Title: LNA Compositions and Uses Thereof
Filed: Sept. 4, 2002
Lead Inventor: Jesper Wengel, Exiqon
The patent, its abstract states, covers "modified LNA units … that comprises unique base groups. Desirable nucleobase and nucleosidic base substitutions can mediate universal hybridization when incorporated into nucleic acid strands. The novel LNA compounds may be used in a wide variety of applications, such as PCR primers, sequencing, synthesis of antisense oligonucleotides, diagnostics, and the like."
Title: RNA Interference-Mediating Small RNA Molecules
Filed: April 27, 2004
Lead Inventor: Thomas Tuschl, Max Planck (Alnylam Pharmaceuticals)
"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," the patent's abstract states. "Using a Drosophila in vitro system, we demonstrate that 19 23 [nucleotide] short RNA fragments are the sequence-specific mediators of RNAi. The short interfering RNAs are generated by an RNase III-like processing reaction from long dsRNA. Chemically synthesized siRNA duplexes with overhanging 3' ends mediate efficient target RNA cleavage in the lysate, and the cleavage site is located near the center of the region spanned by the guiding siRNA. Furthermore, we provide evidence that the direction of dsRNA processing determines whether sense or antisense target RNA can be cleaved by the produced siRNP complex," the abstract adds.
Inventor: Oct. 12, 2005
According to the patent application's abstract, the invention comprises "methods and compositions useful in target sequence suppression, target sequence validation, and target sequence down regulation. The invention provides polynucleotide constructs useful for gene silencing or RNA down regulation, as well as cells, plants, and seeds comprising the polynucleotides. The invention also provides a method for using microRNA to silence a target sequence or to down regulate RNA."
Title: Allele-Specific RNA Interference
Filed: Sept. 30, 2005
Inventor: Zuoshang Xu, University of Massachusetts
"Human diseases caused by dominant, gain-of-function mutations develop in heterozygotes bearing one mutant and one wild-type copy of a gene," the patent application's abstract states. "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 … 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 includes 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 states. "The present invention further provides asysmmetric siRNAs and shRNAs with enhanced efficacy and specificity and mediating RNAi."
Title: RNA Interference-Mediated Inhibition of MAP Kinase Gene Expression or Expression of Genes Involved in MAP Kinase Pathway Using Short Interfering Nucleic Acid
Filed: April 25, 2003
Lead Inventor: James McSwiggen, Sirna Therapeutics
The invention "concerns methods and reagents useful in modulating MAP kinase 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 small nucleic acid molecules … capable of mediating RNA interference against c-JUN, JNK, p38, and ERK gene expression, useful in the treatment of cancer, inflammation, obesity and insulin resistance."