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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. |
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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,... |
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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;... |
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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. |
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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. |
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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. |
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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. |
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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. |
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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;... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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. |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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,... |
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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... |
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US20150252019 |
HETEROCYCLIC COMPOUNDS AND USES THEREOF
The present invention provides compounds, pharmaceutically acceptable compositions thereof, and methods of using the same. |
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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... |
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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... |
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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. |
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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... |
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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.... |
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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... |
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US20150158908 |
SMAC Mimetic for Treating Myelodysplastic Syndromes
A SMAC mimetic and pharmaceutical compositions thereof and methods of use. |
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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. |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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... |
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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). |
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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... |
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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... |
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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... |
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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... |
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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... |
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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. |