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8871918 Multiple exon skipping compositions for DMD  
Provided are antisense molecules capable of binding to a selected target site in the human dystrophin gene to induce exon skipping, and methods of use thereof to treat muscular dystrophy.
8871731 Micro-RNA for the regulation of cardiac apoptosis and contractile function  
The present invention relates to treating or preventing age-related cardiomyopathy by modulating the expression or activity of a miR-34 family member and/or PNUTS. Methods of treating or preventing...
8871729 Treatment of CNS conditions  
Methods and compositions for the treatment of pathologic conditions of the central nervous system (CNS) by means of intranasal administration of a composition that modulates, by means of RNA...
8871730 Chemical modification of short small hairpin RNAs for inhibition of gene expression  
Aspects of the present invention include the production and use of chemically modified RNAi agents (e.g., shRNAs) in gene silencing applications. The chemically modified RNAi agents disclosed...
8865671 RNAi-mediated inhibition of spleen tyrosine kinase-related inflammatory conditions  
RNA interference is provided for inhibition of spleen tyrosine kinase (Syk) mRNA expression, in particular, for treating patients having a Syk-related inflammatory condition or at risk of...
8865674 Antisense modulation of GCGR expression  
Provided herein are methods, compounds, and compositions for reducing expression of GCGR mRNA and protein in an animal. Such methods, compounds, and compositions are useful to treat, prevent,...
8865670 RNAi-related inhibition of TNFα signaling pathway for treatment of glaucoma  
RNA interference is provided for inhibition of tumor necrosis factor α (TNFα) by silencing TNFα cell surface receptor TNF receptor-1 (TNFR1) mRNA expression, or by silencing TNFα converting enz...
8865677 iRNA agents with biocleavable tethers  
The invention relates to iRNA agents, which preferably include a monomer in which the ribose moiety has been replaced by a moiety other than ribose that further includes a tether having one or more...
8865676 Methods and kits for synthesis of siRNA expression cassettes  
Amplification-based methods and kits for rapidly producing siRNA expression cassettes are provided. Also provided are methods for expressing amplified siRNA expression cassettes in cells.
8865884 Antisense modulation of kinesin-like 1 expression  
Antisense compounds, compositions and methods are provided for modulating the expression of kinesin-like 1. The compositions comprise antisense compounds, particularly antisense oligonucleotides,...
8865675 Compositions and methods for silencing apolipoprotein B  
The present invention provides compositions and methods for the delivery of interfering RNAs such as siRNAs that silence APOB expression in cells such as liver cells. In particular, the nucleic...
8865672 Prevention of tissue ischemia and related methods  
Provided herein are methods for preventing, ameliorating, and/or reducing tissue ischemia and/or tissue damage due to ischemia, increasing blood vessel diameter, blood flow and tissue perfusion in...
8859752 SIRNA-based therapy of Fibrodyplasia Ossificans Progressiva (FOP)  
This invention is directed to mutated Activin A type I receptor proteins (ACVR1) and isolated nucleic acids encoding same. The invention also relates to compositions and methods for siRNA-based...
8859514 Modulation of C-reactive protein expression  
Compounds, compositions and methods are provided for modulating the expression of C-reactive protein. The compositions comprise oligonucleotides, targeted to nucleic acid encoding C-reactive...
8859521 Oligomeric compounds and compositions for use in modulation of small non-coding RNAs  
Compounds, compositions and methods are provided for modulating the expression and function of small non-coding RNAs. The compositions comprise oligomeric compounds, targeted to small non-coding...
8859515 Treatment of tumor necrosis factor receptor 2 (TNFR2) related diseases by inhibition of natural antisense transcript to TNFR2  
The present invention relates to antisense oligonucleotides that modulate the expression of and/or function of Tumor Necrosis Factor Receptor 2 (TNFR2), in particular, by targeting natural...
8859749 Modified short interfering RNA  
The present invention refers to a double-stranded siRNA molecule comprising a sense Strand and an antisense Strand which is essentially complementary to the sense Strand, each of the sense and the...
8853179 Reengineering mRNA primary structure for enhanced protein production  
Described herein are rules to modify natural mRNAs or to engineer synthetic mRNAs to increase their translation efficiencies. These rules describe modifications to mRNA coding and 3′ UTR s...
8853180 Methods and materials for modulating deubiquitinases and ubiquitinated polypeptides  
This document relates to methods and materials involved in modulating deubiquitinases (e.g., USP10 polypeptides) and/or ubiquitinated polypeptides (e.g., tumor suppressor polypeptides or mutant...
8852940 Embryonic stem cell specific microRNAs promote induced pluripotency  
The methods of the present application describe that introduction of physiologically relevant miRNAs can enhance or modulate somatic cell reprogramming, generating induced pluripotent stem cells...
8853182 Cell growth inhibitor and screening method thereof  
An object is to provide a cell growth inhibitor also effective for androgen-independent prostate cancer. The present invention provides a cell growth inhibitor having, as an active ingredient, an...
8853178 Antisense modulation of PTP1B expression  
Compounds, compositions and methods are provided for modulating the expression of PTP1B. The compositions comprise antisense compounds, particularly antisense oligonucleotides, targeted to nucleic...
8853183 Prognosis and treatment of breast cancer  
The present invention relates to an antibody which specifically binds a Spot 14 (S14 or THRSP) protein in human breast cancer cells and a method for using the same to predict disease-free survival...
8846633 Method for inhibiting cancer stem cell like properties and chemoradioresistant properties of cancer or tumor cells with microRNA145  
The present invention provides a method for inhibiting cancer stem cell like properties and chemoradioresistant properties of cancer or tumor cells comprising delivering miR145 to the cancer or...
8846631 MicroRNA compositions and methods  
Provided herein are compositions comprising oligomeric compounds. In certain embodiments, the oligomeric compounds are useful as miRNA mimics. The oligomeric compounds may mimic the activity of...
8846081 Liposome compositions and methods of use thereof  
The present application relates to compositions comprising and methods of using a liposome comprising a pHLIP polypeptide, wherein a lipid bilayer of the liposome is substantially free of the pHLIP...
8846632 Nucleic acids for targeting multiple regions of the HCV genome  
Compositions and methods effective for modulating Hepatitis C viral infection are provided.
8846639 Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity  
In certain embodiments, the present invention provides oligomeric compounds having favorable toxicity profiles and therapeutic indexes. Compounds of the present invention comprise bicyclic...
8846894 RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)  
The present invention concerns methods and reagents useful in modulating gene expression in a variety of applications, including use in therapeutic, diagnostic, target validation, and genomic...
8846629 Id-1 and Id-2 genes and products as therapeutic targets for treatment of breast cancer and other types of carcinoma  
A method for treatment and amelioration of breast, cervical, ovarian, endometrial, squamous cells, prostate cancer and melanoma in a patient comprising targeting Id-1 or Id-2 gene expression with a...
8846023 Hepatitis C virus inhibitors  
The present disclosure relates to compounds, compositions and methods for the treatment of hepatitis C virus (HCV) infection. Also disclosed are pharmaceutical compositions containing such...
8846630 Pharmaceutical composition for treating cancer  
The present invention provides a pharmaceutical composition for treating cancer, comprising at least one selected from deoxyribonucleic acids (DNA) for encoding small interfering RNA (siRNA) which...
8841274 Protein arginine N-methyltransferase-5 method of cancer treatment  
A method for the treatment of a proliferative disease comprising providing a E2F-1 protein which is arginine-methylation defective or administering a substance which reduces the expression and/or...
8841432 Shuttle vectors for mycobacteria-escherichia coli and uses thereof  
The present disclosure provides a DNA molecule capable of replication in Mycobacteria having a nucleic acid sequence as disclosed in SEQ ID NO: 1, a shuttle vector constructed using it and a...
8841270 Use of inhibitors of Zdhhc2 activity for modulation of adipogenesis  
The present invention concerns Zdhhc2, a new target involved in adipogenesis modulation. Using a siRNA approach, the inventors demonstrated that decrease in Zdhhc2 activity in adipose tissue...
8841437 Precursor miRNA loop-modulated target regulation  
Modulation of mRNA activity is achieved with precursor miRNAs (ta-RNAs). ta-RNAs, primarily pre-miRNAs and pri-miRNAs, including truncated and mutated ta-RNAs, are employed for modulation of mRNA...
8841273 Methods and compositions for anti-EGFR treatment  
In some embodiments, the present invention provides methods for enhancing the sensitivity of cancer cells to anti-EGFR treatment, wherein the method comprises the introduction of a miR-200 miRNA to...
8841268 Method and kit for detection of cancer, and therapeutic agent for cancer  
The object aims to comprehensively analyze miRNA that undergoes epigenetic silencing in cancer to identify miRNA associated with cancer, elucidate the role of the identified miRNA in cancer, and...
8841272 Double-stranded RNA-based nanoparticles for insect gene silencing  
Nanoparticles for insect RNAi via oral delivery are provided, along with methods of silencing a target gene in a target insect using RNAi are provided. The nanoparticles comprise a polymer matrix...
8841267 Micro-RNA scaffolds, non-naturally occurring micro-RNAs, and methods for optimizing non-naturally occurring micro-RNAs  
The present disclosure provides non-naturally occurring miR-196a-2 miRNAs and non-naturally occurring miR-204 miRNAs. The non-naturally occurring miRNAs of the disclosure have mature strand...
8841269 Polynucleotides for use in treating and diagnosing cancers  
The present invention provides methods for increasing sensitivity of cancer cells to an antiestrogen agent, such as a selective estrogen receptor modulator (SERM). The methods include administering...
8835402 Compound and method for treating myotonic dystrophy  
Provided are 9-base morpholino antisense compounds targeted to polyCUG repeats in the 3′UTR region of dystrophia myotonica protein kinase (DMPK) mRNA, and related methods for treating myotonic d...
8828963 Diagnosis and treatment of chronic lymphocytic leukemia (CLL)  
New markers in the form of miRNA levels in plasma are provided for indicating the presence of CLL in a subject as well as suggesting routes of therapeutic treatment.
8828960 Amino acid vitamin ester compositions for controlled delivery of pharmaceutically active compounds  
The invention relates to pharmaceutical compositions that provide sustained-release of a pharmaceutically active compound and to methods of treating or preventing a condition in an animal by...
8828959 Antisense oligonucleotides capable of inhibiting the formation of capillary tubes by endothelial cells  
A pharmaceutical composition that blocks angiogenesis comprising as active agent at least one substance selected from the group consisting of (i) a nucleic acid molecule of a gene coding for...
8828958 Single-stranded antimicrobial oligonucleotides and uses thereof  
The current invention is directed to oligonucleotide sequences isolated from a sequence designated rbl-1 [SEQ ID NO. 19] that either kill or inhibit growth, or prevent the production of...
8828962 SiRNA compositions and methods for potently inhibiting viral infection  
No antiviral regimen has been consistently successful in treating H5N1 virus infection. We demonstrate that a group of highly effective siRNAs targeting different H5N1 viral genes shares a unique...
8828964 Cancer cell identification marker and cancer cell proliferation inhibitor  
Disclosed is an identification marker which can be utilized for detection of various human cancer cells and whose expression closely relates to malignant alteration of cells, and compositions for...
8828965 MiR-150 for the treatment of blood disorders  
The invention provides methods of treating certain blood related disorders, in particular, thrombocytopenia and anemia comprising increasing miR-150 expression or inhibiting miR-150 in progenitor...
8828961 Methods and compositions to protect aquatic invertebrates from disease  
Compositions and methods of protecting aquatic invertebrates from disease is shown. In one embodiment, dsRNA or antisense RNA to a nucleic acid molecule of the disease-causing microorganism is...