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US20160194636 MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE  
The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides.
US20160185725 c-Met Modulators and Method of Use  
The present invention provides compounds for modulating protein kinase enzymatic activity for modulating cellular activities such as proliferation, differentiation, programmed cell death,...
US20140294763 PEPTIDOMIMETIC PROTEASE INHIBITORS  
The present invention relates to peptidomimetic compounds useful as protease inhibitors, particularly as serine protease inhibitors and more particularly as hepatitis C NS3 protease inhibitors;...
US20130102034 MODIFIED NUCLEOSIDES, NUCLEOTIDES, AND NUCLEIC ACIDS, AND USES THEREOF  
The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using thereof.
US20130058977 COMPOSITIONS AND METHODS RELATED TO INDUCED TOLEROGENIC DENDRITIC CELLS EXTERNALLY LOADED WITH MHC CLASS I-RESTRICTED EPITOPES  
Disclosed are induced tolerogenic dendritic cells (itDCs) produced by combining itDCs with externally loadable MHC Class I-restricted epitopes, as well as related compositions and methods.
US20130058976 ALLERGEN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS FOR ALLERGY THERAPY  
Disclosed are allergen-specific induced tolerogenic dendritic cells (itDCs), as well as related compositions and methods.
US20130058902 DENDRITIC CELL SUBSETS FOR GENERATING INDUCED TOLEROGENIC DENDRITIC CELLS AND RELATED COMPOSITIONS AND METHODS  
Disclosed are induced tolerogenic dendritic cells (itDCs) produced from dendritic cell subsets that possess a desired physiological characteristic, as well as related compositions and methods.
US20130058894 THERAPEUTIC PROTEIN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS AND METHODS OF USE  
Disclosed are therapeutic protein-specific induced tolerogenic dendritic cells (itDCs), as well as related compositions and methods.
US20120064034 Peptidomimetic protease inhibitors  
The present invention relates to peptidomimetic compounds useful as protease inhibitors, particularly as serine protease inhibitors and more particularly as hepatitis C NS3 protease inhibitors;...
US20110318301 IL-17A/F HETEROLOGOUS POLYPEPTIDES AND THERAPEUTIC USES THEREOF  
The present invention is directed to a novel naturally occurring human cytokine that is comprised of a heterodimer of interleukin-17 and interleukin-17F designated herein as interleukin 17A/F...
US20100016241 METHODS AND COMPOSITIONS FOR INHIBITING C-MET DIMERIZATION AND ACTIVATION  
The invention provides methods and compositions for modulating the HGF/c-met signaling pathway, in particular by regulating c-met dimerization and/or binding of ligand to c-met using a c-met...
US20080008682 Modulators of toll-like receptor 7  
The present application provides for a compound of Formula I or II: or a pharmaceutically acceptable salt, solvate, and/or ester thereof, compositions containing such compounds, therapeutic...
US20170152474 SELF-ASSEMBLING MULTICELLULAR BODIES AND METHODS OF PRODUCING A THREE-DIMENSIONAL BIOLOGICAL STRUCTURE USING THE SAME  
Structures and methods for tissue engineering include a multicellular body including a plurality of living cells. A plurality of multicellular bodies can be arranged in a pattern and allowed to...
US20170088835 COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING  
Disclosed herein are compounds, compositions and methods for modulating splicing of SMN2 mRNA in a cell, tissue or animal. Also provided are uses of disclosed compounds and compositions in the...
US20160176966 ANTI CEACAM1 ANTIBODIES AND METHODS OF USING SAME  
A hybridoma cell which has been deposited under ATCC Accession Number PTA-9974 is disclosed. Also provided are Antibodies and methods of using same.
US20160176939 CANCER ANTIGEN HELPER PEPTIDE  
The present invention relates to a WT1 peptide which has an amino acid sequence consisting of contiguous amino acids derived from a WT1 protein and induces WT1-specific helper T cells by binding...
US20160136167 USE OF 3-(5-AMINO-2-METHYL-4-OXOQUINAZOLIN-3(4H)-YL)PIPERIDINE-2,6-DIONE IN TREATMENT OF IMMUNE-RELATED AND INFLAMMATORY DISEASES  
Provided herein are methods of using compounds and compositions for modulating lymphocytic activity, including activity of B cells and/or T cells, in immune-related diseases or inflammatory...
US20160096866 POTENT COMPSTATIN ANALOGS  
Compounds comprising peptides and peptidomimetics capable of binding C3 protein and inhibiting complement activation are disclosed. These compounds display greatly improved complement...
US20160076029 siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis  
The invention relates to a method of treating ocular amyloidosis by reducing TTR expression in a subject by administering a double-stranded ribonucleic acid (dsRNA) that targets a TTR gene to the...
US20160053264 SYNTHETIC MIMICS OF MIR-34  
Embodiments concern methods and compositions involving miR-34 mimics, including miR-34a and miR-34c mimics. In some embodiments, there are double-stranded RNA molecules with modified nucleotides...
US20160053019 MONOCLONAL ANTIBODY CAPABLE OF BINDING TO ANEXELEKTO, AND USE THEREOF  
The present inventors have succeeded in producing anti-AXL antibodies with specific functions. The present inventors also discovered that the antibodies have an angiogenesis-suppressive effect and...
US20160024527 Compositions and Methods for Enhancing Bioenergetic Status in Female Germ Cells  
Compositions and methods comprising bioenergetic agents for restoring the quality of aged oocytes, enhancing oogonial stem cells or improving derivatives thereof (e.g., cytoplasm or isolated...
US20150322434 INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS  
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by...
US20150315186 MODULATORS OF ATP-BINDING CASSETTE TRANSPORTERS  
The present invention relates to modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including Cystic Fibrosis Transmembrane Conductance Regulator, compositions thereof,...
US20150291562 IMIDE-BASED MODULATORS OF PROTEOLYSIS AND ASSOCIATED METHODS OF USE  
The description relates to imide-based compounds, including bifunctional compounds comprising the same, which find utility as modulators of targeted ubiquitination, especially inhibitors of a...
US20150252019 HETEROCYCLIC COMPOUNDS AND USES THEREOF  
The present invention provides compounds, pharmaceutically acceptable compositions thereof, and methods of using the same.
US20150232847 TARGETING OLIGONUCLEOTIDES AND RELATED METHODS FOR MODULATING FXN RNA  
Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally...
US20150232844 METHODS FOR MODULATING RNA USING 5' TARGETING OLIGONUCLEOTIDES  
Aspects of the invention relate to methods for increasing gene expression in a targeted manner. In some embodiments, methods and compositions are provided that are useful for posttranscriptionally...
US20150218559 MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE  
The invention relates to oligonucleotides for inducing skipping of exon 53 of the dystrophin gene. The invention also relates to methods of inducing exon 53 skipping using the oligonucleotides.
US20150203849 Oligonucleotide comprising an inosine for treating DMD  
The invention provides an oligonucleotide comprising an inosine, and/or a nucleotide containing a base able to form a wobble base pair or a functional equivalent thereof, wherein the...
US20150184157 MODULATION OF TRANSTHYRETIN EXPRESSION  
Compounds, compositions and methods are provided for modulating the expression of transthyretin. The compositions comprise oligonucleotides, targeted to nucleic acid encoding transthyretin....
US20150158952 CYSTEINE ENGINEERED ANTI-TENB2 ANTIBODIES AND ANTIBODY DRUG CONJUGATES  
Cysteine engineered anti-TENB2 antibodies are engineered by replacing one or more amino acids of a parent anti-TENB2 antibody with non cross-linked, reactive cysteine amino acids. Methods of...
US20150158908 SMAC Mimetic for Treating Myelodysplastic Syndromes  
A SMAC mimetic and pharmaceutical compositions thereof and methods of use.
US20150152413 Composition and Method for In Vivo and In Vitro Attenuation of Gene Expression Using Double Stranded RNA  
Introduction of double stranded RNA into cells, cell culture, organs and tissues, and whole organisms, particularly vertebrates, specifically attenuates gene expression.
US20150152185 METHOD OF INHIBITION OF LEUKEMIC STEM CELLS  
A method for inhibition of leukemic stem cells expressing IL-3Rα; (CD 123), comprises contacting the cells with an antigen binding molecule comprising a Fc region or a modified Fc region having...
US20150119441 Modulators of ATP-Binding Cassette Transporters  
Compounds of the present invention and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including...
US20150118291 Amphoteric Liposome Formulation  
The invention relates to compositions and methods to inhibit gene expression. In particular, the invention provides DNAi oligonucleotides sequestered by amphoteric liposomes for the treatment of...
US20150105440 ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION  
Antisense compounds, compositions and methods are provided for modulating the expression of apolipoprotein B. The compositions comprise antisense compounds, particularly antisense...
US20150094304 Modulators of ATP-Binding Cassette Transporters  
Compounds of the present invention and pharmaceutically acceptable compositions thereof, are useful as modulators of ATP-Binding Cassette (“ABC”) transporters or fragments thereof, including...
US20150079030 3'-[(2Z)-[1-(3,4-DIMETHYLPHENYL)-1,5-DIHYDRO-3-METHYL-5-OXO-4H-PYRAZOL-4-YLIDENE]HYDRAZINO]-2'-HYDROXY-[1,1'-BIPHENYL]-3-CARBOXYLIC ACID bis-(MONOETHANOLAMINE)  
An improved thrombopoietin mimetic, the bis-(monoethanolamine) salt of 3′-[(2Z)-[1-(3,4-dimethylphenyl)-1,5-dihydro-3-methyl-5-oxo- 4H-pyrazol-4-ylidene]hydrazino]-2′-hydroxy-[1,1′-biphenyl]-3 -car...
US20150056207 COMBINATION THERAPY WITH C-MET AND EGFR ANTAGONISTS  
The present invention relates generally to the fields of molecular biology and growth factor regulation. More specifically, the invention relates to combination therapies for the treatment of...
US20150056199 TGF-BETA RECEPTOR TYPE II VARIANTS AND USES THEREOF  
In certain aspects, the present disclosure relates to polypeptides comprising a truncated, ligand-binding portion of the extracellular domain of TβRII polypeptide useful to selectively antagonize...
US20150050699 RNA-DIRECTED DNA CLEAVAGE BY THE Cas9-crRNA COMPLEX  
Isolation or in vitro assembly of the Cas9-crRNA complex of the Streptococcus thermophilus CRISPR3/Cas system and use for cleavage of DNA bearing a nucleotide sequence complementary to the crRNA...
US20150047064 INHIBITING GENE EXPRESSION WITH dsRNA  
The present invention relates to the specific inhibition of gene expression in mammals by bringing the target gene into contact with double stranded RNA (dsRNA).
US20150017138 BACTERIAL MEDIATED THF ALPHA GENE SILENCING  
Methods are described for the delivery of one or more small interfering RNAs (siRNAs) to a eukaryotic cell using a bacterium. Methods are also described for using this bacterium to regulate gene...
US20150011608 iRNA Agents Targeting VEGF  
The features of the present invention relate to compounds, compositions and methods useful for modulating the expression of vascular endothelial growth factor (VEGF), such as by the mechanism of...
US20140350076 INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS  
The present invention provides a method for at least in part decreasing the production of an aberrant protein in a cell, the cell comprising pre-mRNA comprising exons coding for the protein, by...
US20140349292 OLIGONUCLEOTIDE N3'-->P5' THIOPHOSPHORAMIDATES: THEIR SYNTHESIS AND USE  
Oligonucleotides with a novel sugar-phosphate backbone containing at least one internucleoside 3′-NHP(O)(S−)O-5′ linkage, and methods of synthesizing and using the inventive oligonucleotides are...
US20140343130 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...
US20140343111 DIARYLTHIOHYDANTOIN COMPOUNDS  
The present invention relates to diarylthiohydantoin compounds and methods for synthesizing them and using them in the treatment of hormone refractory prostate cancer.