109226 ⎘
Applications; Uses; Special therapeutic applications based on a specific dosage / administration regimen
RNAi Agents for Inhibiting Expression of Activin Receptor-Like Kinase 7 (ALK7), Compositions Thereof, and Methods of Use
#2POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#3RNA AND DNA BASE EDITING VIA ENGINEERED ADAR RECRUITMENT
#4COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN (TTR) MEDIATED AMYLOIDOSIS
#5RNAi Agents for Inhibiting Expression of Thymic Stromal Lymphopoietin (TSLP), Compositions Thereof, and Methods of Use
#6RNAi Agents for Inhibiting Expression of Mitochondrial Amidoxime Reducing Component 1 (MARC1), Pharmaceutical Compositions Thereof, and Methods of Use
#7COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
#8METHODS OF USING EXTRACELLULAR VESICLE-ASO TARGETING STAT6
#9Compositions and Methods Using CPG Oligonucleotides
#10THROMBOMODULIN PROTEIN AND MIRNA FOR PREVENTING OR TREATING OSTEOATHRITIS
#11OLIGONUCLEOTIDES FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#12METHODS FOR REDUCING TAU EXPRESSION
#13HIGH-AFFINITY THROMBIN APTAMERS AND METHODS OF USE
#14MODULATORS OF DIACYGLYCEROL ACYLTRANSFERASE 2 (DGAT2)
#15BIODIFFUSION CHAMBER
#16Antisense oligonucleotides for treatment of USHER 2A. Exons 30-31
#17METHODS OF TREATING OR PREVENTING SPINAL MUSCULAR ATROPHY
#18NEURAL REPAIR THROUGH INHIBITION OF REG3A SIGNALING
#19Selective Reduction of Allelic Variants
#20RNAi Agents for Inhibiting Expression of Activin Receptor-Like Kinase 7 (ALK7), Compositions Thereof, and Methods of Use
#21OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF FOR EXON SKIPPING
#22IMMUNOTHERAPEUTIC CONSTRUCTS AND METHODS OF THEIR USE
#23METHODS OF TREATMENT OF SCN2A-RELATED DISORDERS
#24MODULATORS OF APOL1 EXPRESSION
#25Subcutaneous Delivery of RNAi Agents for Inhibiting Expression of Receptor for Advanced Glycation End-products
#26RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Compositions Thereof, and Methods of Use
#27COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
#28Compositions and Methods Using CPG Oligonucleotides
#29COMPOSITIONS AND METHODS FOR IMMUNOONCOLOGY
#30METHODS AND COMPOSITIONS FOR GENOME EDITING
#31MICRORNA-29 COMPOUNDS, COMPOSITIONS AND USES IN THERAPY
#32CANCER-SPECIFIC TRANS-SPLICING RIBOZYME AS GENE THERAPY PRODUCT COMBINED WITH RADIOTHERAPY
#33CRISPR/CAS9-BASED COMPOSITIONS AND METHODS FOR TREATING RETINAL DEGENERATIONS
#34MODULATORS OF ENaC EXPRESSION
#35MODULATION OF PREKALLIKREIN (PKK) EXPRESSION
#36METHODS OF TREATING OR PREVENTING SPINAL MUSCULAR ATROPHY
#37COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#38GLP-1 RECEPTOR LIGAND MOIETY CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#39MODULATING APOLIPOPROTEIN (A) EXPRESSION
#40ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#41METHODS OF TREATING MYOTONIC DYSTROPHY TYPE 1 USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES
#42DOSAGES AND METHODS FOR DELIVERING LIPID FORMULATED NUCLEIC ACID MOLECULES
#43COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#44METHODS AND COMPOSITIONS FOR REJUVENATING CNS GLIAL POPULATIONS BY SUPPRESION OF TRANSCRIPTION FACTORS
#45Composition Comprising Antisense Oligonucleotide and Use Thereof for Treatment of Duchenne Muscular Dystrophy
#46METHODS FOR THE TREATMENT OF NASH WITH ADVANCED FIBROSIS AND/OR CIRRHOSIS
#47ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
#48TARGETED INHIBITION USING ENGINEERED OLIGONUCLEOTIDES
#49POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#50METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
#51TGF-BETA THERAPEUTICS FOR AGE DISEASE
#52COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN (TTR) MEDIATED AMYLOIDOSIS
#53NEW THERAPY
#54LIPID NANOPARTICLES (LNPs)-BASED OCULAR DELIVERY
#55ANTI-FIBROTIC COMPOSITIONS COMPRISING FENDRR OR FRAGMENTS OR VARIANTS THEREOF AND METHODS OF PRODUCTION AND USE THEREOF
#56METHODS AND COMPOSITIONS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#57Methods and compositions for genome editing in non-dividing cells
#58METHODS AND COMPOSITIONS FOR TREATING PRIMARY HYPEROXALURIA
#59COMPOUNDS AND METHODS FOR MODULATING PMP22
#60OLIGONUCLEOTIDE TREATMENT OF HEPATITIS B PATIENTS
#61METHODS FOR THE REDUCTION OF Z-AAT PROTEIN LEVELS
#62Targeted inhibition using engineered oligonucleotides
#63Modulators of APOL1 expression
#64UNA OLIGOMERS FOR THE TREATMENT OF POLYGLUTAMINE DISEASES
#65MICRORNA-MEDIATED METHODS FOR REJUVENATING CNS GLIAL POPULATIONS
#66EXOSOME-DERIVED PIWI-INTERACTING RNA AND METHODS OF USE THEREOF
#67APOLIPOPROTEIN E (APOE) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#68METHODS AND COMPOSITIONS FOR REJUVENATING CNS GLIAL POPULATIONS BY SUPPRESION OF TRANSCRIPTION FACTORS
#69Targeted Inhibition Using Engineered Oligonucleotides
#70METHODS FOR REDUCING HTT EXPRESSION
#71Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
#72Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#73PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#74METHODS AND COMPOSITIONS FOR TREATING AN ANGIOTENSINOGEN- (AGT-) ASSOCIATED DISORDER
#75Selective reduction of allelic variants
#76COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#77COMPOUNDS AND COMPOSITIONS INCLUDING PHOSPHOROTHIOATED OLIGODEOXYNUCLEOTIDE, AND METHODS OF USE THEREOF
#78EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
#79OLIGONUCLEOTIDE-BASED THERAPY FOR ULCERATIVE COLITIS
#80Modified antisense oligonucleotide for inhibition of FoxP3 expression
#81METHODS OF TREATING OR PREVENTING SPINAL MUSCULAR ATROPHY
#82DIAGNOSTIC AND THERAPEUTIC USES OF COMPOSITIONS COMPRISING PURIFIED, ENRICHED POTENT EXOSOMES CONTAINING DISEASE-BASED AND THERAPY BASED SIGNATURE CARGO
#83Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
#84Targeted inhibition using engineered oligonucleotides
#85Therapeutic Targeting of a microRNA to Treat Duchenne Muscular Dystrophy
#86ORAL DELIVERY OF OLIGONUCLEOTIDES
#87Modulation of prekallikrein (PKK) expression
#88TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD
#89ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#90Methods for Oral Delivery of Oligonucleotides
#91RNA INTERFERENCE COMPOSITIONS AND METHODS FOR MALIGNANT TUMORS
#92COMPOSITIONS AND METHODS FOR TREATING AND PREVENTING AMYOTROPHIC LATERAL SCLEROSIS
#93POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#94Compounds and methods for reducing prion expression
#95METHODS AND COMPOSITIONS FOR TREATING GLYCOGEN STORAGE DISEASES
#96METHODS AND COMPOSITIONS FOR TREATING A SERPINC1-ASSOCIATED DISORDER
#97RNA AND DNA BASE EDITING VIA ENGINEERED ADAR RECRUITMENT
#98COMPOSITIONS AND METHODS FOR TREATMENT OF HEPATITIS D VIRUS INFECTION
#99METHODS AND COMPOSITIONS FOR TREATMENT OF FRONTOTEMPORAL DEMENTIA
#100Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
#101Compositions and methods for detection of SMN protein in a subject and treatment of a subject
#102Dosages and methods for delivering lipid formulated nucleic acid molecules
#103Oligomers
#104COMPOSITIONS AND METHODS FOR DELIVERY OF AAV
#105Compositions and methods for treating transthyretin (TTR) mediated amyloidosis
#106The Microbiome as a Target of MicroRNAs for the Treatment of Disease
#107Compositions and methods for inhibiting expression of the LECT2 gene
#108Antisense oligomers for treatment of conditions and diseases
#109RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
#110METHODS OF SUPPRESSING DELIVERY OF EXOSOMES TO LIVER AND SPLEEN
#111Combination therapy with liposomal antisense oligonucleotides
#112COMPOSITION COMPRISING ANTISENSE OLIGONUCLEOTIDE AND USE THEREOF FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#113TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD
#114Compositions and methods for treating and preventing muscular disorders and dystrophies, steroid side effects, and inflammation
#115Methods and formulations for the treatment of obesity and obesity-related metabolic diseases
#116MODULATORS OF ENaC EXPRESSION
#117RNA-AIDED IMMUNOTHERAPEUTICS
#118Methods for treating multiple sclerosis using antisense oligonucleotides
#119ANTI-FIBROTIC COMPOSITIONS COMPRISING FENDRR OR FRAGMENTS OR VARIANTS THEREOF AND METHODS OF PRODUCTION AND USE THEREOF
#120POLYNUCLEOTIDE AGENTS TARGETING SERPINC1 (AT3) AND METHODS OF USE THEREOF
#121Modulators of APOL1 expression
#122Exon skipping oligomer conjugates for muscular dystrophy
#123DNA-PK inhibitors
#124BIODIFFUSION CHAMBER
#125Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
#126Immunotherapeutic constructs and methods of their use
#127MODULATORS OF DNM2 EXPRESSION
#128EXON SKIPPING OLIGOMER CONJUGATES FOR MUSCULAR DYSTROPHY
#129Therapeutic targeting of a microRNA to treat Duchenne muscular dystrophy
#130Modulating apolipoprotein (a) expression
#131ASSAYS AND METHODS FOR DETERMINING EXPRESSION OF THE LECT2 GENE
#132Method of treating a localized fibrotic disorder using an IL-33 antagonist
#133Liposomal system for drug delivery
#134Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
#135CANCER TREATMENT
#136Therapy specific, pre-programmed auto injection device
#137GLP-1 RECEPTOR LIGAND MOIETY CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#138Therapy
#139Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#140Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
#141S-ANTIGEN TRANSPORT INHIBITING OLIGONUCLEOTIDE POLYMERS AND METHODS
#142POLYNUCLEOTIDE AGENTS TARGETING SERPINC1 (AT3) AND METHODS OF USE THEREOF
#143CRISPR/CAS9-BASED COMPOSITIONS AND METHODS FOR TREATING RETINAL DEGENERATIONS
#144Exon skipping oligomer conjugates for muscular dystrophy
#145Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#146Combination therapy with liposomal antisense oligonucleotides
#147METHODS FOR DOSING AND MONITORING SMAD7 ANTISENSE OLIGONUCLEOTIDE TREATMENT USING BIOMARKER LEVELS
#148COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#149Antisense oligomers for treatment of conditions and diseases
#150Antisense oligomers for treatment of conditions and diseases
#151COMPOSITIONS AND METHODS FOR PAIN AMELIORATION VIA MODIFICATION OF GENE EXPRESSION
#152Compositions and methods for treating endometriosis
#153Modulators of APOL1 expression
#154METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
#155METHODS AND COMPOSITIONS FOR TREATING A SUBJECT WITH A SMAD7 ANTISENSE OLIGONUCLEOTIDE
#156Consequences of a defective switch in cutaneous squamous cell carcinoma
#157DOSAGES AND METHODS FOR DELIVERING LIPID FORMULATED NUCLEIC ACID MOLECULES
#158Therapeutic targeting of a microRNA to treat Duchenne muscular dystrophy
#159COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#160Modulators of DNM2 expression
#161Modulation of prekallikrein (PKK) expression
#162Methods and compositions for genome editing in non-dividing cells
#163HIV CLINICAL PLAN
#164THERAPEUTICALLY MODULATING APOB AND APOAI
#165Therapy specific, pre-programmed auto injection device
#166Therapy specific, pre-programmed auto injection device
#167Modulating apolipoprotein (a) expression
#168METHODS OF USING SMAD7 ANTISENSE OLIGONUCLEOTIDES
#169Oligomers
#170DOWNREGULATING MIR-132 FOR THE TREATMENT OF LIPID RELATED DISORDERS
#171Therapeutic for treatment of diseases including the central nervous system
#172Methods of treating intestinal fibrosis using SMAD7 inhibition
#173Managing Intercellular Communications
#174Polynucleotide agents targeting programmed cell death 1 ligand 1 (PD-L1) and methods of use thereof
#175Compositions and methods for immunooncology
#176Cancer treatment
#177Argonaute protein-double stranded RNA complexes and uses related thereto
#178MODULATION OF MICRORNA 184 TO TREAT PATHOLOGICAL LYMPHANGIOGENESIS
#179Targeting microRNAs for metabolic disorders
#180Nanoconjugates able to cross the blood-brain barrier
#181Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
#182Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#183Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#184Method of treating a localized fibrotic disorder using an IL-33 antagonist
#185SUSTAINED RELEASE DRUG DELIVERY DEVICES
#186Pharmaceutical composition for preventing or treating diabetic complications and screening method for preventive or therapeutic agent for diabetic complications
#187TREATMENT OF HUTCHINSON-GILFORD PROGERIA SYNDROME AND DISEASES RELATED TO VASCULAR AGEING
#188Methods for treating spinal muscular atrophy
#189Compositions and methods for treating pancreatic cancer
#190Modulation of prekallikrein (PKK) expression
#191Downregulating miR-132 for the treatment of lipid related disorders
#192COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#193IMPAIRED WOUND HEALING COMPOSITION AND TREATMENTS
#194Methods and compositions for treating a subject with a SMAD7 antisense oligonucleotide
#195Polynucleotide agents targeting Serpinc1 (AT3) and methods of use thereof
#196METHODS FOR DOSING AND MONITORING SMAD7 ANTISENSE OLIGONUCLEOTIDE TREATMENT USING BIOMARKER LEVELS
#197COMPOSITIONS AND METHODS FOR TREATING CANCER
#198Compositions and methods for treating pancreatic cancer
#199METHODS FOR MALIGNANT TUMORS WITH RNAI MOLECULES TARGETED TO HSP47
#200APTAMERS FOR TOPICAL DELIVERY
#201Methods and compositions for treating a Serpinc1-associated disorder
#202Cancer treatment
#203MicroRNA induction of cardiac regeneration
#204SiRNAs and their use in methods and compositions for the treatment and/or prevention of eye conditions
#205COMPOSITIONS AND METHODS OF USING SAME FOR CONTROLLING PATHOGENICALLY INFECTED MOSQUITOES
#206Methods for treating cancer using nucleic acids targeting MDM2 or MYCN
#207Methods used to treat cancer
#208Modulation of prekallikrein (PKK) expression
#209Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#210Biocompatible flavonoid compounds for organelle and cell imaging
#211Wound healing compositions and treatments
#212Double-stranded RNA compounds to CASP2 and uses thereof
#213METHODS FOR TREATMENT OF WOUND HEALING UTILIZING CHEMICALLY MODIFIED OLIGONUCLEOTIDES
#214METHODS FOR TREATING OR PREVENTING OPHTHALMOLOGICAL DISEASES
#215RNA interference agents for GST-PI gene modulation
#216Targeting microRNAs for metabolic disorders
#217RNA agents for P21 gene modulation
#218RNA agents for GST-Pi gene modulation
#219Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
#220Low density lipoprotein receptor-mediated siRNA delivery
#221Compounds and methods for trans-membrane delivery of molecules
#222Dosages and methods for delivering lipid formulated nucleic acid molecules
#223Small Interfering RNA Delivery
#224Cancer treatment methods using remote conditioning
#225Oligomers
#226PCSK9 iRNA compositions and methods of use thereof
#227Method for expressing, secreting and transferring functional microRNAs/siRNAs and application thereof
#228Cancer treatment
#229Furin-knockdown bi-functional RNA
#230Plants as functional microRNA and/or functional siRNA carriers, preparation methods and uses thereof
#231Oligonucleotide—containing pharmacological compositions and their use
#232Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#233Targeting MicroRNAs for metabolic disorders
#234Use of miR-23a-3p and/or miR-27a-3p mimics as therapeutic agents for inhibition of neuronal apoptosis following brain injury
#235Nanoconjugates able to cross the blood-brain barrier
#236Double-stranded RNA compounds to CASP2 and uses thereof
#237Anti-clusterin monotherapy for cancer treatment
#238Custirsen treatment with reduced toxicity
#239DOSING REGIMENS FOR TREATING AND PREVENTING OCULAR DISORDERS USING C-RAF ANTISENSE
#240Low density lipoprotein receptor-mediated siRNA delivery
#241Impaired wound healing compositions and treatments
#242Lipid formulated compositions and methods for inhibiting expression of Eg5 and VEGF genes
#243Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#244Targeting MicroRNAs for metabolic disorders
#245SMALL INTERFERING RNA DELIVERY
#246Treatment of neurological disorders
#247Oligomers
#248Oligomers
#249METHODS FOR TREATING OR PREVENTING OPHTHALMOLOGICAL DISEASES
#250Oligomers
#251Optimized Methods For Delivery Of DSRNA Targeting The PCSK9 Gene
#252ANTI-THROMBIN APTAMER FORMULATIONS AND METHODS FOR USE
#253Low density lipoprotein receptor-mediated siRNA delivery
#254Furin-knockdown bi-functional RNA
#255METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#256Oligomers
#257Optimized Methods For Delivery Of DSRNA Targeting The PCSK9 Gene
#258STEADY-STATE SUBCUTANEOUS ADMINISTRATION OF APTAMERS
#259LOW DENSITY LIPOPROTEIN RECEPTOR-MEDIATED siRNA DELIVERY
#260Long term disease modification using immunostimulatory oligonucleotides
#261METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#262METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#263METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#264METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#265METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF GENE EXPRESSION BY DOUBLE-STRANDED RNA
#266Impaired wound healing compositions and treatments
#267Oligonucleotide-containing pharmacological compositions and their use
#268Oligonucleotide-containing pharmacological compositions and their use
#269Oligonucleotide-containing pharmacological compositions and their use
#270Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#271Method for improving immunity in shrimps