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Seven RNAi-Related Patent Applications Published by the USPTO: Jan 5, 2006

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Title: Cre-Lox-Based Method for Conditional RNA Interference

Number: 20050289659

Filed: April 19, 2005

Lead Inventor: Tyler Jacks, Massachusetts Institute of Technology

The invention, the patent application's abstract states, "relates to vectors, compositions, and methods for conditional, Cre-lox regulated, RNA interference. Vectors for use in conditional expression of a coding sequence based on a strategy in which the mouse U6 promoter is modified to include a hybrid between a LoxP site and a TATA box, and their use in conditional expression in transgenic mice are disclosed. The vectors allow for spatial and temporal control of miRNA expression in vivo."


Title: Methods and Compositions for Treating Gain-of-Function Disorders Using RNA Interference

Number: 20050288243

Filed: April 6, 2005

Lead Inventor: Zuoshang Xu, University of Massachusetts (CytRx)

The invention relates to "novel methods for treating dominant gain-of-function diseases," the patent application's abstract states. "The invention provides methods for targeting regions of the copper zinc superoxide dismutase (SOD1), which causes inherited amyotrophic lateral sclerosis, with RNAi agent. The invention further provides RNAi-resistant replacement genes containing mismatches with their respective RNAi agents. The invention also provides for vectors that express RNAi agent and RNAi-resistant replacement gene of the present invention."


Title: RNA Interference-Mediated Inhibition of RAS Gene Expression Using Short Interfering Nucleic Acid

Number: 20050288242

Filed: Aug. 20, 2004

Inventor: James McSwiggen, Sirna Therapeutics

"This invention relates to compounds, compositions, and methods useful for modulating RAS, e.g. K-RAS, H-RAS, and/or N-RAS 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 RAS, e.g. K-RAS, H-RAS, and/or N-RAS 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 RAS genes, such as K-RAS, H-RAS, and/or N-RAS."


Title: RNA Interference Compositions and Screening Methods for the Identification of Novel Genes and Biological Pathways

Number: 20050287668

Filed: Oct. 29, 2004

Inventor: Robert Finney, Cell Therapeutics

The invention "provides compositions and methods for enhancing RNA interference and facilitating the use of long RNA interference molecules," the patent application's abstract states. "Accordingly, the invention includes a variety of novel applications of RNA interference, including methods related to screening RNA interference molecules using reporter genes to identify biological pathways, genes, therapeutic compounds, and biomarkers."


Title: RNA Interference-Mediated Inhibition of TGF-Beta and TGF-Beta Receptor Gene Expression Using Short Interfering Nucleic Acid

Number: 20050287128

Filed: Feb. 9, 2005

Lead Inventor: Roberto Guerciolini, Sirna Therapeutics

"This invention relates to compounds, compositions, and methods useful for modulating TGF-beta and/or TGF-betaR 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 TGF-beta and/or TGF-betaR gene expression and/or activity by RNA interference using small nucleic acid molecules. … Such small nucleic acid molecules are useful, for example, for treating, preventing, inhibiting, or reducing inflammatory, respiratory, autoimmune, and/or proliferative diseases, disorders, conditions, or traits in a cell, subject or organism and any other disease, condition, trait or indication that can respond to the level of TGF-beta and/or TGF-betaR in a cell or tissue; or alternately in providing long term hematopeitic reconstitution in a subject or organism."


Title: Method for Producing Non-Human Mammal Having RNAi Phenotype

Number: 20050283845

Filed: June 3, 2005

Lead Inventor: Motoya Katsuki, Mitsubishi Chemical

"It is an object of the … invention to develop a novel method for producing mammals such as mice having an RNAi phenotype, thereby enabling the production of RNAi-expressed progenies by a new mechanism to transfer information to progenies, and to improve a method for introducing dsRNA to improve the efficiency of obtaining mammals such as mice having an RNAi phenotype," the patent application's abstract states. "The … invention provides a method for producing a non-human mammal with suppressed function of a target gene, which comprises injecting the double-stranded RNA of the target gene into the nucleus of a cell."


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

Number: 20050282188

Filed: April 4, 2005

Lead Inventor: Peter Haeberli, Sirna Therapeutics

"This invention relates to compounds, compositions, and methods useful for modulating gene expression using short interfering nucleic acid molecules," the patent application's abstract states. "In particular, the … 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 genes, such as expressed pseudogenes associated with the maintenance or development of diseases, disorders, traits, and conditions in a subject or organism. The invention also provides small nucleic acid molecules with reduced or attenuated immunostimulatory properties and methods for designing and synthesizing such small nucleic acid molecules having improved toxicologic properties while retaining RNAi activity."

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