Title: Genetic Inhibition by Double-Stranded RNA
Filed: Oct. 30, 2002
Lead Inventor: Andrew Fire, Carnegie Institution
The patent, its abstract states, claims "a process … of introducing an RNA into a living cell to inhibit gene expression of a target gene in that cell. The process may be practiced ex vivo or in vivo.
"The RNA has a region with double-stranded structure," the abstract adds. "Inhibition is sequence-specific in that the nucleotide sequences of the duplex region of the RNA and of a portion of the target gene are identical. The … invention is distinguished from prior art interference in gene expression by antisense or triple-strand methods."
Title: miR-16-Regulated Genes and Pathways as Targets for Therapeutic Intervention
Filed: Dec. 31, 2007
Lead Inventor: Mike Byrom, Asuragen
The invention, the patent application's abstract states, "concerns methods and compositions for identifying genes or genetic pathways modulated by miR-16, using miR-16 to modulate a gene or gene pathway, using this profile in assessing the condition of a patient, and/or treating the patient with an appropriate miRNA."
Title: Methods and Compositions Involving miRNA and miRNA Inhibitor Molecules
Filed: Nov. 14, 2005
Lead Inventor: David Brown, Ambion (Applied Biosystems)
The invention, the patent application's abstract states, "concerns methods and compositions for introducing miRNA activity or function into cells using synthetic nucleic acid molecules. Moreover, the … invention concerns methods and compositions for identifying miRNAs with specific cellular functions that are relevant to therapeutic, diagnostic, and prognostic applications wherein synthetic miRNAs and/or miRNA inhibitors are used in library-screening assays."
Title: Chemically Modified Short Interfering Nucleic Acid Molecules that Mediate RNA Interference
Filed: Aug. 17, 2006 PCT Filed: Aug. 17, 2006
Lead Inventor: David Morrissey, Sirna Therapeutics (Merck)
The invention, the patent application's abstract states, "relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases, and conditions that respond to the modulation of gene expression and/or activity. The … invention is also directed to compounds, compositions, and methods relating to traits, diseases, and conditions that respond to the modulation of expression and/or activity of genes involved in gene-expression pathways or other cellular processes that mediate the maintenance or development of such traits, diseases, and conditions.
"Specifically, the invention relates to double-stranded nucleic acid molecules … capable of mediating RNA interference against gene expression, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules," the abstract adds. "The … invention also relates to small nucleic acid molecules … that can inhibit the function of endogenous RNA molecules … or that can inhibit the function of RISC to modulate gene expression by interfering with the regulatory function of such endogenous RNAs or proteins associated with such endogenous RNAs, including cocktails of such small nucleic acid molecules and lipid nanoparticle formulations of such small nucleic acid molecules.
"Such small nucleic acid molecules are useful, for example, in providing compositions to prevent, inhibit, or reduce diseases, traits, and conditions that are associated with gene expression or activity in a subject or organism," it notes.
Title: Double-Stranded RNA Structures and Constructs, and Methods for Generating and Using the Same
Filed: Oct. 8, 2008
Lead Inventor: Catherine Pachuk, Nucleonics (Alnylam Pharmaceuticals)
The invention "relates to novel double-stranded RNA structures and dsRNA-expression constructs, methods for generating them, and methods of utilizing them for silencing genes," according to the patent application's abstract. "Desirably, these methods specifically inhibit the expression of one or more target genes in a cell or animal without inducing toxicity … [and] can be used to prevent or treat a disease or infection by silencing a gene associated with the disease or infection. The invention also provides methods for identifying nucleic acid sequences that modulate a detectable phenotype, such as the function of a cell, the expression of a gene, or the biological activity of a target polypeptide."
Title: Treatment of CNS Conditions
Filed: March 16, 2007 PCT Filed: March 16, 2007
Lead Inventor: Angela Sesto Yague, Sylentis
According to the patent application's abstract, the invention comprises "methods and compositions for the treatment of pathologic conditions of the central nervous system by means of intranasal administration of a composition that modulates, by means of RNA interference, the expression and/or activity of genes involved in above-mentioned conditions."
The application specifically cites the treatment of conditions including dementia and Alzheimer's, Huntington's, and Parkinson's diseases.
Title: Method of Treating Neurodegenerative Disease
Filed: Dec. 12, 2008
Inventor: Pamela Tan, Alnylam Pharmaceuticals
"Aspects featured in the invention relate to compositions and methods for inhibiting alpha-synuclein gene expression, such as for the treatment of neurodegenerative disorders," the patent application's abstract states. "An anti-SNCA agent featured herein that targets the SNCA gene can have been modified to alter distribution in favor of neural cells."
Title: LNA-Modified Phosphorothiolated Oligonucleotides
Filed: Jan. 29, 2007 PCT Filed: Jan. 29, 2007
Lead Inventor: Joacim Elmen, Karolinska Institutet (Santaris Pharma)
The invention, the patent application's abstract states, "provides oligonucleotides which comprise a dinucleotide consisting of a 5' locked nucleic acid, a phosphorothioate internucleoside linkage bond to a 3' RNA or RNA analogue. The dinucleotide reduces the strength of hybridization of the oligonucleotide to a complementary nucleic acid target.
"The modification can be used to modulate hybridization properties in both single-stranded oligonucleotides and in double-stranded siRNA complexes, particularly in oligonucleotides where the use of LNA results in excessively strong hybridization properties," the abstract notes.