109191 ⎘
Structure or type of the nucleic acid; Chemical structure; Spatial arrangement of the modifications having a combination of backbone and sugar modifications
KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#302MINIATURIZED HAIRPIN RNAi TRIGGERS (mxRNA) AND METHODS OF USES THEREOF
#303Antisense oligonucleotides targeting SCN2A for the treatment of SCN1A encephalopathies
#304SERPINA1 iRNA compositions and methods of use thereof
#305Compositions and methods for modulating neuronal excitability and motor behavior
#306Compounds and Methods for Modulation of Transcript Processing
#307COMPOUNDS OF CHEMICALLY MODIFIED OLIGONUCLEOTIDES AND METHODS OF USE THEREOF
#308MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FRIEDREICH'S ATAXIA
#309RNAi agents for inhibiting expression of PNPLA3, pharmaceutical compositions thereof, and methods of use
#310Oligonucleotides targeting frataxin and related methods
#311METHODS AND COMPOSITIONS FOR TARGETING PD-L1
#312Compounds and methods for the modulation of proteins
#313ANTISENSE OLIGONUCLEOTIDES TARGETING TIA1
#314Compounds and methods for increasing STMN2 expression
#315Antisense oligonucleotides to restore dysferlin protein expression in dysferlinopathy patient cells
#316PCSK9 targeting oligonucleotides for treating hypercholesterolemia and related conditions
#317RNAi Agents And Compositions for Inhibiting Expression of Angiopoietin-Like 3 (ANGPTL3), And Methods Of Use
#318Compositions for Modulating C9ORF72 Expression
#319MUSCLE-TARGETING COMPLEXES AND USES THEREOF
#320METHODS FOR REACTIVATING GENES ON THE INACTIVE X CHROMOSOME
#321COMPOSITIONS FOR MODULATING EXPRESSION OF C9ORF72 ANTISENSE TRANSCRIPT
#322Compositions and methods for modulating growth hormone receptor expression
#323SEQUENTIAL PAIRED-END SEQUENCING
#324Patatin-like phospholipase domain containing 3 (PNPLA3) iRNA compositions and methods of use thereof
#325S-ANTIGEN TRANSPORT INHIBITING OLIGONUCLEOTIDE POLYMERS AND METHODS
#326Compositions and methods for inhibiting gene expression of Hif2alpha
#327MODULATORS OF ENaC EXPRESSION
#328Modulation of factor 11 expression
#329BBB-crossing lipid ligand of hetero nucleic acid
#330Methods for targeting pain directed at metabolic pathways
#331METHODS FOR TREATING SICKLE CELL DISEASE
#332Methods for treating multiple sclerosis using antisense oligonucleotides
#333SiRNAs with vinylphosphonate at the 5′ end of the antisense strand
#334Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#335Oligonucleotides for reduction of PD-L1 expression
#336Oligonucleotide therapy for Leber congenital amaurosis
#337Two-tailed self-delivering siRNA
#338Modulation of Huntington expression
#339COMPOSITIONS AND METHODS
#340POLYNUCLEOTIDE AGENTS TARGETING SERPINC1 (AT3) AND METHODS OF USE THEREOF
#341COMPOSITIONS COMPRISING EICOSAPENTAENOIC ACID AND MIPOMERSEN AND METHODS OF USE THEREOF
#342Modified oligonucleotides for use in treatment of tauopathies
#343HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
#344Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA
#345Compositions and methods for modulating apolipoprotein (a) expression
#346Modulation of alpha synuclein expression
#347Modulators of APOL1 expression
#348GUIDE RNA WITH CHEMICAL MODIFICATIONS
#349Compositions for modulating Tau expression
#350Interfering RNA molecules
#351Oligomers and oligomer conjugates
#352Compounds and methods for the modulation of amyloid-β precursor protein
#353Compositions and Methods for Correcting Limb Girdle Muscular Dystrophy Type 2C Using Exon Skipping
#354Compositions and methods for modulation of SMN2 splicing in a subject
#355Oligonucleotide compositions and methods thereof
#356Fully stabilized asymmetric siRNA
#357COMPOSITIONS AND METHODS FOR MODULATING TTR EXPRESSION
#358COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN
#359Modified double-stranded RNA agents
#360Inhibition of polyomavirus replication
#361Sterol regulatory element binding protein (SREBP) chaperone (SCAP) iRNA compositions and methods of use thereof
#362Selective antisense compounds and uses thereof
#363Targeted compositions
#364COMPOUNDS OF CHEMICALLY MODIFIED OLIGONUCLEOTIDES AND METHODS OF USE THEREOF
#365ANTISENSE OLIGONUCLEOTIDES FOR MODULATING HTRA1 EXPRESSION
#366Oligonucleotides matching COL7A1 exon 73 for epidermolysis bullosa therapy
#367MODULATORS OF DNM2 EXPRESSION
#368METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#369Short interfering nucleic acid (siNA) compositions
#370POLYNUCLEOTIDE CONSTRUCTS HAVING BIOREVERSIBLE AND NON-BIOREVERSIBLE GROUPS
#371Compositions and methods for treatment of cardiac diseases
#372Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#373MODULATION OF SIGNAL TRANSDUCER AND ACTIVATOR OF TRANSCRIPTION 3 (STAT3) EXPRESSION
#374Angelman syndrome antisense treatment
#375Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
#376METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#377Compositions for modulating SOD-1 expression
#378Modified RNAi agents
#379ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
#380Oligonucleotides for inducing paternal UBE3A expression
#381COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#382ANTI-TNF COMPOUNDS
#383EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#384OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#385High Specificity Genome Editing Using Chemically Modified Guide RNAs
#386ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES FROM PROTEINS
#387GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#388Compositions and their uses directed to huntingtin
#389CANCER TREATMENT
#390RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
#391STEREODEFINED SUB-MOTIF OPTIMISATION METHODS
#392Compositions and methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
#393HIGH MOBILITY GROUP BOX-1 (HMGB1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#394Thiomorpholino oligonucleotides for the treatment of muscular dystrophy
#395Oligonucleotide compounds for targeting huntingtin mRNA
#396PRIMARY CELL GENE EDITING
#397Oligonucleotide compositions and methods thereof
#398RNAi agents for inhibiting expression of alpha-ENaC and methods of use
#399RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
#400Methods for the treatment of Leber congenital amaurosis
#401Gapmers and methods of using the same for the treatment of muscular dystrophy
#402Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#403Compositions and methods for inhibiting expression of RRM2 genes
#404ANTISENSE COMPOUNDS
#405Single-Stranded RNAI Oligonucleotides Targeting APOC-III
#406Modulation of huntingtin expression
#407Complement component iRNA compositions and methods of use thereof
#408Antisense oligonucleotides for the treatment of leber congenital amaurosis
#409Oligonucleotides for reduction of PD-L1 expression
#410Compositions and methods for modulation of protein aggregation
#411Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA
#412COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
#413Reducing intron retention
#414Nucleic acid oligomers and uses therefor
#415ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES, THE HCHWA-D MUTATION, AND TRINUCLEOTIDE REPEAT EXPANSIONS
#416Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#417COMPOSITIONS AND METHODS FOR MODULATING HBV EXPRESSION
#418STABILIZED SARNA COMPOSITIONS AND METHODS OF USE
#419METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
#420Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
#421siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
#422MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION
#423MICRO-RNA FAMILY THAT MODULATES FIBROSIS AND USES THEREOF
#424Primary cell gene editing
#425Compositions and methods for detecting and treating insulin resistance
#426Primary cell gene editing
#427Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
#428Methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
#429Therapeutic splice-switching oligonucleotides
#430COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#431ANTISENSE OLIGONUCLEOTIDES FOR MODULATING HTRA1 EXPRESSION
#432METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#433RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
#434Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
#435Methods and means for treating DNA repeat instability associated genetic disorders
#436Enzymatic replacement therapy and antisense therapy for Pompe disease
#437Compositions and methods for modulating growth hormone receptor expression
#438Functionally-modified oligonucleotides and subunits thereof
#439POLYNUCLEOTIDE AGENTS TARGETING SERPINC1 (AT3) AND METHODS OF USE THEREOF
#440BCL11A guide delivery
#441Compositions for modulating Ataxin 2 expression
#442Serpina1 iRNA compositions and methods of use thereof
#443OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#444Allele specific modulators of P23H rhodopsin
#445Oligonucleotide therapy for Leber Congenital Amaurosis
#446Oligonucleotides for inducing paternal UBE3A expression
#447Compounds and methods for reducing ATXN2 expression
#448MODULATION OF APOLIPOPROTEIN CIII (APOCIII) EXPRESSION
#449Oligonucleotide compositions and methods thereof
#450COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
#451Oligonucleotides for reduction of PD-L1 expression
#452Compositions for modulating SOD-1 expression
#453Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#454Treatment of CNS injury with RNAi therapeutics
#455Methods for treating Alzheimer's disease
#456OLIGONUCLEOTIDES CONTAINING 2'-DEOXY-2'FLUORO-BETA-D-ARABINOSE NUCLEIC ACID (2'-FANA) FOR TREATMENT AND DIAGNOSIS OF RETROVIRAL DISEASES
#457Oligomers and oligomer conjugates
#458MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS
#459Targeted control of pests and pathogens by plant delivery of 2'F-ANA-oligonucleotides
#460Modulation of Factor 11 expression
#461Interfering RNA molecules
#462MODIFIED GUIDE RNAS, METHODS AND USES
#463MODIFIED CRISPR RNA AND USES THEREOF
#464Primary cell gene editing
#465Primary cell gene editing
#466ANTISENSE OLIGONUCLEOTIDES FOR MODULATING NFKB1 EXPRESSION
#467Antisense oligonucleotides for the treatment of Stargardt disease
#468Conjugated antisense compounds and their use
#469Modulators of APOL1 expression
#470Oligonucleotide complexes for use in RNA editing
#471METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
#472METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#473MODULATION OF TMPRSS6 EXPRESSION
#474RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#475ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF
#476Pharmaceutical compositions and methods for potentiating gene silencing
#477COMPOUNDS AND METHODS FOR MODULATION OF TRANSCRIPT PROCESSING
#478Compositions comprising eicosapentaenoic acid and mipomersen and methods of use thereof
#479Modified guide RNAs
#480Oligonucleotides for inducing paternal UBE3A expression
#481Antisense compounds targeting Leucine-Rich repeat kinase 2(LRRK2) for the treatment of Parkinsons disease
#482Antisense oligonucleotides
#483Antiviral agents
#484Compounds and methods for enhanced cellular uptake
#485METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#486METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS
#487MicroRNAs and methods of their use
#488Materials and methods for treatment of hemoglobinopathies
#489Antisense compounds and uses thereof
#490Compositions for modulating C9ORF72 expression
#491TGF-beta oligonucleotide for use in treatment of ophthalmic diseases
#492Antisense oligomers targeting HOXB-AS3 long non-coding RNA
#493Materials and methods for treatment of hemoglobinopathies
#494Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
#495Compositions and methods for treatment of cardiac diseases
#496Modulation of huntingtin expression
#497Compounds and methods for reducing ATXN3 expression
#498Modulators of DNM2 expression
#499Modified double-stranded RNA agents
#500Modified double-stranded RNA agents
#501RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#502Oligonucleotide compounds for targeting huntingtin mRNA
#503MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#504Anti-TNF compounds
#505Natural cryptic exon removal by pairs of antisense oligonucleotides
#506RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
#507Conjugated oligonucleotides
#508Tuning CRISPR/Cas9 activity with chemically modified nucleotide substitutions
#509OLIGOMERIC COMPOUNDS COMPRISING BICYCLIC NUCLEOTIDES AND USES THEREOF
#510Targeted nucleic acid conjugate compositions
#511Targeting MicroRNAs for Metabolic Disorders
#512Compositions and methods for modulating neuronal excitability and motor behavior
#513LNCRNA MEG3 for therapy and diagnosis of cardiac remodelling
#514SINGLE-STRANDED RNAI OLIGONUCLEOTIDES TARGETING APOC-III
#515MIR-29 MIMICS AND USES THEREOF
#516Compositions and methods for inhibiting expression of TMPRSS6 gene
#517Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#518Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
#519Methods and means for efficient skipping of at least one of the following exons of the human Duchenne muscular dystrophy gene: 43, 46, 50-53
#520Defined multi-conjugate oligonucleotides
#521Antisense oligonucleotides for the treatment of Leber congenital amaurosis
#522RNAi agents and compositions for inhibiting expression of angiopoietin-like 3 (ANGPTL3), and methods of use
#523RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
#524Methods of modulating KEAP1
#525Enhancing plasmin activity to prevent soft tissue calcification
#526Modulation of DYRK1B expression
#527Modulation of angiopoietin-like 3 expression
#528RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#529Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#530Multimeric oligonucleotide compounds
#531Methods and compositions for inhibiting PMP22 expression
#532Approach for treating inflammatory disorders
#533COMPOSITIONS AND METHODS FOR MODULATING HBV EXPRESSION
#534Structure-guided chemical modification of guide RNA and its applications
#535Modified RNAi agents
#536RNAi-Mediated Inhibition of Tumor Necrosis Factor Alpha-Related Conditions
#537SYNTHETIC GUIDE RNA FOR CRISPR/CAS ACTIVATOR SYSTEMS
#538Interfering RNA molecules
#539Two-tailed self-delivering siRNA
#540Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#541Methods for modulating Ataxin 2 expression
#542Compositions and methods for modulating angiopoietin-like 3 expression
#543RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
#544RNAi agents for inhibiting expression of alpha-ENaC and methods of use
#545Methods of treating cancer by inhibition of DNA repair proteins using antisense based therapies
#546SELECTIVE REDUCTION OF ALLELIC VARIANTS
#547Polynucleotide agents targeting programmed cell death 1 ligand 1 (PD-L1) and methods of use thereof
#548Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
#549Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
#550Methods for reducing LRRK2 expression
#551RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#552Mobilizing leukemia cells
#553Antisense-induced exon exclusion in myostatin
#554Serpina1 iRNA compositions and methods of use thereof
#555Cancer treatment
#556Compositions and their uses directed to huntingtin
#557COMPOSITIONS AND METHODS FOR MODULATING GROWTH HORMONE RECEPTOR EXPRESSION
#558Compounds and methods for modulating C90RF72
#559Nanoparticle formulations for delivery of nucleic acid complexes
#560Antisense oligonucleotides for the treatment of leber congenital amaurosis
#561MicroRNA Compounds and Methods for Modulating MIR-21 Activity
#562Linkage modified oligomeric compounds
#563Interfering RNA molecules
#564Methods and compositions for the specific inhibition of antithrombin 3 (AT3) by double-stranded RNA
#565Sterol regulatory element binding protein (SREBP) chaperone (SCAP) iRNA compositions and methods of use thereof
#566Compositions and methods for modulating apolipoprotein (a) expression
#567Compositions and methods for modulating apolipoprotein C-III expression
#568MODULATION OF TRANSTHYRETIN EXPRESSION
#569Compositions and their uses directed to huntingtin
#570Oligonucleotide-based inhibitors comprising locked nucleic acid motif
#571Modulation of SMN expression
#572MODULATORS OF KRAS EXPRESSION
#573PDGF and VEGF aptamers having improved stability and their use in treating PDGF and VEGF mediated diseases and disorders
#574Targeting microRNAs for metabolic disorders
#575COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#576RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
#577Notch 1 specific siRNA molecule
#578Methods and compositions for modlating apolipoprotein (A) expression
#579Compositions and methods for inhibiting gene expression of Hif2alpha
#580METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#581Modulation of TMPRSS6 expression
#582Interfering RNA molecules
#583Functionally-modified oligonucleotides and subunits thereof
#584SITE-SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS
#585MicroRNA-328 anti-sense composition and therapeutic use
#586Oligonucleotide compositions and methods thereof
#587OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#588Compositions and methods for inhibiting expression of RRM2 genes
#589Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
#590MODULATION OF TRANSTHYRETIN EXPRESSION
#591MODULATION OF TRANSTHYRETIN EXPRESSION
#592MODULATION OF TRANSTHYRETIN EXPRESSION
#593MODULATION OF TRANSTHYRETIN EXPRESSION
#594MODULATION OF TRANSTHYRETIN EXPRESSION
#595MODULATION OF TRANSTHYRETIN EXPRESSION
#596MODULATION OF TRANSTHYRETIN EXPRESSION
#597MODULATION OF TRANSTHYRETIN EXPRESSION
#598MODULATION OF TRANSTHYRETIN EXPRESSION
#599MODULATION OF TRANSTHYRETIN EXPRESSION
#600CRISPR-BASED COMPOSITIONS AND METHODS OF USE