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9012425 Methods for the treatment of Leber congenital amaurosis  
The present invention relates to a method for treating a Leber congenital amaurosis in a patient harboring the mutation c.2991+1655 A>G in the CEP290 gene, comprising the step of administering to...
9012419 Methods and compositions related to eosinophil regulation  
Disclosed are compositions and methods for regulating eosinophils.
9012140 Target-dependent nucleic acid adapter  
In accordance with the present invention, there is provided a nucleic acid target-dependent adapter linked to a nucleic acid sequence. The adapter comprises linked together a biosensor having a...
9012624 Compositions and methods for inhibiting expression of a gene from the JC virus  
The invention relates to a double-stranded ribonucleic acid (dsRNA) for inhibiting the expression of a gene from the JC Virus (JC virus genome), comprising an antisense strand having a nucleotide...
9011903 Cationic lipids for therapeutic agent delivery formulations  
Here described are compounds of formula I: wherein R1 and R2 is independently selected from a group consisting of C10 to C18 alkyl, C12 to C18 alkenyl, and oleyl group; wherein R3 and R4 are...
9012225 Lipophilic polynucleotide conjugates  
Disclosed are lipophilic polynucleotide conjugates including polynucleotide-cholesterol conjugates and methods of delivering therapeutic polynucleotides to a mammalian cell or patient in need of...
9012426 Pharmaceutical anti-angiogenic composition including a MicroRNA-382 inhibitor  
The present invention relates to an anti-angiogenic composition, and more particularly, to a pharmaceutical anti-angiogenic composition including a microRNA-382 inhibitor. The inventors of the...
9012615 Methods for characterizing kidney function  
Embodiments of the invention relate generally to methods of characterizing kidney function. In particular, several embodiments quantify kidney-associated marker RNA isolated from vesicles contained...
9012421 Bicyclic cyclohexose nucleic acid analogs  
The present invention provides bicyclic cyclohexose nucleoside analogs and oligomeric compounds comprising these nucleoside analogs. These bicyclic nucleoside analogs are useful for enhancing...
9006198 Selective reduction of allelic variants  
Disclosed herein are antisense compounds and methods for selectively reducing expression of an allelic variant of a huntingtin gene containing a single nucleotide polymorphism (SNP). Such methods,...
9006206 Composition and methods for modulating cell proliferation and cell death  
Described herein are compositions and methods for modulation of p53-dependent cell death and cell proliferation. The compositions are microRNAs and associated nucleic acids.
9006195 Regulation of hematopoietic stem cell functions through microRNAs  
The present disclosure relates to regulation of functions of hematopoietic stem cells (HSCs) by delivering of miRNAs, including miR-125b, miR-126, and miR-155, to HSCs. For example, in some...
9006204 Aptamers for prion diagnostics and aptamer binding detection system  
There is disclosed PrPSc aptamers. There is further disclosed PrPSc aptamers. There is further disclosed an infectious agent or neurodegenerative disease bifunctional aptamer comprising a first...
9006201 Prophylactic or therapeutic agent for diabetes  
The present invention provides a prophylactic or therapeutic drug for diabetes, which contains a polynucleotide such as miR-199b* and the like. Moreover, the present invention provides a method for...
9006197 Compositions and methods for inhibiting expression of Eg5 and VEGF genes  
This invention relates to compositions containing double-stranded ribonucleic acid (dsRNA) in a SNALP formulation, and methods of using the compositions to inhibit the expression of the Eg5 and...
9006196 Oligoribonucleotides and methods of use thereof for treatment of alopecia, acute renal failure and other diseases  
The invention relates to a double-stranded compound, preferably an oligoribonucleotide, which down-regulates the expression of a human p53 gene. The invention also relates to a pharmaceutical...
9006205 Antisense oligonucleotides against neutral sphingomyelinase and neutral sphingomyelinase inhibitor GW4869 for degenerative neurological disorders  
Alzheimer's disease (AD) is the most common human neurodegenerative disease of the CNS resulting in progressive neuronal death and memory loss. Despite intense investigations, no effective therapy...
9006200 MicroRNA-based approach to treating malignant pleural mesothelioma  
The invention relates to microRNA mimics, corresponding to the miR-15/107 family, and to methodology for using microRNA mimics to treat malignant pleural mesothelioma (MPM) by restoring regulation...
9005906 Tetrahydropyran nucleic acid analogs  
The present disclosure describes tetrahydropyran nucleoside analogs, oligomeric compounds prepared therefrom and methods of using the oligomeric compounds. More particularly, tetrahydropyran...
9006194 Compositions and methods for diminishing viral infection and inflammation associated with viral infection  
The invention relates to compositions and methods for preventing and diminishing virus infection. The invention further relates to compositions and methods for diminishing inflammation associated...
8999943 Antigene oligomers inhibit transcription  
Transcription of a gene in a mammalian cell is methylase-independently inhibited by contacting the cell with a nucleic acid oligomer of 12-28 bases complementary for a partially single-stranded...
8999950 Compositions and methods for silencing aldehyde dehydrogenase  
The present invention provides compositions comprising therapeutic nucleic acids such as interfering RNA (e.g., dsRNA such as siRNA) that target aldehyde dehydrogenase (ALDH) gene expression, lipid...
8999945 Methods, compositions and systems for local delivery of drugs  
Implantable medical device eluting drug locally and in a prolonged fashion are provided.
8999944 Thioredoxin interacting protein (TXNIP) as regulator of vascular function  
The present invention relates to screening assays for the identification of agents that can modify the interaction of thioredoxin interacting protein (TXNEP) on thioredoxin (TRX)5 preferably by...
8993528 Oligomeric compounds comprising tricyclic nucelosides and methods for their use  
The present disclosure provides tricyclic nucleosides, oligomeric compounds comprising at least one of the tricyclic nucleosides and methods of using the oligomeric compounds. The methods provided...
8993529 Antisense modulation of superoxide dismutase 1, soluble expression  
Antisense compounds, compositions and methods are provided for modulating the expression of superoxide dismutase 1, soluble. The compositions comprise antisense compounds, particularly antisense...
8993530 RNAi expression constructs  
The present invention provides compositions and methods suitable for expressing 1-x RNAi agents against a gene or genes in cells, tissues or organs of interest in vitro and in vivo so as to treat...
8987224 MicroRNA-198 as a tumor suppressor in pancreatic cancer  
A novel network of tumorigenic prognostic factors is identified that plays a critical role in advanced pancreatic cancer (PC) pathogenesis. This interactome is interconnected through a central...
8987226 Modified single-stranded polynucleotides  
It is intended to provide a polynucleotide that is resistant to RNase and has an RNA interference effect, etc. The present invention provides a single-stranded polynucleotide that is derived from a...
8987223 Treatment of pulmonary and pleural fibrosis using HSP27 inhibitors  
Reduction of HSP27 expression is in beneficial in the treatment of pleural and pulmonary fibrosis and in particular subpleural fibrosis and IPF. Pharmaceutical compositions for this purpose contain...
8987215 Composition for use in gene therapy  
A composition useful in gene therapy and a method of treatment to effect gene therapy is provided. In particular, a pharmaceutical composition comprising a nonimmunogenic net polyanionic...
8987227 Hepatitis C dsRNA effector molecules, expression constructs, compositions, and methods of use  
The present invention provides agents, compositions, constructs and methods for silencing HCV polynucleotides, as well as methods and compositions for treating or preventing HCV infection in a...
8980858 Treatment of methionine sulfoxide reductase a (MSRA) related diseases by inhibition of natural antisense transcript to MSRA  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Methionine Sulfoxide Reductase A (MSRA), in particular, by targeting natural antisense...
8980860 Treatment of discs large homolog (DLG) related diseases by inhibition of natural antisense transcript to DLG  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Discs large homolog (DLG), in particular, by targeting natural antisense...
8980856 Treatment of colony-stimulating factor 3 (CSF3) related diseases by inhibition of natural antisense transcript to CSF3  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Colony-stimulating factor 3 (CSF3), in particular, by targeting natural antisense...
8980855 Minor groove binder (MGB)-oligonucleotide miRNA antagonists  
Compositions and methods for inhibiting the actions of non-coding RNAs such as miRNAs and piRNAs are provided. The compositions comprise single or double stranded oligonucleotides conjugated with...
8980853 Compositions and methods for modulation of SMN2 splicing in a subject  
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a subject. Also provided are uses of disclosed compounds and compositions in the manufacture of a...
8980857 Treatment of PAR4 related diseases by inhibition of natural antisense transcript to PAR4  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of PAR4, in particular, by targeting natural antisense polynucleotides of PAR4. The...
8980861 Method for determining activators of excitatory synapse formation  
The invention provides methods of screening a compound that can increase spine/excitatory synapse formation and/or numbers. The compound is identified by contacting Ephexin5 with a test compound...
8975079 Reducible polymers for nonviral gene delivery  
Provided herein are biodegradable copolymers and nanoplex delivery systems comprising the same and a cargo molecule, such as a nucleic acid, a polynucleotide or other biomolecule. The biodegradable...
8969317 MicroRNA compounds and methods for modulating miR-21 activity  
Described herein are compositions and methods for the inhibition of miR-21 activity. The compositions have certain nucleoside modification patterns that yield potent inhibitors of miR-21 activity....
8969316 Compounds and methods for modulating expression of DGAT2  
The present disclosure describes short antisense compounds, including such compounds comprising chemically-modified high-affinity monomers 8-16 monomers in length. Certain such short antisense...
8969318 Bispecific aptamers mediating tumour cell lysis  
Disclosed are bispecific aptamers binding with high specifity to a tumour specific antigen (TSA) and a effector cell specific antigen (ESA) for treatment of cancer.
8969314 Methods for use in modulating miR-122a  
Methods are provided for the treatment of cardiovascular or metabolic diseases characterized by elevated serum total cholesterol, elevated serum LDL-cholesterol, or elevated serum triglycerides,...
8962585 Treatment of tumor protein 63 (p63) related diseases by inhibition of natural antisense transcript to p63  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Tumor Protein 63 (p63), in particular, by targeting natural antisense polynucleotides...
8962592 Methods of treating a meiotic kinesin associated disease  
The invention provides methods of treating a meiotic kinase-associated disease, preferably the meiotic kinase HSET, by administering an inhibitor of the meiotic kinase. Preferably, the disease is...
8962584 Compositions for controlling Varroa mites in bees  
An isolated nucleic acid agent is disclosed comprising a nucleic acid sequence which downregulates expression of a gene product of a Varroa destructor mite. Compositions comprising same and uses...
8962588 Micro-RNAS that control myosin expression and myofiber identity  
The present invention relates to the identification of two microRNAs, miR-499 and miR-208b, that repress fast skeletal muscle contractile protein genes. Expression of miR-499 and/or miR-208b can be...
8962586 Treatment of pyrroline-5-carboxylate reductase 1 (PYCR1) related diseases by inhibition of natural antisense transcript to PYCR1  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Pyrroline-5-carboxylate reductase 1 (PYCR1), in particular, by targeting natural...
8962583 Treatment of inflammatory diseases using miR-124  
Methods of treating, reducing the risk of developing, or delaying the onset of an inflammatory disease are disclosed. The methods involved providing a subject with or at risk of developing an...