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USPTO Publishes 10 RNAi-Related Patent Applications

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Title: RNA Interference-Mediated Inhibition of Gene Expression Using Chemically Modified Short Interfering Nucleic Acid
 
Number: 20070167393
 
Filed: Feb. 16, 2007
 
Lead Inventor: James McSwiggen, Sirna Therapeutics (Merck)
 
The invention, according to the patent application’s abstract, “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. Specifically, the invention relates to synthetic chemically modified small nucleic acid 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: Double-Stranded Compositions Comprising Differentially Modified Strands for Use in Gene Modulation
 
Numbers: 20070167392, 20070167391, 20070167390, 20070166734
 
Filed: Dec. 1, 2006
 
Lead Inventor: Balkrishen Bhat, Eli Lilly
 
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,” according to the patent applications’ abstracts. “More particularly, the present 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 abstract notes. “The compositions are useful for targeting selected nucleic acid molecules and modulating the expression of one or more genes. In some embodiments, the compositions of the present invention hybridize to a portion of a target RNA resulting in loss of normal function of the target RNA. The … invention also provides methods for modulating gene expression.”
 

 
Title: Compositions, Devices, and Methods for Treatment of Huntington’s Disease Through Intracranial Delivery of siRNA
 
Numbers: 20070167389
 
Filed: Aug. 8, 2006
 
Inventor: William Kaemmerer, Medtronic
 
The invention, the patent application’s abstract states, “provides devices, small interfering RNAs, and methods for treating a neurodegenerative disorder comprising the steps of surgically implanting a catheter so that a discharge portion of the catheter lies adjacent to a predetermined infusion site in a brain, and discharging through the discharge portion of the catheter a predetermined dosage of at least one substance capable of inhibiting production of at least one neurodegenerative protein. The … invention also provides valuable small interfering RNA vectors, systems, and methods for treating Huntington's disease in vivo without impairment of cell endoplasmic reticulum, spontaneous motor activity, or locomotor activity of a patient.”
 

 
Title: Modified Polynucleotides for Use in RNA Interference
 
Numbers: 20070167384
 
Filed: April 1, 2004 PCT Filed: April 1, 2004
 
Lead Inventor: Devin Leake, Dharmacon (Thermo Fisher Scientific)
 
The patent application, its abstract states, claims “methods and compositions for performing RNA interference comprising a wide variety of stabilized siRNAs suitable for use in serum-containing media and for in vivo applications, such as therapeutic applications. … These siRNAs permit effective and efficient applications of RNA interference to applications such as diagnostics and therapeutics through the use of one or more modifications including orthoesters, terminal conjugates, modified linkages and 2' modified nucleotides. Uniquely modified siRNAs have been developed that reduce off-target effects incurred in gene-silencing,” the abstract adds. “The modifications include phosphorylation of the first 5' terminal antisense nucleotide; 2' carbon modifications of the first and second or first, second, and third 5' terminal antisense nucleotides; and optionally 2' carbon modifications of the first and second or first, second, and third 5' terminal sense nucleotide.”
 

 
Title: microRNAs and Related Nucleic Acids
 
Numbers: 20070166724
 
Filed: Jan. 19, 2006
 
Lead Inventor: Isaac Bentwich, Rosetta Genomics
 
The patent application, its abstract states, claims “novel polynucleotides associated with disease. The polynucleotides are miRNAs, miRNA precursors, and associated nucleic acids. Methods and compositions are described that can be used for diagnosis, prognosis, and treatment of disease. Also described herein are methods that can be used to identify modulators of the disease-associated polynucleotides.”
 

 
Title: Small Interfering RNA Libraries and Methods of Synthesis and Use
 
Numbers: 20070166720
 
Filed: May 10, 2004     
 
Lead Inventor: Mark Nichols, University of Pittsburgh (Cellumen)
 
“In one aspect, the invention provides a random or semi-random siRNA encoding library,” the patent application’s abstract states. Other aspects of the invention pertain to “methods for construction of random or semi-random siRNA encoding libraries … [and] vector systems for use in constructing siRNA libraries and/or that can express single siRNAs and siRNA libraries both constitutively and in an inducible fashion.
 
”In another aspect, the invention provides a method of using an siRNA library,” the abstract states. “The siRNA library is introduced into a population of cells, [which] then is subjected to a selection process to select a subpopulation of cells exhibiting a different behavioral, biochemical, chemical, functional, molecular, morphological, phenotypic, or physical property from the remainder of population. Following the selection process, the subpopulation of cells can be isolated, analyzed, and/or cloned as desired. Such analysis of the subpopulation can be identification and sequencing of the siRNA species responsible for the different properties of the subpopulation relative to the remainder of the population. Alternatively, the subpopulation can be further analyzed by genomic, proteomic, and/or cellomic assays. Where such genomic, proteomic, and/or cellomic assays are employed, the method can produce several useful bioinformatics products. Specific siRNAs identified through this process may have direct therapeutic value.”
 

 
Title: Method for Evaluating RNAi Activity and miRNA Activity
 
Numbers: 20070166716
 
Filed: Nov. 18, 2004 PCT Filed: Nov. 18. 2004
 
Lead Inventor: Yuki Naito, Bio-Think Tank
 
The invention, according to the patent application’s abstract, “provides methods whereby an RNAi activity and a miRNA activity can be evaluated simply and conveniently … by supplying a target-expressing molecule, which is a polynucleotide containing at least a target sequence and an expression regulatory region regulating the expression of an RNA containing the target sequence, and a subject nucleic acid molecule to be evaluated on whether or not it has RNAi activity on the RNA containing the target sequence into an expression system in which the target-expressing molecule can express the RNA containing the target sequence.”

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