109162 ⎘
Structure or type of the nucleic acid; Chemical structure of the backbone Phosphorothioates
siRNA SUPPRESSING EXPRESSION OF TRANSFERRIN RECEPTOR 2
#302CHEMICALLY MODIFIED OLIGONUCLEOTIDE HAVING RNAi ACTIVITY
#303COMPOUNDS AND METHODS FOR REDUCING IFNAR1 EXPRESSION
#304KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#305SARM1 RNA INTERFERENCE AGENTS
#306Methods and Compositions for Treatment of Polycystic Kidney Disease
#307POLYNUCLEIC ACID MOLECULES TARGETING APOC3 AND USES THEREOF
#308POLYNUCLEIC ACID MOLECULES FOR INHIBITING EXPRESSION OF LP(A), PHARMACEUTICAL COMPOSITIONS, AND USES THEREOF
#309ANTISENSE OLIGONUCLEOTIDES FOR TARGETING PROGRANULIN
#310Antisense Nucleic Acids
#311FULLY STABILIZED ASYMMETRIC SIRNA
#312HIGH MOBILITY GROUP BOX-1 (HMGB1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#313LIPOPHILIC OLIGONUCLEOTIDE CONJUGATES
#314SIGNAL ACTIVATABLE NUCLEIC ACID CONSTRUCTS WITH WOBBLE BASE PAIRINGS
#315CONDITIONALLY ACTIVATABLE NUCLEIC ACID COMPLEXES
#316MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#317METHODS OF USING OLIGOMERIC COMPOUNDS TO TREAT SCN2A-RELATED DISORDERS
#318RNAI AGENT TARGETING MARC1 GENE, AND USE THEREOF
#319COMPOUNDS AND METHODS FOR MODULATING SMN2
#320COMPOSITIONS OF MODIFIED TREMS AND USES THEREOF
#321GENETICALLY ENGINEERED T CELLS WITH REGNASE-1 AND/OR TGFBRII DISRUPTION HAVE IMPROVED FUNCTIONALITY AND PERSISTENCE
#322COMPOSITIONS FOR THE MODIFICATION OF THE HUMAN NRAS AND KRAS GENES
#323COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
#324COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
#325COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#326Linear DNA with Enhanced Resistance Against Exonucleases
#327Microtubule Associated Protein Tau (MAPT) iRNA Agent Compositions and Methods of Use Thereof
#328NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
#329MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#330NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#331BACTERIA-FREE APPROACH FOR EXPRESSING NUCLEIC ACIDS AND PROTEINS IN EUKARYOTIC CELLS
#332THERAPEUTIC COMPOSITIONS FOR TREATING PAIN VIA MULTIPLE TARGETS
#333COMPOSITIONS AND METHODS FOR IMPROVING STRAND BIASED
#334ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
#335COMPOSITION FOR PREVENTION OR TREATMENT OF LIVER CANCER, COMPRISING MODIFIED RT-LET7 AS ACTIVE INGREDIENT
#336MODIFIED TRANSFER RNA WITH IMPROVED ACTIVITY
#337ENGINEERED RNA LIGASES
#338NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#339RNAI CONSTRUCTS FOR INHIBITING PNPLA3 EXPRESSION AND METHODS OF USE THEREOF
#340METHODS AND COMPOSITIONS FOR ACTIVATING TRANSLATION
#341LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
#342GUIDE OLIGONUCLEOTIDES FOR NUCLEIC ACID EDITING IN THE TREATMENT OF HYPERCHOLESTEROLEMIA
#343BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
#344NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
#345COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF PREKALLIKREIN (PKK) PROTEIN
#346Antisense Nucleic Acids
#347OLIGONUCLEOTIDES TARGETING RNA BINDING PROTEIN SITES
#348OLIGONUCLEOTIDES WITH ETHYLENE GLYCOL MODIFICATION
#349SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE
#350TREATMENT FOR SOD1 ASSOCIATED DISEASE
#351ANTISENSE OLIGONUCLEOTIDES TARGETING ADENOSINE KINASE
#352COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#353ANTISENSE OLIGONUCLEOTIDES TARGETING ALPHA-SYNUCLEIN AND USES THEREOF
#354MODULATORS OF MALAT1 EXPRESSION
#355OLIGONUCLEOTIDE AGONISTS TARGETING PROGRANULIN
#356Composition and Methods for Modulating Nuclear Enriched Abundant Transcript 1 to Treat Cognitive Impairment
#357MODULATION OF ALPHA SYNUCLEIN EXPRESSION
#358Methods and Compositions for Avoiding Off-Target Effects
#359iRNA Compositions and Methods for Silencing ANGPTL4
#360NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF COMPLEMENT FACTOR B (CFB) IN A CELL
#361COMPOSITIONS CONTAINING POLYNUCLEOTIDE AMPHIPHILES AND METHODS OF USE THEREOF
#362METHOD FOR REDUCING HEPATIC TRIGLYCERIDES
#363COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#364COMPOSITIONS AND METHODS FOR GENE MODIFICATION
#365COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#366COMBINATIONS WITH MODULATORS OF PNPLA3 EXPRESSION
#367RNAi OLIGONUCLEOTIDE CONJUGATES
#368OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#369PREKALLIKREIN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#370SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE
#371COMPOUNDS FOR INHIBITING AMANZI
#372COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF LPA
#373COMPOUNDS AND METHODS FOR MODULATING PROGRANULIN EXPRESSION
#374COMPOUNDS AND METHODS FOR THE MODULATION OF PROTEINS
#375METHODS OF TREATING IMMUNE DYSFUNCTION IN LIVER CANCER WITH TOLL-LIKE RECEPTOR AGONISTS
#376DEGRADATION OF RNA BY THE LYSOSOMAL PATHWAY
#377MODIFICATION PATTERNS FOR SMALL INTERFERING RNA MOLECULES WITH HIGH STABILITY AND GENE SILENCING ACTIVITIES
#378ADVANCED RNA TARGETING (ARNATAR)
#379METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#380MODIFIED siRNA FOR SELECTIVELY INHIBITING EXPRESSION OF MUTANT FUS
#381CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES (ASOS) AND COMPOSITIONS COMPRISING THE SAME FOR RNA EDITING
#382COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS
#383MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FRIEDREICH'S ATAXIA
#384TARGETED COMPOSITIONS
#385METHODS OF TREATMENT OF SCN2A-RELATED DISORDERS
#386PIKFYVE ANTISENSE OLIGONUCLEOTIDES
#387COMPOSITIONS AND METHODS FOR INHIBITING KETOHEXOKINASE (KHK)
#388COMBINATION THERAPY FOR TREATING HEPATITIS B VIRUS INFECTIONS
#389Modified Oligonucleotides and Methods of Use
#390GUIDE RNA WITH CHEMICAL MODIFICATIONS
#391COMPOSITIONS AND METHODS FOR SILENCING CARBONIC ANHYDRASE 2 EXPRESSION
#392HEPARIN SULFATE BIOSYNTHESIS PATHWAY ENZYME IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#393Antisense Nucleic Acid
#394COMPOSITIONS AND METHODS FOR CAPPING RNAS
#395Sequence Multiplicity within Spherical Nucleic Acids
#396PEPTIDE LIGANDS FOR CNS AND OCULAR DELIVERY OF RNAI COMPOUNDS
#397COMPOSITIONS AND METHODS FOR MODULATING NEURONAL EXCITABILITY AND MOTOR BEHAVIOR
#398RNAi Agents for Inhibiting Expression of Inhibin Subunit Beta E (INHBE), Pharmaceutical Compositions Thereof, and Methods of Use
#399TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES
#400RNAS FOR COMPLEMENT INHIBITION
#401AFFINITY REAGENT, MARKER AND METHOD FOR ANALYSING A BIOLOGICAL SAMPLE
#402MODIFIED AMINE LIPIDS
#403PREKALLIKREIN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#404Application of PRDX5 and GPX4 Genes in Preparation of Drugs for Castration-resistant Prostate Cancer
#405COMPOUNDS AND METHODS FOR MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION
#406RNA INTERFERENCE MEDIATED INHIBITION OF PROLYL HYDROXYLASE DOMAIN 2 (PHD2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#407MODULATORS OF APOL1 EXPRESSION
#408THERAPEUTIC OLIGONUCLEOTIDES
#409OLIGONUCLEOTIDES WITH INTERNUCLEOTIDE LINKAGE MODIFICATIONS FOR KIDNEY TARGETING
#410Advanced RNA targeting (ARNATAR) for angiotensinogen
#411METHODS AND AGENTS FOR INCREASING RBM3 EXPRESSION
#412ANTISENSE OLIGOMERS FOR TREATMENT OF CHRONIC KIDNEY DISEASE
#413OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#414RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#415AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS
#416COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#417CELL DEATH-INDUCING DFFA-LIKE EFFECTOR B (CIDEB) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#418SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE
#419SGLT-2 TARGETING OLIGONUCLEOTIDES
#420THERAPEUTIC OLIGONUCLEOTIDES HAVING AN INTER-NUCLEOSIDE AMIDE LINKAGE
#421TUNABLE REVERSIR TM COMPOUNDS
#422ANTISENSE OLIGONUCLEOTIDES (ASO) FOR EFFICIENT AND PRECISE RNA EDITING WITH ENDOGENOUS ADENOSINE DEAMINASE ACTING ON RNA (ADAR)
#423Modified Guide RNAs
#424NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID, PREPARATION METHOD THEREFOR AND USE THEREOF
#425COMPOUNDS AND METHODS FOR MODULATING GLYCOGEN SYNTHASE 1
#426OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#427OLIGONUCLEOTIDES FOR MODULATING TAU EXPRESSION
#428THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
#429Antisense Oligonucleotide for Targeting Progranulin
#430COMPOSITIONS COMPRISING THERAPEUTIC NUCLEIC ACID AND SAPONIN FOR THE TREATMENT OF MUSCLE-WASTING DISORDERS
#431ANTISENSE OLIGONUCLEOTIDE TARGETING ATN1 MRNA OR PRE-MRNA
#432TREATMENT OF NEUROLOGICAL DISEASES USING MODULATORS OF UNC13A GENE TRANSCRIPTS
#433APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#4343-HYDROXY-3-METHYLGLUTARYL-COA REDUCTASE (HMGCR) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#435OLIGONUCLEOTIDES FOR SNCA MODULATION
#436TREATMENT OF FGG RELATED DISEASES AND DISORDERS
#437SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#438ANTISENSE NUCLEIC ACIDS
#439CRISPR-CAS9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells and Uses Thereof
#440Subcutaneous Delivery of RNAi Agents for Inhibiting Expression of Receptor for Advanced Glycation End-products
#441RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Compositions Thereof, and Methods of Use
#442Double Stranded Oligonucleotide For Modulating JAK1 Expression
#443OLIGONUCLEOTIDES FOR DGAT2, FASN, OPN, AND NOX4 MODULATIONS
#444STEROL REGULATORY ELEMENT BINDING PROTEIN (SREBP) CHAPERONE (SCAP) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#445CELL DEATH-INDUCING DFFA-LIKE EFFECTOR B (CIDEB) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#446COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#447GLYCAN CONJUGATE COMPOSITIONS AND METHODS
#448Methods and Compositions for ADAR-Mediated Editing
#449Methods and Compositions for ADAR-Mediated Editing
#450COMPOSITIONS AND METHODS OF USING PLN-TARGETING ANTIBODY-OLIGONUCLEOTIDE CONJUGATES
#451MODULATING EXPRESSION OF ALAS1 (5'-AMINOLEVULINATE SYNTHASE 1) GENE
#452COMPOSITIONS AND METHODS FOR TREATMENT OF CANCERS USING MODIFIED siRNA-GEM AGENTS
#453METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
#454NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF TMPRSS6 AND IRON CHELATORS
#455ANTISENSE OLIGONUCLEOTIDE FOR REGULATING BCL2L12 EXPRESSION AND THE USE THEREOF IN TREATMENT OF DISEASES
#456IRNA COMPOSITIONS AND METHODS FOR SILENCING COMPLEMENT COMPONENT 3 (C3)
#457Oligonucleotides for Modulating Synaptogyrin-3 Expression
#458Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
#459ANTIVIRAL ANTISENSE OLIGONUCLEOTIDE
#460RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF NEUROMUSCULAR DISORDERS
#461COMPOSITIONS AND METHODS FOR ENHANCING GENE SILENCING ACTIVITY OF OLIGONUCLEOTIDE COMPOUNDS
#462HUNTINGTIN (HTT) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#463NEURODEGENERATIVE DISORDERS
#464OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF MAKING THE SAME
#465Methods and Compositions for Treating Cancers and Enhancing Therapeutic Immunity by Selectively Reducing Immunomodulatory M2 Monocytes
#466Compositions and Methods Using CPG Oligonucleotides
#467NUCLEIC ACID AGENTS FOR TREATMENT OF NON-SMALL-CELL LUNG CANCER
#468COMPOSITIONS AND METHODS FOR THE TREATMENT OF PCDH19 RELATED DISORDERS
#469COMPOSITIONS AND METHODS FOR INHIBITING COMPLEMENT COMPONENT 3 EXPRESSION
#470PARN AS A BIOMARKER AND THERAPEUTIC TARGET
#471OLIGONUCLEOTIDES FOR SARS-CoV-2 MODULATION
#472PHARMACEUTICAL COMBINATIONS FOR TREATMENT OF HBV
#473KISSPEPTIN 1 (KISS1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#474MODIFIED SMALL INTERFERING RNA MOLECULES WITH REDUCED OFF-TARGET EFFECTS
#475POLYNUCLEOTIDES USEFUL FOR CORRECTING MUTATIONS IN THE RAG1 GENE
#476THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
#477OLIGONUCLEOTIDES FOR TARGETING COMPLEMENT C5
#478OLIGONUCLEOTIDES FOR TISSUE SPECIFIC GENE EXPRESSION MODULATION
#479COMPOSITIONS AND METHODS FOR IMMUNOONCOLOGY
#480COMPOSITIONS AND METHODS FOR SKIPPING EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY
#481iRNA Compositions and Methods for Targeting ANGPTL7
#482COMBINATION OF OLIGONUCLEOTIDES FOR MODULATING RTEL1 AND FUBP1
#483LIPOPHILIC SIRNA CONJUGATES FOR THE TREATMENT OF CENTRAL NERVOUS SYSTEM DISEASES
#484CONDITION-ACTIVATABLE NUCLEIC ACID CONSTRUCTS AND THEIR USES FOR TREATING NEUROLOGICAL DISEASES
#485COMPOSITIONS AND METHODS FOR MODULATING NLRP3 OR NLRP1 EXPRESSION
#486TRANSTHYRETIN (TTR) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#487PREVENTIVE OR THERAPEUTIC AGENT FOR BENIGN ADULT FAMILIAL MYOCLONIC EPILEPSY
#488COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF MUTANT EGFR GENE
#489COMPOSITIONS AND METHODS FOR MODULATING PNPLA3 EXPRESSION
#490COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION
#491Compositions and methods for inhibiting LPA expression
#492UNC13A ANTISENSE OLIGONUCLEOTIDES
#493Tuning cascade assay kinetics via molecular design
#494TREM COMPOSITIONS AND METHODS OF USE
#495ANGIOTENSINOGEN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#496Compounds and Methods for Reducing ATXN2 Expression
#497COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#498AGENTS USEFUL IN TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#499THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
#500Chemically Modified Guide RNAs for CRISPR/CAS-Mediated Gene Correction
#501COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#502Muscle targeting complexes and uses thereof for treating myotonic dystrophy
#503Nucleic Acids and Methods for the Treatment of Pompe Disease
#504Treatment of MST1 related diseases and disorders
#505TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#506IRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
#507COMPOSITIONS AND METHODS FOR MODULATING KRAS EXPRESSION
#508RNAi Agents for Inhibiting Expression of Coronavirus (CoV) Viral Genomes, Compositions Thereof, and Methods of Use
#509BASE EDITING OF PCSK9 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
#510INHIBITOR OLIGONUCLEOTIDES AND METHODS OF USE THEREOF
#511HSD17B13-RELATED DOUBLE STRANDED OLIGONUCLEOTIDE COMPOSITIONS AND METHODS RELATING THERETO
#512ANGPTL4 OLIGONUCLEOTIDES INFLUENCING THE REGULATION OF THE FATTY ACID METABOLISM
#513NUCLEIC ACID MOLECULE AND ITS USE
#514CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#515MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR MODULATION OF GENES ASSOCIATED WITH MUSCLE HEALTH
#516COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#517COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3) PROTEIN
#518ANTISENSE OLIGONUCLEOTIDES FOR MODULATING TMEM106B EXPRESSION
#519Conditional Double Stranded Antisense Oligonucleotides
#520COMPOUNDS AND METHODS FOR MODULATING HUNTINGTIN
#521ENGINEERED PRODUCER CELL LINES AND METHODS OF MAKING AND USING THE SAME
#522USE OF microRNA MIMICS TO INHIBIT OR TREAT LIVER DISEASE
#523DYSTROPHIN EXON SKIPPING OLIGONUCLEOTIDES
#524HUNTINGTIN (HTT) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#525GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#526Selective Antisense Compounds and Uses Thereof
#527OLIGOMERIC COMPOUND FOR DYSTROPHIN RESCUE IN DMD PATIENTS THROUGHOUT SKIPPING OF EXON-51
#528LINEAR DNA WITH ENHANCED RESISTANCE AGAINST EXONUCLEASES AND METHODS FOR THE PRODUCTION THEREOF
#529CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING BETA HEMOGLOBINOPATHIES
#530Compounds and methods for reducing LRRK2 expression
#531METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
#532ANTISENSE OLIGONUCLEOTIDE THERAPY FOR H3.3 K27M DIFFUSE MIDLINE GLIOMAS
#533COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
#534LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
#535NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#536HAIRPIN NUCLEIC ACID COMPOSITION
#537CONJUGATE
#538ANTISENSE INHIBITORS OF MIR17HG PRE-RNA AS THERAPEUTIC AGENTS IN CANCER
#539DOUBLE STRANDED NUCLEIC ACID COMPOUNDS INHIBITING ZPI
#540Compositions and methods for modulating apolipoprotein (a) expression
#541TREATMENT FOR TYROSINE DEGRADATION-ASSOCIATED DISORDERS
#542DUAL FUNCTION HYBRID NANOPARTICLES AND METHODS OF USING THE SAME TO TREAT DISEASES AND DISORDERS
#543BETA-CATENIN (CTNNB1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#544COMPOSITIONS AND METHODS FOR INHIBITING LPA EXPRESSION
#545COMPOSITIONS AND METHODS FOR BCMA MODIFICATION
#546METHODS OF SYNTHESIZING OLIGONUCLEOTIDES USING TETHERED NUCLEOTIDES
#547NOVEL BICYCLIC NUCLEOSIDES AND OLIGOMERS PREPARED THEREFROM
#548MODIFIED NUCLEIC ACID COMPOSITIONS AND ASSOCIATED METHODS FOR TREATMENT OF PHENYLKETONURIA
#549Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs
#550RNA EDITING
#551COMPOUNDS AND METHODS FOR REDUCING DUX4 EXPRESSION
#5522'-MODIFIED NUCLEOSIDE BASED OLIGONUCLEOTIDE PRODRUGS
#553Compounds and Methods for Reducing DMPK Expression
#554NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF LPA IN A CELL
#555OLIGONUCLEOTIDE FOR INHIBITING QUAKING ACTIVITY
#556NOVEL THERAPEUTIC DELIVERY MOIETIES AND USES THEREOF
#557MODULATORS OF ENaC EXPRESSION
#558CONJUGATE OF SAPONIN, OLIGONUCLEOTIDE AND GALNAC
#559STRUCTURE-BASED DESIGN OF ANTISENSE OLIGONUCLEOTIDE DRUGS
#560ALLELE-SELECTIVE COMPOUNDS AND METHODS FOR MODULATING HUNTINGTIN EXPRESSION
#561METHODS OF GENERATING CORE STRANDS IN CONDITIONALLY ACTIVATABLE NUCLEIC ACID COMPLEXES
#562MODULATION OF PREKALLIKREIN (PKK) EXPRESSION
#563Antisense oligonucleotide targeting ATN1 mRNA or pre-mRNA
#564ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B
#565Multi-Valent Oligonucleotide Agent and Methods of Use Thereof
#566CRISPR-BASED COMPOSITIONS AND METHODS OF USE
#567Compositions and Methods for Gene Editing in T Cells Using CRISPR/Cpf1
#568COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION
#569COMPOSITIONS AND METHODS FOR IMPROVED GENE EDITING
#570COMPOSITIONS AND METHODS FOR INHIBITING TRANSMEMBRANE SERINE PROTEASE 6 (TMPRSS6) EXPRESSION
#571TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#572COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#573SMALL INTERFERING RNA TARGETING HBV AND USES THEREOF
#574REDUCING INTRON RETENTION
#575COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
#576METABOLIC DISORDER-ASSOCIATED TARGET GENE IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#577METHODS FOR ENHANCING UTROPHIN PRODUCTION VIA INHIBITION OF MICRORNA
#578CHEMICALLY MODIFIED OLIGONUCLEOTIDES
#579COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#580METHODS AND COMPOSITIONS FOR TREATING A PROPROTEIN CONVERTASE SUBTILISIN KEXIN (PCSK9) GENE-ASSOCIATED DISORDER
#581COMPOUNDS AND METHODS FOR REDUCING TAU EXPRESSION
#582SMALL INTERFERING RNA TARGETING C3 AND USES THEREOF
#583iRNA Compositions and Methods for Silencing Filamin A (FLNA)
#584COMPOUNDS FOR MODULATING UNC13A EXPRESSION
#585MICRORNA-134 INHIBITORS
#586COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#587COMPOUNDS AND METHODS FOR REDUCING APP EXPRESSION
#588OLIGONUCLEOTIDES FOR HTT-1A MODULATION
#589Chemically Modified Small Activating RNA
#590COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#591NEW CONJUGATED NUCLEIC ACID MOLECULES AND THEIR USES
#592ANTISENSE-OLIGONUCLEOTIDES FOR PREVENTION OF KIDNEY DYSFUNCTION PROMOTED BY ENDOTHELIAL DYSFUNCTION BY EPHRIN-B2 SUPPRESSION
#593Selective Antisense Compounds and Uses Thereof
#594FIDGETIN-LIKE 2 AS A TARGET TO ENHANCE WOUND HEALING
#595SMALL INTERFERING RNA TARGETING CFB AND USES THEREOF
#596Oligonucleotides for Modulating Apolipoprotein E4 Expression
#597Methods and Compositions for Treatment of Polycystic Kidney Disease
#598COMPLEMENT COMPONENT C3 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#599INSULIN-LIKE GROWTH FACTOR BINDING PROTEIN, ACID LABILE SUBUNIT (IGFALS) AND INSULIN-LIKE GROWTH FACTOR 1 (IGF-1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#600Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of use thereof to target dystrophin and to treat Duchenne muscular dystrophy