109191 ⎘
Structure or type of the nucleic acid; Chemical structure; Spatial arrangement of the modifications having a combination of backbone and sugar modifications
Targeting microRNAs for metabolic disorders
#602siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
#603Allele specific modulators of P23H rhodopsin
#604MODULATION OF DYSTROPHIA MYOTONICA-PROTEIN KINASE (DMPK) EXPRESSION
#605Micro-RNA family that modulates extracellular matrix genes and uses thereof
#606Tau antisense oligomers and uses thereof
#607Chemical modification of short small hairpin RNAs for inhibition of gene expression
#608Single-stranded oligonucleotides for use in the medical treatment of skin disorders
#609Modulation of nuclear-retained RNA
#610Targeting microRNAs for the treatment of liver cancer
#611Chemically modified guide RNAs for CRISPR/CAS-mediated gene regulation
#612Stereospecific Phosphorothioate LNA
#613Treatment of genetic disorders associated with DNA repeat instability
#614Therapeutic UNA oligomers and uses thereof
#615Short interfering nucleic acid (siNA) compositions
#616Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
#617Oligonucleotide compounds for targeting huntingtin mRNA
#618Inhibitors of MYH7B and uses thereof
#619Compositions for modulating Tau expression
#620EFFECTS OF APOLIPOPROTEIN B INHIBITION ON GENE EXPRESSION PROFILES IN ANIMALS
#621Polynucleotide agents targeting hydroxyacid oxidase (glycolate oxidase, HAO1) and methods of use thereof
#622Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#623Modulation of huntingtin expression
#624Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
#625Modulation of alpha synuclein expression
#626RNA Modulating Oligonucleotides with Improved Characteristics for the Treatment of Neuromuscular Disorders
#627Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#628Compositions and methods for modulating Tau expression
#629Guide RNA with chemical modifications
#630Compositions and methods for modulating TTR expression
#631RNA interference mediated inhibition of catenin (cadherin-associated protein), beta 1 (CTNNB1) gene expression using short interfering nucleic acid (siNA)
#632Oligonucleotide therapy for leber congenital amaurosis
#633Acyl-amino-LNA and/or hydrocarbyl-amino-LNA oligonucleotides
#634LNA OLIGONUCLEOTIDES WITH ALTERNATING FLANKS
#635Compositions for modulating expression of C9ORF72 antisense transcript
#636Antisense conjugates for decreasing expression of DMPK
#637Methods for the treatment of Leber congenital amaurosis
#638Treatment of facioscapulohumeral dystrophy
#639Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#640Compounds and methods for the modulation of proteins
#641Antisense oligonucleotides for modulating HTRA1 expression
#642Reducing intron retention
#643COMPOSITIONS AND METHODS
#644MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION
#645FULLY STABILIZED ASYMMETRIC SIRNA
#646EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#647REVERSIR TM COMPOUNDS
#648ANTISENSE MODULATION OF APOLIPOPROTEIN B EXPRESSION
#649High specificity genome editing using chemically modified guide RNAs
#650Compositions for modulating C9ORF72 expression
#651ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF LEBER CONGENITAL AMAUROSIS
#652ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF
#653Chiral library screen
#654Antisense-oligonucleotides as inhibitors of TGF-R signaling
#655Anti-TNF compounds
#656Compositions and methods for inhibition of HAO1 (Hydroxyacid Oxidase 1 (Glycolate Oxidase)) gene expression
#657RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#658Modified double-stranded RNA agents
#659Methods of Inducing Exon Skipping
#660MODULATION OF APOLIPOPROTEIN C-III EXPRESSION
#661METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF MYC BY DOUBLE-STRANDED RNA
#662MODULATION OF APOLIPOPROTEIN CIII (APOCIII) EXPRESSION
#663Methods of Inducing Exon Skipping
#664Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#665PDGF and VEGF aptamers having improved stability and their use in treating PDGF and VEGF mediated diseases and disorders
#666Polynucleotide agents targeting Serpinc1 (AT3) and methods of use thereof
#667MicroRNA compounds and methods for modulating miR-21 activity
#668MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS
#669Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions
#670Targeting microRNAs for metabolic disorders
#671Methods and materials for activating an internal ribosome entry site in exon 5 of the DMD gene
#672Multimetric oligonucleotide compounds
#673Polynucleotide agents targeting aminolevulinic acid synthase-1 (ALAS1) and uses thereof
#674Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#675Oligonucleotides for inducing paternal UBE3A expression
#676Antisense compounds
#677Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
#678Compositions and methods for decreasing tau expression
#679Compositions for modulating Ataxin 2 expression
#680Methods for modulating Ataxin 2 expression
#681Compositions and methods for modulating growth hormone receptor expression
#682Compositions and methods for modulating complement factor B expression
#683Methods and compositions for treating a Serpinc1-associated disorder
#684COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#685Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom
#686LIPID FORMULATION
#687Compositions and methods for inhibiting expression of RRM2 genes
#688Antisense compounds targeting leucine-rich repeat kinase 2 (LRRK2) for the treatment of parkinsons disease
#689Targeting microRNAS for the treatment of liver cancer
#690Compositions comprising eicosapentaenoic acid and mipomersen and methods of use thereof
#691Small molecules that enhance the activity of oligonucleotides
#692Methods and compositions for modulating apolipoprotein (a) expression
#693Serpina1 iRNA compositions and methods of use thereof
#694POLYNUCLEOTIDE CONSTRUCTS HAVING BIOREVERSIBLE AND NON-BIOREVERSIBLE GROUPS
#695METHODS AND MEANS FOR EFFICIENT SKIPPING OF EXON 45 IN DUCHENNE MUSCULAR DYSTROPHY PRE-mRNA
#696Compounds and methods for enhanced cellular uptake
#697METHODS AND COMPOSITIONS FOR INCREASING EFFICIENCY OF TARGETED GENE MODIFICATION USING OLIGONUCLEOTIDE-MEDIATED GENE REPAIR
#698Modulation of TMPRSS6 expression
#699Compositions and Methods for Selective Delivery of Oligonucleotide Molecules to Cell Types
#700Targeting microRNAs for metabolic disorders
#701METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#702Polycomb-associated non-coding RNAs
#703CRISPR-based compositions and methods of use
#704CRISPR-based compositions and methods of use
#705METHODS AND MEANS FOR EFFICIENT SKIPPING OF AT LEAST ONE OF THE FOLLOWING EXONS OF THE HUMAN DUCHENNE MUSCULAR DYSTROPHY GENE: 43, 46, 50-53
#706Linkage modified oligomeric compounds
#707Compositions for modulating SOD-1 expression
#708Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#709MODULATION OF FACTOR 11 EXPRESSION
#710RNAi agents modified at the 4′-C position
#711Polycomb-associated non-coding RNAs
#712Polycomb-associated non-coding RNAS
#713Functionally-modified oligonucleotides and subunits thereof
#714RNAi-Mediated Inhibition of Tumor Necrosis Factor Alpha-Related Conditions
#715Compositions and their uses directed to huntingtin
#716Compositions comprising synthetic polynucleotides encoding CRISPR related proteins and synthetic sgRNAs and methods of use
#717Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#718Methods and compositions for modulating alpha-1 antitrypsin expression
#719Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
#720RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
#721miR-29 mimics and uses thereof
#722Polycomb-associated non-coding RNAs
#723Modulation of Exon Recognition in Pre-mRNA By Interfering with the Secondary RNA Structure
#724Oligonucleotide compounds for targeting huntingtin mRNA
#725Polycomb-associated non-coding RNAs
#726Double stranded oligonucleotide compounds comprising positional modifications
#727Compositions and methods for inhibiting gene expression of Hif2alpha
#728Compounds of chemically modified oligonucleotides and methods of use thereof
#729Modulation of signal transducer and activator of transcription 3 (STAT3) expression
#730MODULATION OF TRANSTHYRETIN EXPRESSION
#731Oligonucleotide-based inhibitors comprising locked nucleic acid motif
#732Modulation of huntingtin expression
#733METHODS FOR TREATMENT OF WOUND HEALING UTILIZING CHEMICALLY MODIFIED OLIGONUCLEOTIDES
#734MEANS AND METHODS FOR COUNTERACTING MUSCLE DISORDERS
#735Complement component iRNA compositions and methods of use thereof
#736Guide RNA with chemical modifications
#737METHODS AND COMPOSITIONS FOR THE SPECIFIC INHIBITION OF MYC BY DOUBLE-STRANDED RNA
#738Compositions and methods for inhibiting JC virus (JCV)
#739RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#740Identification of structurally similar small molecules that enhance therapeutic exon skipping
#741MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#742Method for reducing circulating leukocytes in a human subject
#743Reducing intron retention
#744RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#745Compositions and methods for modulating neuronal excitability and motor behavior
#746MicroRNA compounds and methods for modulating miR-21 activity
#747Modulation of apolipoprotein C-III expression
#748Tau antisense oligomers and uses thereof
#749Notch 1 specific siRNA molecule
#750Targeting microRNAs for metabolic disorders
#751Compositions and methods for modulating angiopoietin-like 3 expression
#752Modified nucleosides, analogs thereof and oligomeric compounds prepared therefrom
#753PDGF and VEGF aptamers having improved stability and their use in treating PDGF and VEGF mediated diseases and disorders
#754CRISPR-based compositions and methods of use
#755Compositions and methods for inhibiting expression of TMPRSS6 gene
#756Compositions for modulating Tau expression
#757Micro-RNA family that modulates fibrosis and uses thereof
#758Micro-RNA family that modulates fibrosis and uses thereof
#759Micro-RNA family that modulates fibrosis and uses thereof
#760Antisense-oligonucleotides as inhibitors of TGF-R signaling
#761Interfering RNA molecules
#762COMPOSITIONS AND METHODS FOR MODULATION OF TARGET NUCLEIC ACIDS
#763Compositions and methods for inhibiting expression of the HAMP gene
#764Antisense compounds and uses thereof
#765Compositions and methods for modulating apolipoprotein (a) expression
#766Compositions and methods for modulating apolipoprotein C-III expression
#767Compositions and methods
#768Phosphorous-linked oligomeric compounds and their use in gene modulation
#769Compositions and methods for modulating HBV expression
#770siRNA Therapy for Transthyretin (TTR) Related Ocular Amyloidosis
#771Compositions and methods for modulation nucleic acids through nonsense mediated decay
#772Modulation of androgen receptor expression
#773Modulation of dystrophia myotonica-protein kinase (DMPK) expression
#774miR-29 mimics and uses thereof
#775RNAI MODULATION OF SCAP AND THERAPEUTIC USES THEREOF
#776MODULATION OF APOLIPOPROTEIN CIII (APOCIII) EXPRESSION
#777RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#778Method for efficient exon (44) skipping in duchenne muscular dystrophy and associated means
#779COMPOSITIONS AND THEIR USES DIRECTED TO HUNTINGTIN
#780Treatment of genetic disorders associated with DNA repeat instability
#781Structure and use of 5′ phosphate oligonucleotides
#782Modulation of hepatitis B virus (HBV) expression
#783Targeting microRNAs for the treatment of liver cancer
#784Targeting microRNAs for metabolic disorders
#785Methods for enhancing utrophin production via inhibition of microRNA
#786Inhibitors of MYH7B and uses thereof
#787Compositions and methods for modulating Tau expression
#788Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#789DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION
#790Reducing intron retention
#791Oligomers and oligomer conjugates
#792Bridged bicyclic nucleosides
#793Cancer treatment
#794Antisense oligonucleotides directed against connective tissue growth factor and uses thereof
#795Modulation of apolipoprotein C-III (ApoCIII) expression in lipoprotein lipase deficient (LPLD) populations
#796OLIGONUCLEOTIDES COMPRISING ALTERNATING SEGMENTS AND USES THEREOF
#797Inhibitors of miRNAs 221 and 222 for anti-tumor activity in multiple myeloma
#798Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#799SELECTIVE REDUCTION OF ALLELIC VARIANTS
#800Oligomeric compounds and compositions for the use in modulation of micrornas
#801Antisense modulation of PTP1B expression
#802Methods of delivering multiple targeting oligonucleotides to a cell using cleavable linkers
#803Multimeric oligonucleotides compounds having cleavable linkers
#804DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION
#805Compositions and methods for selective delivery of oligonucleotide molecules to cell types
#806Compositions and their uses directed to huntingtin
#807Multimeric oligonucleotides compounds
#808Antisense compounds targeting genes associated with fibronectin
#809Oligonucleotide complex compositions and methods of use as gene alteration tools
#810Methods for modulating Tau expression for reducing seizure and modifying a neurodegenerative syndrome
#811Oligomeric compounds comprising bicyclic nucleosides and uses thereof
#812Compositions for modulating C9ORF72 expression
#813Short interfering ribonucleic acid (siRNA) for oral administration
#814MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#815MicroRNA compounds and methods for modulating miR-21 activity
#816COMPOSITIONS AND METHODS FOR MODULATING APOA1 AND ABCA1 EXPRESSION
#817ANTISENSE MODULATION OF FIBROBLAST GROWTH FACTOR RECEPTOR 4 EXPRESSION
#818MODULATION OF APOLIPOPROTEIN (a) EXPRESSION
#819Compositions and methods for modulating HBV expression
#820Antisense modulation of superoxide dismutase 1, soluble expression
#821Lipid formulations
#822Modulation of huntingtin expression
#823Chemical modification of short small hairpin RNAs for inhibition of gene expression
#824RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
#825Antisense modulation of GCCR expression
#826Interfering RNA molecules
#827Methods and compositions for modulating alpha-1-antitrypsin expression
#828Antisense conjugates for decreasing expression of DMPK
#829Targeting MicroRNAs for metabolic disorders
#830METHODS AND COMPOSITIONS FOR MODULATING FACTOR VII EXPRESSION
#831Methods for modulating metastasis-associated-in-lung-adenocarcinoma-transcript-1 (MALAT-1) expression
#832Modulation of hepatitis B virus (HBV) expression
#833Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#834Compositions and methods for prognosis and treatment of prostate cancer
#835Antisense oligonucleotides for the treatment of Leber congenital amaurosis
#836Modulation of signal transducer and activator of transcription 3 (STAT3) expression
#837Oligomeric compounds and compositions for use in modulation of small non-coding RNAS
#838RNAi agents, compositions and methods of use thereof for treating transthyretin (TTR) associated diseases
#839Modulation of TMPRSS6 expression
#840Complement antagonists and uses thereof
#841Compositions and methods for inhibiting expression of the HAMP gene
#842Modified RNAi agents
#843Exon skipping therapy for dystrophic epidermolysis bullosa
#844Anti-clusterin monotherapy for cancer treatment
#845Custirsen treatment with reduced toxicity
#846Site specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#847Compositions comprising eicosapentaenoic acid and mipomersen and methods of use thereof
#848Clusterin antisense therapy for treatment of cancer
#849Antisense modulation of PTP1B expression
#850MicroRNA inhibitors
#851Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#852Targeting microRNAs for the treatment of liver cancer
#853Compositions and methods for inhibiting expression of TMPRSS6 gene
#854Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#855Antisense compounds targeting genes associated with usher syndrome
#856Method of treating cancer by inhibition of DNA repair proteins
#857Oligomeric compounds comprising bicyclic nucleotides and uses thereof
#858Modulation of hepatitis B virus (HBV) expression
#859TRPM-2 antisense therapy
#860Antisense modulation of fibroblast growth factor receptor 4 expression
#861Oligomeric compounds and compositions for use in modulation of small non-coding RNAS
#862Antimir-451 for the treatment of polycythemias
#863Chimeric oligomeric compounds for modulation of splicing
#864Modulation of alpha synuclein expression
#865Compositions and methods for characterizing and treating muscular dystrophy
#866Inhibitors of the miR-15 family of micro-RNAs
#867Oligonucleotide-based inhibitors comprising locked nucleic acid motif
#868Methods for modulating factor 12 expression
#869Modulation of apolipoprotein C-III expression
#870Modified iRNA agents
#871MicroRNA compounds and methods for modulating miR-21 activity
#872Compositions and their uses directed to huntingtin
#873Antisense modulation of fibroblast growth factor receptor 4 expression
#874Micro-RNA family that modulates fibrosis and uses thereof
#875Micro-RNA family that modulates fibrosis and uses thereof
#876Short interfering ribonucleic acid (siRNA) for oral administration
#877RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#878Oligonucleotides comprising alternating segments and uses thereof
#879Micro-RNA family that modulates fibrosis and uses thereof
#880Compositions and their uses directed to huntingtin
#881Selective inhibition of polyglutamine protein expression
#882Antisense modulation of GCCR expression
#883MicroRNA as a cancer progression predictor and its use for treating cancer
#884Interfering RNA molecules
#885Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#886METHODS AND COMPOSITIONS USEFUL IN TREATMENT OF DISEASES OR CONDITIONS RELATED TO REPEAT EXPANSION
#887MODULATION OF GLUCOSE-6-PHOSPHATASE TRANSLOCASE EXPRESSION
#888Modulation of growth hormone receptor expression and insulin-like growth factor expression
#889Selective reduction of allelic variants
#890miR-150 for the treatment of blood disorders
#891DOUBLE STRAND COMPOSITIONS COMPRISING DIFFERENTIALLY MODIFIED STRANDS FOR USE IN GENE MODULATION
#892Modulation of Hepatitis B virus (HBV) expression
#893Modulation of forkhead box O1A expression
#894DOSING REGIMENS FOR TREATING AND PREVENTING OCULAR DISORDERS USING C-RAF ANTISENSE
#895Compositions and methods for inhibiting expression of Eg5 and VEGF genes
#896RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#897Duplex oligonucleotide complexes and methods for gene silencing by RNA interference
#898TRPM-2 antisense therapy
#899Compositions and methods for inhibiting expression of the HAMP gene
#900MODULATION OF APOLIPOPROTEIN(A) EXPRESSION