109191 ⎘
Structure or type of the nucleic acid; Chemical structure; Spatial arrangement of the modifications having a combination of backbone and sugar modifications
Compositions for Modulating C9ORF72 Expression
#2COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#3Modulation of Apolipoprotein C-III (ApoCIII) Expression in Lipoprotein Lipase Deficient (LPLD) Populations
#4Compounds and Methods for Reducing ATXN3 Expression
#5POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#6MODIFIED DOUBLE-STRANDED RNA AGENTS
#7TREATMENT OF THYMIC STROMAL LYMPHOPOIETIN (TSLP) RELATED DISEASES BY INHIBITION OF LONG-FORM TSLP TRANSCRIPTS
#8Cyclic Antisense Therapeutics
#9COMPOSITIONS AND METHODS FOR TREATING MENINGIOMA
#10METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#11RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
#12Compositions and Methods for Correcting Limb Girdle Muscular Dystrophy Type 2C Using Exon Skipping
#13COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#14EXTRACELLULAR VESICLE-ASO CONSTRUCTS TARGETING CEBP/BETA
#15Compositions and Methods for Modulating Complement Factor B Expression
#16Modified Guide RNAs
#17SIRNA THERAPY FOR TRANSTHYRETIN (TTR) RELATED OCULAR AMYLOIDOSIS
#18OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#19NUCLEIC ACID COMPOUNDS
#20COMPOSITIONS AND METHODS FOR TREATING DISEASES ASSOCIATED WITH PATHOGENIC FUS VARIANTS
#21Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#22NANOPARTICLE FORMULATIONS FOR DELIVERY OF NUCLEIC ACID COMPLEXES
#23CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
#24Antisense Oligonucleotide for Targeting Progranulin
#25BBB-CROSSING LIPID LIGAND OF HETERO NUCLEIC ACID
#26OLIGONUCLEOTIDES FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#27NUCLEIC ACID MOLECULE FOR REDUCTION OF PAPD5 AND PAPD7 mRNA FOR TREATING HEPATITIS B INFECTION
#28Compounds and Methods for Reducing LRRK2 Expression
#29MODULATORS OF DIACYGLYCEROL ACYLTRANSFERASE 2 (DGAT2)
#30FN3 DOMAIN-siRNA CONJUGATES AND USES THEREOF
#31Selective Antisense Compounds and Uses Thereof
#32KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#33COMPOSITION AND METHOD FOR INHIBITING EXPRESSION OF PROTEIN LPA(APO(A))
#34REVERSIR TM COMPOUNDS
#35PRIMARY CELL GENE EDITING
#36COMPOSITIONS AND METHODS FOR INHIBITING STAG1 EXPRESSION AND USES THEREOF
#37Selective Reduction of Allelic Variants
#38MODIFIED RNAi AGENTS
#39METHODS OF DESIGNING CONDITIONAL-ACTIVATABLE SMALL INTERFERING RNA SENSORS
#40FULLY STABILIZED ASYMMETRIC SIRNA
#41HIGH MOBILITY GROUP BOX-1 (HMGB1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#42TARGETED COMPOSITIONS
#43THERAPEUTIC COMPOSITIONS FOR TREATING PAIN VIA MULTIPLE TARGETS
#44MODULATION OF ALPHA SYNUCLEIN EXPRESSION
#45OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#46COMPOUNDS AND METHODS FOR THE MODULATION OF PROTEINS
#47METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#48TARGETED COMPOSITIONS
#49PIKFYVE ANTISENSE OLIGONUCLEOTIDES
#50GUIDE RNA WITH CHEMICAL MODIFICATIONS
#51COMPOSITIONS AND METHODS FOR MODULATING NEURONAL EXCITABILITY AND MOTOR BEHAVIOR
#52ANGELMAN SYNDROME ANTISENSE TREATMENT
#53MODULATORS OF APOL1 EXPRESSION
#54OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#55COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#56ANTISENSE OLIGONUCLEOTIDES (ASO) FOR EFFICIENT AND PRECISE RNA EDITING WITH ENDOGENOUS ADENOSINE DEAMINASE ACTING ON RNA (ADAR)
#57Modified Guide RNAs
#58Antisense Oligonucleotide for Targeting Progranulin
#59RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Compositions Thereof, and Methods of Use
#60MODIFIED DOUBLE-STRANDED RNA AGENTS
#61STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#62METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
#63RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF NEUROMUSCULAR DISORDERS
#64MODIFIED DOUBLE-STRANDED RNA AGENTS
#65Compounds and Methods for Reducing ATXN2 Expression
#66COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#67Chemically Modified Guide RNAs for CRISPR/CAS-Mediated Gene Correction
#68COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#69GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#70Compounds and methods for reducing LRRK2 expression
#71METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
#72LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
#73Compositions and methods for modulating apolipoprotein (a) expression
#74MODULATORS OF ENaC EXPRESSION
#75CRISPR-BASED COMPOSITIONS AND METHODS OF USE
#76COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION
#77COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#78REDUCING INTRON RETENTION
#79COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
#80COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#81COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#82COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#83ANTISENSE OLIGONUCLEOTIDES TARGETING SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHIES
#84RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (SINA)
#85Compounds and Methods for Modulating Splicing of Pre-MRNA
#86RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
#87METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#88METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
#89RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#90CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
#91TARGETED NUCLEIC ACID CONJUGATE COMPOSITIONS
#92COMPOUNDS AND METHODS FOR REDUCING KCNT1 EXPRESSION
#93TUNING CRISPR/CAS9 ACTIVITY WITH CHEMICALLY MODIFIED NUCLEOTIDE SUBSTITUTIONS
#94PHARMACEUTICAL COMPOSITION CONTAINING CELL-DERIVED NATURAL OR ARTIFICIAL NANOVESICLES LOADED WITH ANTISENSE OLIGONUCLEOTIDE-BASED DRUG
#95ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#96Compositions for Modulating C9ORF72 Expression
#97DEFINED MULTI-CONJUGATES OLIGONUCLEOTIDES
#98DOUBLE-STRANDED SIRNA HAVING PATTERNED CHEMICAL MODIFICATIONS
#99KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#100OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#101ALPHA-1 ANTITRYPSIN (AAT) RNAI AGENTS, COMPOSITIONS INCLUDING AAT RNAI AGENTS, AND METHODS OF USE
#102METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#103OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#104POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#105METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
#106COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#107OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#108CONJUGATED OLIGONUCLEOTIDES
#109COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#110COMPOSITIONS AND METHODS FOR TREATMENT OF CARDIAC DISEASES
#111ANTISENSE COMPOUNDS
#112CHEMICALLY MODIFIED CRISPR-CAS13 GUIDE RNAS
#113Oligomers and oligomer conjugates
#114Stabilized saRNA Compositions and Methods of Use
#115MODIFIED OLIGONUCLEOTIDES FOR USE IN TREATMENT OF TAUOPATHIES
#116OLIGONUCLEOTIDES CONTAINING 2'-DEOXY-2'FLUORO-BETA-D-ARABINOSE NUCLEIC ACID (2'-FANA) FOR TREATMENT AND DIAGNOSIS OF RETROVIRAL DISEASES
#117GUIDE RNA WITH CHEMICAL MODIFICATIONS
#118COMPOUNDS AND METHODS FOR MODULATING PMP22
#119COMBINATION
#120COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
#121COMPOUNDS AND METHODS FOR REDUCING APOE EXPRESSION
#122Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
#123SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
#124SIRNA THERAPY FOR TRANSTHYRETIN (TTR) RELATED OCULAR AMYLOIDOSIS
#125Angelman syndrome antisense treatment
#126OLIGONUCLEOTIDES FOR REDUCTION OF PD-L1 EXPRESSION
#127OLIGOMERS HAVING BICYCLIC SCAFFOLD MOIETIES
#128COMPLEMENT COMPONENT C1R INHIBITORS FOR TREATING A NEUROLOGICAL DISEASE, AND RELATED COMPOSITIONS, SYSTEMS AND METHODS OF USING SAME
#129Compositions for modulating tau expression
#130RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
#131ANTISENSE OLIGONUCLEOTIDES FOR RNA EDITING
#132Oligonucleotides for inducing paternal UBE3A expression
#133MICRORNAS AND METHODS OF THEIR USE
#134G PROTEIN-COUPLED RECEPTOR 146 (GPR146) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#135Modified guide RNAs
#136siRNAs WITH VINYLPHOSPHONATE AT THE 5' END OF THE ANTISENSE STRAND
#137THERAPEUTICS FOR SYNGAP HAPLOINSUFFICIENCY
#138Modulators of APOL1 expression
#139MODULATION OF FACTOR 11 EXPRESSION
#140Nucleic acid oligomers and uses therefor
#141PCSK9 TARGETING OLIGONUCLEOTIDES FOR TREATING HYPERCHOLESTEROLEMIA AND RELATED CONDITIONS
#142GENOME EDITING OF HUMAN NEURAL STEM CELLS USING NUCLEASES
#143COMPOUNDS AND METHODS FOR REDUCING MSH3 EXPRESSION
#144Compounds and Methods for Reducing ATXN3 Expression
#145METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#146INHIBITION OF POLYOMAVIRUS REPLICATION
#147METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#148Modified double-stranded RNA agents
#149Compositions and Methods for Modulation of SMN2 Splicing in a Subject
#150Reducing intron retention
#151Compositions and Methods for Genome Editing
#152Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
#153COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#154Compositions for FNIP1/FNIP2 Gene Modulation and Methods Thereof
#155COMPOUND COMPRISING A NUCLEIC ACID AND A HALF-LIFE EXTENSION MOTIF
#156COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
#157ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#158DOUBLE-STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE COMPRISING DOUBLE-STRANDED OLIGONUCLEOTIDE, PREPARATION METHOD THEREOF AND USE THEREOF
#159Fc FRAGMENT OF IgG RECEPTOR AND TRANSPORTER (FCGRT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#160RNAi Agents for Inhibiting Expression of Xanthine Dehydrogenase (XDH), Pharmaceutical Compositions Thereof, and Methods of Use
#161Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#162COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#163COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION
#164USE OF SCAMP3 INHIBITORS FOR TREATING HEPATITIS B VIRUS INFECTION
#165COMPOUNDS AND METHODS FOR MODULATING SPLICING OF PRE-MRNA
#166Compounds and methods for reducing LRRK2 expression
#167Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
#168Angleman syndrome antisense treatment
#169MODIFIED DOUBLE-STRANDED RNA AGENTS
#170Targeted compositions
#171PRODRUG INCORPORATED sgRNA SYNTHESIS
#172EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#173RNAS FOR COMPLEMENT INHIBITION
#174STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#175CHEMICALLY MODIFIED OLIGONUCLEOTIDES WITH IMPROVED SYSTEMIC DELIVERY
#176ANTISENSE OLIGOMERS AND METHODS FOR TREATING PARKIN-RELATED PATHOLOGIES
#177MODIFIED IMMUNE CELLS HAVING ADENOSINE DEAMINASE BASE EDITORS FOR MODIFYING A NUCLEOBASE IN A TARGET SEQUENCE
#178Optimized anti-FLT1 oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
#179RNAi CONSTRUCTS AND METHODS FOR INHIBITING LPA EXPRESSION
#180TREATMENT OF THYMIC STROMAL LYMPHOPOIETIN (TSLP) RELATED DISEASES BY INHIBITION OF LONG-FORM TSLP TRANSCRIPTS
#181Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
#182STABLE TARGET-EDITING GUIDE RNA HAVING CHEMICALLY MODIFIED NUCLEIC ACID INTRODUCED THEREINTO
#183Compositions and methods for modulating complement factor B expression
#184Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#185METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXAPNSION DISORDERS ASSOCIATED WITH OGG1 ACTIVITY
#186RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#187Modulation of huntingtin expression
#188IDENTIFYING NON-PRODUCTIVE SPLICE SITES
#189COMPOUNDS AND METHODS FOR MODULATING C90RF72
#190RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
#191RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
#192Compounds and methods for modulating factor XII
#193Selective antisense compounds and uses thereof
#194OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#195PIKFYVE antisense oligonucleotides
#196COMPOSITIONS AND METHODS FOR DECREASING TAU EXPRESSION
#197Modulation of Angiopoietin-Like 3 Expression
#198Selective reduction of allelic variants
#199Modified double-stranded RNA agents
#200SHORT INTERFERING NUCLEIC ACID (siNA) MOLECULES AND USES THEREOF FOR CORONAVIRUS DISEASES
#201PPM1A INHIBITORS AND METHODS OF USING SAME
#202FN3 domain-siRNA conjugates and uses thereof
#203OLIGONUCLEOTIDE COMPOUNDS FOR TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS
#204PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#205CHEMICALLY MODIFIED OLIGONUCLEOTIDES FOR RNA EDITING
#206Long Noncoding RNA Implicated in Cardiovascular Disease and Use Thereof
#207OLIGONUCLEOTIDES AND METHODS OF USE FOR TREATING NEUROLOGICAL DISEASES
#208Modified RNAi agents
#209COMPOSITIONS AND METHODS FOR MODULATING ANGIOPOIETIN-LIKE 3 EXPRESSION
#210DOUBLE-STRANDED NUCLEIC ACID COMPLEX AND USE THEREOF
#211COMPOSITIONS AND METHODS FOR CLL1 MODIFICATION
#212Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#213Defined multi-conjugates oligonucleotides
#214COMPOSITIONS AND METHODS FOR MODULATING SPLICING AND PROTEIN EXPRESSION
#215COMPOSITIONS AND METHODS FOR DETECTING AND TREATING INSULIN RESISTANCE
#216COMPOUNDS AND METHODS USEFUL FOR MODULATING GENE SPLICING
#217RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (SINA)
#218COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
#219METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
#220UBE3A ANTISENSE THERAPEUTICS
#221EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#222REVERSIR™ compounds
#223MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
#224COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#225OLIGONUCLEOTIDE COMPOUNDS FOR TARGETING HUNTINGTIN MRNA
#226Methods and compositions for inhibiting PMP22 expression
#227COMPOSITIONS AND METHODS FOR MODULATING TAU EXPRESSION
#228Antisense oligonucleotides for the treatment of Leber congenital amaurosis
#229PD-L1 antisense oligonucleotides for use in tumor treatment
#230COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#231METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#232MODULATION OF TRANSTHYRETIN EXPRESSION
#233MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS
#234ORAL DELIVERY OF OLIGONUCLEOTIDES
#235Antisense oligonucleotide for targeting progranulin
#236Antisense oligonucleotides for the treatment of Stargardt disease
#237TAU-TARGETING OLIGONUCLEOTIDE GAPMERS
#238RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#239COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#240Compositions for modulating Ataxin 2 expression
#241OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#242Compositions comprising chemically modified guide RNAs for CRISPR/Cas-mediated editing of HBB
#243CHEMICALLY MODIFIED GUIDE RNAS FOR CRISPR/CAS-MEDIATED GENE CORRECTION
#244Chemically modified guide RNAS for CRISPR/Cas-mediated gene regulation
#245AIM2 INHIBITORS AND USES THEREOF
#246Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
#247OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#248Oligonucleotides for inducing paternal UBE3A expression
#249TARGETS AND METHODS FOR TREATING EPSTEIN-BARR VIRUS MEDIATED NEURODEGENERATION
#250OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#251TAU ANTISENSE OLIGOMERS AND USES THEREOF
#252TREATMENT OF CNS INJURY WITH RNAi THERAPEUTICS
#253Functionally-Modified Oligonucleotides And Subunits Thereof
#254OLIGONUCLEOTIDES FOR USE IN THE TREATMENT OF DYSTROPHIC EPIDERMOLYSIS BULLOSA
#255ANTISENSE OLIGONUCLEOTIDES FOR NUCLEIC ACID EDITING
#256METHODS AND MATERIALS FOR ACTIVATING AN INTERNAL RIBOSOME ENTRY SITE IN EXON 5 OF THE DMD GENE
#257REVERSIR TM COMPOUNDS
#258ANTISENSE OLIGOMERS FOR CONTROLLING CANDIDA ALBICANS INFECTIONS
#259Targeted control of pests and pathogens by plant delivery of 2′F-ANA-oligonucleotides
#260COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#261Polycomb-associated Non-Coding RNAs
#262ANGIOPOIETIN-LIKE 3 (ANGPTL3) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#263Therapeutic compositions for treating pain via multiple targets
#264COMPOSITIONS COMPRISING SYNTHETIC POLYNUCLEOTIDES ENCODING CRISPR RELATED PROTEINS AND SYNTHETIC SGRNAs AND METHODS OF USE
#265Antiviral agents
#266ANTISENSE-INDUCED EXON EXCLUSION IN MYOSTATIN
#267RNAi Agents for Inhibiting Expression of Alpha-ENaC And Methods of Use
#268Angelman syndrome antisense treatment
#269OLIGONUCLEOTIDES FOR HTT-1A MODULATION
#270ANTISENSE OLIGOMERS FOR TREATMENT OF AUTOSOMAL DOMINANT MENTAL RETARDATION-5 AND DRAVET SYNDROME
#271ARTIFICIAL NUCLEIC ACIDS FOR RNA EDITING
#272METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#273MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION
#274Compounds and methods for reducing ATXN2 expression
#275Compounds and methods for reducing ATXN3 expression
#276COMPOSITIONS AND METHODS FOR THE TREATMENT OF KCNT1 RELATED DISORDERS
#277Modified double-stranded RNA agents
#278RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (SINA)
#279CHEMICALLY-MODIFIED RNAi CONSTRUCTS AND USES THEREOF
#280Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method thereof and use thereof
#281SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
#282ANTISENSE OLIGONUCLEOTIDES FOR MODULATING HTRA1 EXPRESSION
#283Compositions and their uses directed to huntingtin
#284Treatment of thymic stromal lymphopoietin (TSLP) related diseases by inhibition of long-form TSLP transcripts
#285POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#286Compositions and methods for treatment of cardiac diseases
#287METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MLH1 ACTIVITY
#288METHODS AND COMPOSITIONS FOR TREATING A SERPINC1-ASSOCIATED DISORDER
#289POLYNUCLEOTIDE AGENTS TARGETING AMINOLEVULINIC ACID SYNTHASE-1 (ALAS1) AND USES THEREOF
#290MODIFIED DOUBLE-STRANDED RNA AGENTS
#291POLYNUCLEOTIDE AGENTS TARGETING HYDROXYACID OXIDASE (GLYCOLATE OXIDASE, HAO1) AND METHODS OF USE THEREOF
#292PRIMARY CELL GENE EDITING
#293Oligonucleotides for the treatment of nucleotide repeat expansion disorders associated with MSH3 activity
#294COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#295Methods and Compositions for the ADAR-Mediated Editing of SERPINA1
#296Soluble TNF Receptors and Their Use in Treatment of Disease
#297OLIGONUCLEOTIDES FOR MSH3 MODULATION
#298Substances for targeting various selected organs or tissues
#299Allele specific modulators of P23H rhodopsin
#300Methods for reducing LRRK2 expression