Title: RNA Interference-Mediating Small RNA Molecules
Number: 20050234007
Filed: June 5, 2005
Lead Inventor: Thomas Tuschl, Max Planck
"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," the patent application's abstract states. "Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide long] 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.
Title: siRNA Delivery into Mammalian Nerve Cells
Number: 20050234000
Filed: Dec. 10, 2004
Lead Inventor: Gordon Mitchell, University of Wisconsin-Madison
"The … invention relates to methods of affecting expression of a target gene, suitably brain-derived neurotrophic factor (BDNF) or related genes in a nerve cell in the central nervous system of a mammal," the patent application's abstract states. "The method includes formulating and delivering an siRNA composition to a target site on the mammal to affect expression of the target gene in the nerve cell, wherein the target site is cerebrospinal space or muscle tissue innervated by a nerve cell, to down-regulate the target gene. Also disclosed are kits for use in practicing the novel methods of in vivo siRNA.delivery into target cells and gene regulation."
Title: RNA Interference-Mediating Small RNA Molecules
Number: 20050234006
Filed: June 2, 2005
Lead Inventor: Thomas Tuschl, Max Planck
"Double-stranded RNA induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference," the patent application's abstract states. "Using a Drosophila in vitro system, we demonstrate that 19-23 [nucleotide long] 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.
Title: RNA Interference-Mediated Inhibition of Vascular Endothelial Growth Factor and Vascular Endothelial Growth Factor Receptor Gene Expression Using Short Interfering Nucleic Acid
Number: 20050233998
Filed: Sept. 16, 2004
Lead Inventor: Vasant Jadhav, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating VEGF and/or VEGFR 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 VEGF and/or VEGFR gene expression and/or activity by RNA interference using small nucleic acid molecules. 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 VEGF and/or VEGFR genes."
Title: RNA Interference-Mediated Inhibition of Matrix Metalloproteinase 13 Gene Expression Using Short Interfering Nucleic Acid
Number: 20050233997
Filed: Aug. 17, 2004
Lead Inventor: Ivan Richards, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating matrix metalloproteinase (e.g., MMP13) 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 MMP13 gene expression and/or activity by RNA interference using small nucleic acid molecules. 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 MMP13 genes."
Title: RNA Interference-Mediated Inhibition of Hairless Gene Expression Using Short Interfering Nucleic Acid
Number: 20050233996
Filed: April 26, 2004
Inventor: James McSwiggen, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating hairless 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 hairless gene expression and/or activity by RNA interference using small nucleic acid molecules. 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 hairless genes."
Title: Methods and Vectors for Expressing siRNA
Number: 20050233994
Filed: April 16, 2004
Lead Inventor: Ajamete Kaykas, University of Washington School of Medicine
"In a first aspect, the invention provides expression vectors comprising: (a) a first RNA polymerase III promoter operably associated with a first RNA polymerase III termination signal and (b) a second RNA polymerase III promoter operably associated with a second RNA polymerase III termination signal, wherein the first and second RNA polymerase III promoters are oriented to promote bidirectional transcription of an insert disposed between the first and the second RNA polymerase III termination signals," the patent application's abstract states. "In other aspects, the invention provides methods for using these expression vectors for inhibiting expression of target genes, for determining the effect of siRNAs on biological processes, and for identifying siRNAs that affect biological processes."
Title: RNA Interference-Mediated Inhibition of Platele-Derived Growth Factor and Platelet-Derived Growth Factor Receptor Gene Expression Using Short Interfering Nucleic Acid
Number: 20050233344
Filed: Aug. 20, 2004
Lead Inventor: James McSwiggen, Sirna Therapeutics
"This invention relates to compounds, compositions, and methods useful for modulating platelet derived growth factor and/or platelet derived growth factor receptor 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 platelet derived growth factor and/or platelet derived growth factor receptor gene expression and/or activity by RNA interference using small nucleic acid molecules. 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 platelet derived growth factor and/or platelet derived growth factor receptor genes."
Title: Methods of Preventing Off-Target Gene Silencing
Number: 20050233342
Filed: July 26, 2004
Lead Inventor: Muthiah Manoharan, Alnylam Pharmaceuticals
According to the patent application's abstract, "aspects featured in the invention relate to compositions and methods for inhibiting off-target gene silencing by iRNA agents."
The application specifically claims "a method of preventing off-target gene silencing in a cell comprising contacting a duplex RNA with the cell, wherein the duplex RNA comprises a modification on the sense strand, and (a) the sense strand of said duplex RNA has a region of at least 70 percent complementarity to at least 10 nucleotides of a preselected gene; or (b) the modified or unmodified sense strand has been tested for the ability to silence the off-target gene."