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Five RNAi-Related Patent Applications Published by the US Patent Office: Oct 8, 2004


Title: Allele-Specific RNA Interference. Number: 20040192629. Filed: Nov. 4, 2003. Lead Inventor: Zuoshang Xu, University of Massachusetts Medical School.

According to the patent application’s abstract, the invention relates to “human diseases caused by dominant, gain-of-function mutations developed in heterozygotes bearing one mutant and one wild-type copy of a gene.

“Because the wild-type gene often performs important functions, whereas the mutant gene is toxic, any therapeutic strategy must selectively inhibit the mutant while retaining wild-type gene expression,” the abstract states. “The present invention includes methods of specifically inhibiting the expression of a mutant allele, while preserving the expression of a co-expressed wild-type allele using RNAi, a therapeutic strategy for treating genetic disorders associated with dominant, gain-of-function gene mutations.”

The invention also comprises “small interfering RNAs and small hairpin RNAs that selectively suppress mutant, but not wild-type, expression of copper zinc superoxide dismutase, which causes inherited amyotrophic lateral sclerosis,” the abstract notes.

Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid. Number: 20040192626. Filed: May 23, 2003. Lead Inventor: James McSwiggen, Sirna Therapeutics.

“The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic discovery applications,” the patent application’s abstract states. “Specifically, the invention relates to synthetic chemically modified small nucleic acid molecules, such as short interfering nucleic acid, short interfering RNA, double-stranded RNA, micro-RNA, and short hairpin RNA molecules capable of mediating RNA interference against target nucleic acid sequences. The small nucleic acid molecules are useful in the treatment of any disease or condition that responds to modulation of gene expression or activity in a cell, tissue, or organism.”

Title: Modulation of HIV Replication by RNA Interference. Number: 20040191905. Filed: Nov. 24, 2003. Lead Inventor: Mario Stevenson, University of Massachusetts Medical School.

The patent application’s abstract states that the invention comprises “small interfering RNAs and vectors encoding one or more siRNAs, including short hairpin siRNAs, that are sufficiently homologous to a portion of the HIV genome to mediate RNA interference in vivo.”

Also covered by the application “are methods wherein siRNAs, or vectors encoding siRNAs, are administered to prevent or inhibit HIV infection in a subject, cell or tissue,” the abstract adds.

Title: Oligoribonucleotides and Ribonucleases for Cleaving RNA. Number: 20040191773. Filed: Feb. 21, 2003. Inventor: Stanley Crooke, Isis Pharmaceuticals.

The patent application’s abstract states that the invention comprises “oligomeric compounds including oligoribonucleotides and oligoribonucleosides … that have subsequences of 2’-pentoribofuranosyl nucleosides that activate dsRNase.

“The oligoribonucleotides and oligoribonucleosides can include substituent groups for increasing binding affinity to complementary nucleic acid strand as well as substituent groups for increasing nuclease resistance,” the abstract adds. “The oligomeric compounds are useful for diagnostics and other research purposes, for modulating the expression of a protein in organisms, and for the diagnosis, detection and treatment of other conditions susceptible to oligonucleotide therapeutics.”

The invention also comprises “mammalian ribonucleases, i.e., enzymes that degrade RNA, and substrates for such ribonucleases,” the abstract notes. “Such a ribonuclease is referred to herein as a dsRNase, wherein ‘ds’ indicates the RNase’s specificity for certain double-stranded RNA substrates.”

Title: Compositions and Methods for Diagnosing and Treating Autoimmune Diseases. Number: 20040191818. Filed: Feb. 26, 2004. Lead Inventor: Margot Mary O’Toole, Wyeth.

The patent application, its abstract states, covers “compositions and methods for diagnosing, preventing, or treating lupus nephritis, systemic lupus erythematosus, or other autoimmune diseases.”

The abstract notes that “lupus-related genes are identified in the present invention, [and that] these genes are differentially expressed in lupus-affected or lupus-predisposed tissues as compared to disease-free tissues. The genes and their expression products can be used as markers for diagnosing or monitoring SLE or LN. Modulators of the expression or protein activities of these genes can be used for the prevention or treatment of SLE/LN or other autoimmune diseases,” it notes.

The application specifically claims a pharmaceutical composition, with an RNA interference molecule as its active component, that is capable of “modulating the expression of a gene which is differentially expressed in pre-symptomatic lupus-affected or predisposed tissues relative to disease-free tissues.”


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