead Inventor: Ian James Popoff, Agouron Pharmaceuticals

According to the patent application's abstract, the invention comprises "compositions and methods for modulating the expression of the serine/threonine kinase PAK4. In particular, the invention relates to antisense compounds, particularly oligonucleotides and double-stranded RNA molecules, which specifically hybridize to nucleic acid molecules encoding PAK4. The oligonucleotides and RNA molecules decrease or inhibit PAK4 expression and thus, can be used in target identification and/or validation, to examine PAK4 pathways and the cellular effects of PAK4 expression, and to diagnose and/or treat abnormal cell growth or inflammation associated with PAK4 expression," the abstract adds.


Title: RNA Interference-Mediated Treatment of Polyglutamine (polyQ) Repeat Expansion Diseases Using Short Interfering Nucleic Acid

Number: 20050191638

Filed: April 24, 2004

Inventor: James McSwiggen, Sirna Therapeutics

"The … invention concerns compounds, compositions, and methods for the study, diagnosis, and treatment of diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of gene expression and/or activity," the patent application's abstract states. "The … invention also concerns compounds, compositions, and methods relating to diseases and conditions associated with polyglutamine repeat (polyQ) allelic variants that respond to the modulation of expression and/or activity of genes involved in polyQ repeat gene expression pathways or other cellular processes that mediate the maintenance or development of polyQ repeat diseases and conditions such as Huntinton disease and related conditions such as progressive chorea, rigidity, dementia, and seizures, spinocerebellar ataxia, spinal and bulbar muscular dystrophy, dentatorubropallidoluysian atrophy, and any other diseases or conditions that are related to or will respond to the levels of a repeat expansion protein in a cell or tissue, alone or in combination with other therapies. Specifically, the invention relates to small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, microRNA, and short hairpin RNA molecules capable of mediating RNA interference against the expression disease related genes or alleles having polyQ repeat sequences."


Title: RNA Interference-Mediated Inhibition of Human Immunodeficiency Virus Gene Expression Using Short Interfering Nucleic Acid

Number: 20050191618

Filed: Aug. 20, 2004

Lead Inventor: James McSwiggen, Sirna Therapeutics

According to the patent application's abstract, "this invention relates to compounds, compositions, and methods useful for modulating human immunodeficiency virus gene expression using short interfering nucleic acid molecules. This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of human immunodeficiency virus gene expression and/or activity by RNA interference using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, microRNA, and short hairpin RNA molecules and methods used to modulate the expression of HIV genes," the abstract adds. "The small nucleic acid molecules are useful in the treatment of HIV infection, AIDS, and/or diseases and conditions related to HIV infection and/or AIDS in a subject or organism."


Title: Compositions and Methods Using RNA Interference for Control of Nematodes

Number: 20050188438

Filed: Feb. 22, 2005

Lead Inventor: Peifeng Ren, BASF

"The … invention concerns double stranded RNA compositions and transgenic plants capable of inhibiting expression of essential genes in parasitic nematodes, and methods associated therewith," the patent application's abstract states. "Specifically, the invention relates to the use of RNA interference to inhibit expression of a target essential parasitic nematode gene selected from the group consisting of a parasitic nematode cytosolic chaperonin gene, a parasitic nematode gene encoding heat shock protein-90, a parasitic nematode gene homologous to the C. elegans Y65B4BR.5a gene, and a parasitic nematode gene homologous to a C. elegans pat-10 gene, and relates to the generation of plants that have increased tolerance to parasitic nematodes."


Title: RNA Interference-Mediated Inhibition of Intercellular Adhesion Molecule (ICAM) Gene Expression Using Short Interfering Nucleic Acid

Number: 20050187174

Filed: Aug. 20, 2004

Lead Inventor: Ivan Richards, Sirna Therapeutics

"This invention relates to compounds, compositions, and methods useful for modulating intercellular adhesion molecule (ICAM) gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "This invention also relates to compounds, compositions, and methods useful for modulating the expression and activity of other genes involved in pathways of ICAM gene expression and/or activity by RNA interference using small nucleic acid molecules. In particular, the instant invention features small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, microRNA, and short hairpin RNA molecules and methods used to modulate the expression of ICAM genes."


Title: Method of Treating Neurodegenerative Disease

Number: 20050186591

Filed: Nov. 17, 2004

Lead Inventor: David Bumcrot, Alnylam Pharmaceuticals

According to the patent application's abstract, "aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein (SNCA) gene expression, such as for the treatment of neurodegenerative disorders. An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells."


Title: Interspersed Repetitive Element RNAs as Substrates, Inhibitors, and Delivery Vehicles for RNAi

Number: 20050186589

Filed: Nov. 8, 2004

Lead Inventor: Timothy Kowalik, University of Massachusetts

"The … invention provides methods for identifying druggable targets in assays that feature compositions, cells and/or organisms having interspersed repetitive element (IRE) RNAs and an RNA interference pathway," the patent application's abstract states. "Methods for identifying therapeutic agents and creating vaccines are also featured. The invention further provides methods for inhibiting RNAi involving IRE RNAs or inhibitory derivatives thereof. The invention also provides compositions for delivering siRNA and miRNA molecules derived from IRE loci and methods of use thereof. Therapeutic methods are also featured."

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