109170 ⎘
Structure or type of the nucleic acid; Chemical structure of the sugar 2'-O-R Modification
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#302SARM1 RNA INTERFERENCE AGENTS
#303NONCOVALENTLY BRANCHED OLIGONUCLEOTIDE COMPOSITIONS
#304MULTIPLEXING TARGETING LIGANDS THROUGH CLICK CHEMISTRY AT THE ANOMERIC SITE OF SUGARS
#305Methods and Compositions for Treatment of Polycystic Kidney Disease
#306POLYNUCLEIC ACID MOLECULES TARGETING APOC3 AND USES THEREOF
#307POLYNUCLEIC ACID MOLECULES FOR INHIBITING EXPRESSION OF LP(A), PHARMACEUTICAL COMPOSITIONS, AND USES THEREOF
#308ANTISENSE OLIGONUCLEOTIDES FOR TARGETING PROGRANULIN
#309Antisense Nucleic Acids
#310FULLY STABILIZED ASYMMETRIC SIRNA
#311CONDITIONALLY ACTIVATABLE NUCLEIC ACID COMPLEXES
#312METHODS OF USING OLIGOMERIC COMPOUNDS TO TREAT SCN2A-RELATED DISORDERS
#313RNAI AGENT TARGETING MARC1 GENE, AND USE THEREOF
#314USE OF DINUCLEOTIDE REPEAT RNAS TO TREAT CANCER
#315ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TREATING DYSTROPHIES
#316COMPOUNDS AND METHODS FOR MODULATING SMN2
#317COMPOSITIONS OF MODIFIED TREMS AND USES THEREOF
#318ATXN2 RNA INTERFERENCE AGENTS
#319METHODS OF TREATING GLAUCOMA
#320HETERONUCLEIC ACID INCLUDING 2'-MODIFIED NUCLEOSIDE
#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
#323COMPOSITIONS AND METHODS FOR THE TREATMENT OF EXPANDED REPEAT-ASSOCIATED DISORDERS
#324APTAMERS, PHARMACEUTICAL COMPOSITIONS THEREOF, AND METHODS FOR PREVENTING OR TREATING BCMA-ASSOCIATED DISEASES OR CONDITIONS
#325COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE SIGNAL REGULATORY PROTEIN ALPHA (SIRPa) GENE
#326COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
#327COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#328Microtubule Associated Protein Tau (MAPT) iRNA Agent Compositions and Methods of Use Thereof
#329NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
#330THERAPEUTIC COMPOSITIONS FOR TREATING PAIN VIA MULTIPLE TARGETS
#331ANTISENSE OLIGONUCLEOTIDES FOR INDUCING EXON SKIPPING AND METHODS OF USE THEREOF
#332COMPOSITION FOR PREVENTION OR TREATMENT OF LIVER CANCER, COMPRISING MODIFIED RT-LET7 AS ACTIVE INGREDIENT
#333MODIFIED TRANSFER RNA WITH IMPROVED ACTIVITY
#334COMPOUND FOR INHIBITING C3 GENE EXPRESSION, PHARMACEUTICAL COMPOSITION AND USE THEREOF
#335RNAI CONSTRUCTS FOR INHIBITING PNPLA3 EXPRESSION AND METHODS OF USE THEREOF
#336MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
#337BASE EDITING OF ANGPTL3 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
#338Antisense Nucleic Acids
#339OLIGONUCLEOTIDES TARGETING RNA BINDING PROTEIN SITES
#340OLIGONUCLEOTIDES WITH ETHYLENE GLYCOL MODIFICATION
#341SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE
#342COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#343MODULATORS OF MALAT1 EXPRESSION
#344Methods and Compositions for Avoiding Off-Target Effects
#345iRNA Compositions and Methods for Silencing ANGPTL4
#346COMPOSITIONS FOR EDITING MECP2 TRANSCRIPTS AND METHODS THEREOF
#347COMPOSITIONS AND METHODS FOR PREPARING CAPPED CIRCULAR RNA MOLECULES
#348METHOD FOR REDUCING HEPATIC TRIGLYCERIDES
#349COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#350ACETYLATED RIBONUCLEIC ACIDS AND USES THEREOF
#351COMPOSITIONS AND METHODS FOR GENE MODIFICATION
#352COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#353COMBINATIONS WITH MODULATORS OF PNPLA3 EXPRESSION
#354Bifunctional Molecules and Methods of Using Thereof
#355RNAi OLIGONUCLEOTIDE CONJUGATES
#356ENGINEERED ADENYLATE KINASE VARIANTS
#357OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#358PREKALLIKREIN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#359COMPOUNDS FOR INHIBITION OF IL-1BETA EXPRESSION
#360SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE
#361MODULATING THE CELLULAR STRESS RESPONSE
#362COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF LPA
#363COMPOSITIONS AND METHODS FOR TREATMENT OF PAIN
#364COMPOSITIONS AND METHODS OF USING PRKAG2-TARGETING ANTIBODY-OLIGONUCLEOTIDE CONJUGATES
#365DEGRADATION OF RNA BY THE LYSOSOMAL PATHWAY
#366MODIFICATION PATTERNS FOR SMALL INTERFERING RNA MOLECULES WITH HIGH STABILITY AND GENE SILENCING ACTIVITIES
#367ADVANCED RNA TARGETING (ARNATAR)
#368METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#369MODIFIED siRNA FOR SELECTIVELY INHIBITING EXPRESSION OF MUTANT FUS
#370CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES (ASOS) AND COMPOSITIONS COMPRISING THE SAME FOR RNA EDITING
#371COMPOSITIONS AND METHODS FOR TREATMENT OF TRANSTHYRETIN AMYLOIDOSIS
#372MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FRIEDREICH'S ATAXIA
#373TARGETED COMPOSITIONS
#374IONIZABLE LIPIDS, LIPID NANOPARTICLES, AND USES THEREOF
#375METHODS OF TREATMENT OF SCN2A-RELATED DISORDERS
#376PIKFYVE ANTISENSE OLIGONUCLEOTIDES
#377COMPOSITIONS AND METHODS FOR INHIBITING KETOHEXOKINASE (KHK)
#378COMBINATION THERAPY FOR TREATING HEPATITIS B VIRUS INFECTIONS
#379ANTISENSE OLIGONUCLEOTIDES AND THEIR USE FOR TREATMENT OF NEURODEGENERATIVE DISORDERS
#380GUIDE RNA WITH CHEMICAL MODIFICATIONS
#381HEPARIN SULFATE BIOSYNTHESIS PATHWAY ENZYME IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#382COMPOSITIONS COMPRISING THERAPEUTIC NUCLEIC ACID AND TARGETED SAPONIN FOR THE TREATMENT OF MUSCLE-WASTING DISORDERS
#383PEPTIDE LIGANDS FOR CNS AND OCULAR DELIVERY OF RNAI COMPOUNDS
#384COMPOSITIONS AND METHODS FOR MODULATING NEURONAL EXCITABILITY AND MOTOR BEHAVIOR
#385RNAi Agents for Inhibiting Expression of Inhibin Subunit Beta E (INHBE), Pharmaceutical Compositions Thereof, and Methods of Use
#386RNAS FOR COMPLEMENT INHIBITION
#387MODIFIED AMINE LIPIDS
#388PREKALLIKREIN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#389ACVR1R206H ALLELE-SPECIFIC THERAPY AND USES THEREOF FOR TREATMENT OF FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
#390Application of PRDX5 and GPX4 Genes in Preparation of Drugs for Castration-resistant Prostate Cancer
#391ANTI-SENSE OLIGONUCLEOTIDES AND USES THEREOF
#392RNA INTERFERENCE MEDIATED INHIBITION OF PROLYL HYDROXYLASE DOMAIN 2 (PHD2) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#393ANTISENSE OLIGONUCLEOTIDES FOR MODIFYING PROTEIN EXPRESSION
#394MODULATORS OF APOL1 EXPRESSION
#395THERAPEUTIC OLIGONUCLEOTIDES
#396METHODS AND AGENTS FOR INCREASING RBM3 EXPRESSION
#397OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#398RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#399AMYLOID PRECURSOR PROTEIN (APP) RNAi AGENTS
#400CELL DEATH-INDUCING DFFA-LIKE EFFECTOR B (CIDEB) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#401SNCA-TARGETING SIRNA COMPOSITIONS FOR TREATING SNCA-ASSOCIATED DISEASE
#402THERAPEUTIC OLIGONUCLEOTIDES HAVING AN INTER-NUCLEOSIDE AMIDE LINKAGE
#403ANTISENSE OLIGONUCLEOTIDES (ASO) FOR EFFICIENT AND PRECISE RNA EDITING WITH ENDOGENOUS ADENOSINE DEAMINASE ACTING ON RNA (ADAR)
#404NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID, PREPARATION METHOD THEREFOR AND USE THEREOF
#405INHIBIN SUBUNIT BETA E (INHBE) MODULATOR COMPOSITIONS AND METHODS OF USE THEREOF
#406OLIGONUCLEOTIDES FOR MODULATING TAU EXPRESSION
#407THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
#408COMPOSITIONS COMPRISING THERAPEUTIC NUCLEIC ACID AND SAPONIN FOR THE TREATMENT OF MUSCLE-WASTING DISORDERS
#409ANTISENSE OLIGONUCLEOTIDE TARGETING ATN1 MRNA OR PRE-MRNA
#410iRNA Compositions and Methods for Silencing MYLIP
#411TREATMENT OF NEUROLOGICAL DISEASES USING MODULATORS OF UNC13A GENE TRANSCRIPTS
#412APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#413Antisense Compounds Targeting Genes Associated with Cystic Fibrosis
#4143-HYDROXY-3-METHYLGLUTARYL-COA REDUCTASE (HMGCR) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#415PRODUCTS AND COMPOSITIONS
#416OLIGONUCLEOTIDES FOR SNCA MODULATION
#417TREATMENT OF FGG RELATED DISEASES AND DISORDERS
#418ANTISENSE NUCLEIC ACIDS
#419CRISPR-CAS9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells and Uses Thereof
#420RNAi Agents for Inhibiting Expression of PNPLA3, Pharmaceutical Compositions Thereof, and Methods of Use
#421Double Stranded Oligonucleotide For Modulating JAK1 Expression
#422OLIGONUCLEOTIDES FOR DGAT2, FASN, OPN, AND NOX4 MODULATIONS
#423METHODS OF SILENCING EXPRESSION OF GENES AND USES THEREOF
#424NON-LIPOSOMAL SYSTEMS FOR NUCLEIC ACID DELIVERY
#425ANTISENSE OLIGONUCLEOTIDE DRUG TARGETS
#426CELL DEATH-INDUCING DFFA-LIKE EFFECTOR B (CIDEB) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#427COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#428GLYCAN CONJUGATE COMPOSITIONS AND METHODS
#429Methods and Compositions for ADAR-Mediated Editing
#430Methods and Compositions for ADAR-Mediated Editing
#431COMPOSITIONS AND METHODS OF USING PLN-TARGETING ANTIBODY-OLIGONUCLEOTIDE CONJUGATES
#432DOUBLE STRANDED RNA TARGETING PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) AND METHODS OF USE THEREOF
#433MODULATING EXPRESSION OF ALAS1 (5'-AMINOLEVULINATE SYNTHASE 1) GENE
#434COMPOSITIONS AND METHODS FOR TREATMENT OF CANCERS USING MODIFIED siRNA-GEM AGENTS
#435COMPOSITIONS FOR INHIBITING DIPEPTIDE REPEAT PROTEIN-RIBOSOMAL RNA INTERACTION AND USES THEREOF
#436METHODS AND COMPOSITIONS FOR INHIBITING PMP22 EXPRESSION
#437NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF TMPRSS6 AND IRON CHELATORS
#438GATA4-TARGETED THERAPEUTICS FOR TREATMENT OF CARDIAC HYPERTROPHY
#439COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE LECT2 GENE
#440IRNA COMPOSITIONS AND METHODS FOR SILENCING COMPLEMENT COMPONENT 3 (C3)
#441LACTATE DEHYDROGENASE A (LDHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#442Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
#443RNA MODULATING OLIGONUCLEOTIDES WITH IMPROVED CHARACTERISTICS FOR THE TREATMENT OF NEUROMUSCULAR DISORDERS
#444COMPOSITIONS AND METHODS FOR ENHANCING GENE SILENCING ACTIVITY OF OLIGONUCLEOTIDE COMPOUNDS
#445COMPOSITIONS AND METHODS FOR TREATING TDP-43 PROTEINOPATHY
#446Stabilized CRISPR Complexes
#447METHOD OF TREATMENT FOR OPTIC ATROPHY
#448MOLECULAR THERAPEUTICS FOR THE TREATMENT OF HYPERTROPHIC CARDIOMYOPATHY AND HEART FAILURE ASSOCIATED WITH MYBPC3 GENE MUTATIONS
#449NUCLEIC ACIDS CONTAINING ABASIC NUCLEOSIDES
#450COMPOSITIONS AND METHODS FOR THE TREATMENT OF PCDH19 RELATED DISORDERS
#451TREATMENT OF COMPLEMENT-MEDIATED DISORDERS
#452COMPOSITIONS AND METHODS FOR INHIBITING COMPLEMENT COMPONENT 3 EXPRESSION
#453TREM COMPOSITIONS AND USES THEREOF
#454METHODS FOR TREATING OR PREVENTING OPHTHALMOLOGICAL CONDITIONS
#455SIRNA STRUCTURES FOR HIGH ACTIVITY AND REDUCED OFF TARGET
#456OLIGONUCLEOTIDES FOR SARS-CoV-2 MODULATION
#457PHARMACEUTICAL COMBINATIONS FOR TREATMENT OF HBV
#458MODIFIED SMALL INTERFERING RNA MOLECULES WITH REDUCED OFF-TARGET EFFECTS
#459METHODS AND COMPOSITIONS FOR MODULATING A GENOME
#460TIS-L TARGETING ANTIVIRAL ANTISENSE OLIGONUCLEOTIDE WITH ABILITY TO INHIBIT VIRAL REPLICATION OF SARS-COV-2
#461METHODS OF MODULATING RNA TRANSLATION
#462THERAPEUTIC TREATMENT FOR FRAGILE X-ASSOCIATED DISORDER
#463OLIGONUCLEOTIDES FOR TARGETING COMPLEMENT C5
#464COMPOSITIONS AND METHODS FOR IMMUNOONCOLOGY
#465SINGLE-STRANDED RNA-EDITING OLIGONUCLEOTIDES
#466COMPOSITIONS AND METHODS FOR SKIPPING EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY
#467CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR KNOCKING OUT C5
#468CONDITION-ACTIVATABLE NUCLEIC ACID CONSTRUCTS AND THEIR USES FOR TREATING NEUROLOGICAL DISEASES
#469CORRECTION OF SPLICING MUTATIONS CAUSING PRIMARY CILIARY DYSKINESIA USING OLIGONUCLEOTIDES
#470PREVENTIVE OR THERAPEUTIC AGENT FOR BENIGN ADULT FAMILIAL MYOCLONIC EPILEPSY
#471Aptamer for DKK1 and Use Thereof
#472COMPOUNDS AND METHODS FOR REDUCING PLN EXPRESSION
#473COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF MUTANT EGFR GENE
#474COMPOSITIONS AND METHODS FOR MODULATING PNPLA3 EXPRESSION
#475Compositions and methods for inhibiting LPA expression
#476UNC13A ANTISENSE OLIGONUCLEOTIDES
#477TREM COMPOSITIONS AND METHODS OF USE
#478ANGIOTENSINOGEN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#479COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#480AGENTS USEFUL IN TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#481THERAPEUTICS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
#482Chemically Modified Guide RNAs for CRISPR/CAS-Mediated Gene Correction
#483COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#484THERAPEUTIC USES AND METHODS
#485Nucleic Acids and Methods for the Treatment of Pompe Disease
#486TREATMENT OF MST1R RELATED DISEASES AND DISORDERS
#487Treatment of MST1 related diseases and disorders
#488TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#489IRNA COMPOSITIONS AND METHODS FOR SILENCING ANGIOTENSINOGEN (AGT)
#490COMPOSITIONS AND METHODS FOR MODULATING KRAS EXPRESSION
#491RNAi Agents for Inhibiting Expression of Coronavirus (CoV) Viral Genomes, Compositions Thereof, and Methods of Use
#492NUCLEOTIDE AND USE THEREOF
#493BASE EDITING OF PCSK9 AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
#494GENE EDITING OF PCSK9 OR ANGPTL3 AND COMPOSITIONS AND METHODS OF USING SAME FOR TREATMENT OF DISEASE
#495MICRORNA-29 COMPOUNDS, COMPOSITIONS AND USES IN THERAPY
#496Oligonucleotides
#497GENOME EDITING COMPOSITIONS AND METHODS FOR TREATMENT OF USHER SYNDROME TYPE 3
#498CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#499TREATMENT OF POLYCYTHEMIA VERA VIA CRISPR/AAV6 GENOME EDITING
#500APTAMERS FOR TARGETED ACTIVATION OF T CELL-MEDIATED IMMUNITY
#501COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#502COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3) PROTEIN
#503ANTISENSE OLIGONUCLEOTIDES FOR MODULATING TMEM106B EXPRESSION
#504ANTISENSE COMPOUND FOR MODULATING WFDC2 EXPRESSION
#505ENGINEERED PRODUCER CELL LINES AND METHODS OF MAKING AND USING THE SAME
#506USE OF microRNA MIMICS TO INHIBIT OR TREAT LIVER DISEASE
#507IL-34 ANTISENSE AGENTS AND METHODS OF USING SAME
#508OLIGONUCLEOTIDES COMPRISING SEGMENTED GAP STRUCTURES
#509Antisense Nucleic Acids
#510DYSTROPHIN EXON SKIPPING OLIGONUCLEOTIDES
#511HUNTINGTIN (HTT) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#512GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#513Selective Antisense Compounds and Uses Thereof
#514OLIGOMERIC COMPOUND FOR DYSTROPHIN RESCUE IN DMD PATIENTS THROUGHOUT SKIPPING OF EXON-51
#515COMPOSITIONS AND METHODS FOR CD34 GENE MODIFICATION
#516Compounds and methods for reducing LRRK2 expression
#517METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
#518MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
#519COMPOUNDS AND METHODS FOR MODULATING SCN1A EXPRESSION
#520LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
#521MICRORNA-29 COMPOUNDS, COMPOSITIONS AND USES IN THERAPY, AND METHODS FOR EVALUATING TENDINOPATHIC LESIONS
#522COMPOSITIONS AND METHODS FOR SIRNA TREATMENT OF MUSCULAR DYSTROPHY
#523Compositions and methods for modulating apolipoprotein (a) expression
#524PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#525DUAL FUNCTION HYBRID NANOPARTICLES AND METHODS OF USING THE SAME TO TREAT DISEASES AND DISORDERS
#526BETA-CATENIN (CTNNB1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#527COMPOSITIONS AND METHODS FOR INHIBITING LPA EXPRESSION
#528COMPOSITIONS AND METHODS FOR BCMA MODIFICATION
#529THREOSE NUCLEIC ACID ANTISENSE OLIGONUCLEOTIDES AND METHODS THEREOF
#530RNA EDITING
#5312'-MODIFIED NUCLEOSIDE BASED OLIGONUCLEOTIDE PRODRUGS
#532ANTISENSE OLIGONUCLEOTIDES TARGETING UNC13A
#533ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TREATING DYSTROPHIES
#534OLIGONUCLEOTIDE FOR INHIBITING QUAKING ACTIVITY
#535NOVEL THERAPEUTIC DELIVERY MOIETIES AND USES THEREOF
#536MODULATORS OF ENaC EXPRESSION
#537LIPID FORMULATED COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF SERUM AMYLOID A GENE
#538DISE-INDUCING SRNA-POLYPLEXES AND SRNA-LIPOPOLYPLEXES AND METHODS OF USING THE SAME TO TREAT CANCER
#539STRUCTURE-BASED DESIGN OF ANTISENSE OLIGONUCLEOTIDE DRUGS
#540NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING SAME, AND PREPARATION METHOD AND USE
#541METHODS OF GENERATING CORE STRANDS IN CONDITIONALLY ACTIVATABLE NUCLEIC ACID COMPLEXES
#542PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#543MODULATION OF PREKALLIKREIN (PKK) EXPRESSION
#544Antisense oligonucleotide targeting ATN1 mRNA or pre-mRNA
#545ANTISENSE OLIGONUCLEOTIDES TARGETING ACTL6B
#546MORPHOLINO OLIGOMERS FOR TREATMENT OF PERIPHERAL MYELIN PROTEIN 22 RELATED DISEASES
#547Multi-Valent Oligonucleotide Agent and Methods of Use Thereof
#548CRISPR-BASED COMPOSITIONS AND METHODS OF USE
#549NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS
#550COMPOUNDS AND METHODS FOR INCREASING STMN2 EXPRESSION
#551TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#552COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#553SMALL INTERFERING RNA TARGETING HBV AND USES THEREOF
#554COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#555DOUBLE-STRANDED OLIGONUCLEOTIDE, COMPOSITION AND CONJUGATE CONTAINING DOUBLE-STRANDED OLIGONUCLEOTIDE, AND PREPARATION METHODS AND USES
#556REDUCING INTRON RETENTION
#557COMPOSITIONS FOR MODULATING ATAXIN 2 EXPRESSION
#558STABILIZATION OF OLIGONUCLEOTIDE AND OLIGONUCLEOTIDE-PROTEIN COMPLEX USING ALKYLATED PHOSPHATE
#559METABOLIC DISORDER-ASSOCIATED TARGET GENE IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#560METHODS FOR ENHANCING UTROPHIN PRODUCTION VIA INHIBITION OF MICRORNA
#561MODIFIED DOUBLE-STRANDED DONOR TEMPLATES
#562CHEMICALLY MODIFIED OLIGONUCLEOTIDES
#563COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#564METHODS AND COMPOSITIONS FOR TREATING A PROPROTEIN CONVERTASE SUBTILISIN KEXIN (PCSK9) GENE-ASSOCIATED DISORDER
#565SMALL INTERFERING RNA TARGETING C3 AND USES THEREOF
#566Combination of Antisense Oligomers
#567COMPOUNDS FOR MODULATING UNC13A EXPRESSION
#568OLIGONUCLEOTIDES FOR HTT-1A MODULATION
#569Chemically Modified Small Activating RNA
#570COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#571ANTISENSE OLIGONUCLEOTIDES TARGETING SCN2A FOR THE TREATMENT OF SCN1A ENCEPHALOPATHIES
#572MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING POMPE DISEASE
#573MODULATION OF NOX4 EXPRESSION
#574FIDGETIN-LIKE 2 AS A TARGET TO ENHANCE WOUND HEALING
#575SMALL INTERFERING RNA TARGETING CFB AND USES THEREOF
#576Methods and Compositions for Treatment of Polycystic Kidney Disease
#577COMPLEMENT COMPONENT C3 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#578INSULIN-LIKE GROWTH FACTOR BINDING PROTEIN, ACID LABILE SUBUNIT (IGFALS) AND INSULIN-LIKE GROWTH FACTOR 1 (IGF-1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#579GLP-1 RECEPTOR LIGAND MOIETY CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#580NOVEL PROCESSES FOR THE PRODUCTION OF OLIGONUCLEOTIDES
#581COMPOSITIONS AND METHODS FOR DELIVERING THERAPEUTIC OLIGONUCLEOTIDES TO THE CENTRAL NERVOUS SYSTEM
#582GLUCOKINASE (GCK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#583TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#584COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#585Allele-specific silencing therapy for DFNA21 using antisense oligonucleotides
#586TREATMENT OF DKK2 RELATED DISEASES AND DISORDERS
#587TREATMENT OF MTRES1 RELATED DISEASES AND DISORDERS
#588TREATMENT OF PLIN1 RELATED DISEASES AND DISORDERS
#589RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACIDS (SINA)
#590OLIGONUCLEOTIDES FOR IFN-GAMMA SIGNALING PATHWAY MODULATION
#591LIPID CONJUGATION FOR TARGETING NEURONS OF THE CENTRAL NERVOUS SYSTEM
#592OLIGONUCLEOTIDES
#593Compounds and Methods for Modulating GFAP
#594Conjugated antisense compounds and their use
#595DOUBLE STRANDED OLIGONUCLEOTIDE COMPOSITIONS AND METHODS RELATING THERETO
#596COMPOUNDS FOR REDUCING PTBP1 EXPRESSION
#597FOLLICULIN iRNA COMPOSITIONS AND METHODS THEREOF
#598RNAi AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
#599MODIFIED MRNA, MODIFIED NON-CODING RNA, AND USES THEREOF
#600SODIUM-GLUCOSE COTRANSPORTER-2 (SGLT2) IRNA COMPOSITIONS AND METHODS OF USE THEREOF