109162 ⎘
Structure or type of the nucleic acid; Chemical structure of the backbone Phosphorothioates
Liposomal spherical nucleic acid (SNA) constructs presenting antisense oligonucleotides (ASO) for specific knockdown of interleukin 17 receptor mRNA
#1802PRIMARY CELL GENE EDITING
#1803Xanthine dehydrogenase (XDH) IRNA compositions and methods of use thereof
#1804Oligonucleotide compositions and methods thereof
#1805RNAi agents for inhibiting expression of alpha-ENaC and methods of use
#1806RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
#1807MODIFIED CPF1 GUIDE RNA
#1808Compositions and methods for inhibiting HMGB1 expression
#1809TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF
#1810PREVENTIVE OR THERAPEUTIC AGENT FOR FIBROSIS
#1811HEPATITIS D VIRUS (HDV) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#1812Oligonucleotide comprising an inosine for treating DMD
#1813Gapmers and methods of using the same for the treatment of muscular dystrophy
#1814Exosomal loading using hydrophobically modified oligonucleotides
#1815Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#1816Methods and compositions for inhibiting expression of LDHA
#1817Antisense oligonucleotides for inducing exon skipping and methods of use thereof
#1818Compositions and Methods for Inhibiting Expression of Transthyretin
#1819Methods for Treatment of Alport Syndrome
#1820NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#1821Compositions and methods for inhibiting expression of RRM2 genes
#1822Modulators of MALAT1 expression
#1823Insulin-like growth factor binding protein, acid labile subunit (IGFALS) and insulin-like growth factor 1 (IGF-1) iRNA compositions and methods of use thereof
#1824Peptide compositions and methods of use thereof
#1825METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
#18262'-METHOXY SUBSTITUTED OLIGOMERIC COMPOUNDS AND COMPOSITIONS FOR USE IN GENE MODULATIONS
#1827ANTISENSE COMPOUNDS
#1828Single-Stranded RNAI Oligonucleotides Targeting APOC-III
#1829Modulation of huntingtin expression
#1830Dynamic pharmacokinetic-modifying anchors
#1831RNA-modulating agents
#1832GLP-1 RECEPTOR LIGAND MOIETY CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#1833Amphiphilic oligodeoxynucleotide conjugates as adjuvant enhancers
#1834Complement component iRNA compositions and methods of use thereof
#1835COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF LPA
#1836Compositions and Methods for Inhibition of Expression of Apolipoprotein C-III (APOC3) Genes
#1837CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING BETA HEMOGLOBINOPATHIES
#1838SPHERICAL NUCLEIC ACID TLR9 AGONISTS
#1839NUCLEIC ACID APTAMER FOR INHIBITING ACTIVITY OF GENOME-EDITING ENZYME
#1840Therapeutic compositions
#1841Stabilized HNF4A saRNA compositions and methods of use
#1842HNF4A saRNA compositions and methods of use
#1843Methods and compositions using RNA interference and antisense oligonucleotides for inhibition of KRAS
#1844TLR9-TARGETED SPHERICAL NUCLEIC ACIDS HAVING POTENT ANTITUMOR ACTIVITY
#1845Oligonucleotides for reduction of PD-L1 expression
#1846Agents that modulate TMEM230 as angiogenesis regulators and that detect TMEM230 as markers of metastasis
#1847Therapy
#1848Compositions and methods for inhibition of expression of protein C (PROC) genes
#1849Compositions and methods for modulation of protein aggregation
#1850Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA
#1851Compositions for modulating expression of C9ORF72 antisense transcript
#1852COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
#1853Morpholino modified oligomeric compounds
#1854Oligonucleotides to treat eye disease
#1855Methods for treatment of polycystic kidney disease
#1856Reducing intron retention
#1857Double-stranded RNA molecule targeting CKIP-1 and use thereof
#1858Agents useful in treating facioscapulohumeral muscular dystrophy
#1859Nucleic acid oligomers and uses therefor
#1860ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES, THE HCHWA-D MUTATION, AND TRINUCLEOTIDE REPEAT EXPANSIONS
#1861Immunosuppression-reverting oligonucleotides inhibiting the expression of CD39
#1862Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#1863COMPOSITIONS AND METHODS FOR MODULATING HBV EXPRESSION
#1864Antisense Compounds and Uses Thereof
#1865ANTISENSE OLIGONUCLEOTIDES FOR MODULATING RELA EXPRESSION
#1866SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#1867CHEMICALLY MODIFIED OLIGONUCLEOTIDES
#1868Products and compositions
#1869ALLELE SELECTIVE INHIBITION OF MUTANT C9ORF72 FOCI EXPRESSION BY DUPLEX RNAS TARGETING THE EXPANDED HEXANUCLEOTIDE REPEAT
#1870STABILIZED SARNA COMPOSITIONS AND METHODS OF USE
#1871METHODS FOR TREATING OR PREVENTING CONTACT-ACTIVATION PATHWAY-ASSOCIATED DISEASES USING iRNA COMPOSITIONS TARGETING FACTOR XII (HAGEMAN FACTOR) (F12)
#1872Alpha-1 antitrypsin (AAT) RNAi agents, compositions including AAT RNAi agents, and methods of use
#1873Modulators of IRF5 expression
#1874GENOME-EDITED NK CELL AND METHODS OF MAKING AND USING
#1875RNA interference mediated inhibition of hepatitis B virus (HBV) gene expression using short interfering nucleic acid (SINA)
#1876Allele-specific therapy for Huntington disease haplotypes
#1877Apolipoprotein C3 (APOC3) iRNA compositions and methods of use thereof
#1878Antisense oligonucleotides for inducing exon skipping and methods of use thereof
#1879Compounds and methods for reducing Tau expression
#1880Targeted RNA editing
#1881REFILLABLE DRUG DELIVERY DEVICES AND METHODS OF USE THEREOF
#1882Modulators of PCSK9 expression
#1883Methods and compositions for treating cancers and enhancing therapeutic immunity by selectively reducing immunomodulatory M2 monocytes
#1884PKLR inhibition in the treatment of NAFLD and HCC
#1885COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
#1886MODULATION OF HEPATITIS B VIRUS (HBV) EXPRESSION
#1887Organic compositions to treat EPAS1-related diseases
#1888Oligonucleotide compositions and methods of use thereof
#1889Nucleic acid molecules and their use in therapy
#1890MICRO-RNA FAMILY THAT MODULATES FIBROSIS AND USES THEREOF
#1891Primary cell gene editing
#1892Antisense oligonucleotides (ODN) against SMAD7 and uses thereof in medical field
#1893COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#1894Modulators of FOXP3 expression
#1895Modified Cpf1 MRNA, modified guide RNA, and uses thereof
#1896Ketohexokinase (KHK) iRNA compositions and methods of use thereof
#1897ENA ANTISENSE OLIGONUCLEOTIDE FOR INHIBITION OF alpha-SYNUCLEIN EXPRESSION
#1898Compositions and methods for detecting and treating insulin resistance
#1899Methods for treating hepatitis B infection
#1900Primary cell gene editing
#1901Oligonucleotide compounds for treatment of preeclampsia and other angiogenic disorders
#1902Modified Oligonucleotides for Treatment of Polycystic Kidney Disease
#1903Methods for cancer treatment
#1904Methods and compositions for inducing protective immunity
#1905RNAi inhibition of alpha-ENaC expression
#1906COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#1907ANTISENSE OLIGONUCLEOTIDES FOR MODULATING HTRA1 EXPRESSION
#1908Oligonucleotide compositions and methods of use thereof
#1909Modulating RNA Interactions with Polycomb Repressive Complex 1 (PRC1)
#1910METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#1911Compositions and methods for inhibiting TIGIT gene expression
#1912MODULATORS OF COMPLEMENT FACTOR B
#1913RNA modulating oligonucleotides with improved characteristics for the treatment of neuromuscular disorders
#1914Antisense nucleic acids
#1915Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
#1916Methods and compositions for treating a proprotein convertase subtilisin kexin (PCSK9) gene-associated disorder
#1917Modulators of PNPLA3 expression
#1918Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#1919Hepatitis B virus (HBV) iRNA compositions and methods of use thereof
#1920COMPOUNDS THAT TARGET MYC microRNA RESPONSIVE ELEMENTS FOR THE TREATMENT OF MYC-ASSOCIATED CANCER
#1921Methods and means for treating DNA repeat instability associated genetic disorders
#1922Myostatin iRNA compositions and methods of use thereof
#1923Enzymatic replacement therapy and antisense therapy for Pompe disease
#1924Treatment of Sirtuin (SIRT) related diseases by inhibition of natural antisense transcript to a Sirtuin (SIRT)
#1925Molecules and agents for treating hepatitis B virus
#1926Compositions and methods for modulating growth hormone receptor expression
#1927TREATMENT OF ANGIOGENESIS-ASSOCIATED DISEASES USING RNA COMPLEXES THAT TARGET ANGPT2 AND PDGFB
#1928NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND USES THEREOF
#1929Modulation of angiopoietin-like 3 expression
#1930HETERO DOUBLE-STRANDED antimiR
#1931Antisense nucleic acids
#1932Treatment of atopic dermatitis and asthma using RNA complexes that target IL4Rα, TRPA1, or F2RL1
#1933Methods of reducing expression of x-inactivation escapee genes and autosomal genes
#1934Antisense nucleic acids
#1935POLYNUCLEOTIDE AGENTS TARGETING SERPINC1 (AT3) AND METHODS OF USE THEREOF
#1936Antisense nucleic acids
#1937Methods and compositions for managing vascular conditions
#1938BCL11A guide delivery
#1939MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPODYSTROPHY POPULATIONS
#1940O-methyl rich fully stabilized oligonucleotides
#1941Inhibition of poly(A) binding protein and the treatment of pain
#1942Compositions for modulating Ataxin 2 expression
#1943NUCLEIC ACIDS COMPRISING FORMULA (NuGlXmGnNv)a AND DERIVATIVES THEREOF AS IMMUNOSTIMULATING AGENT/ADJUVANT
#1944APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#1945Serpina1 iRNA compositions and methods of use thereof
#1946OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#1947Therapeutic drug for Alport's syndrome
#1948Therapeutic agent for treating cancer comprising anti-miRNA-albumin composite
#1949Compositions of asymmetric interfering RNA and uses thereof
#1950COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#1951Allele specific modulators of P23H rhodopsin
#1952MicroRNA (miRNA) and Downstream Targets for Diagnostic and Therapeutic Purposes
#1953Anti-connexin compounds targeted to connexins and methods of use thereof
#1954Oligonucleotide therapy for Leber Congenital Amaurosis
#1955Products and compositions
#1956Compositions of asymmetric interfering RNA and uses thereof
#1957Oligonucleotides for inducing paternal UBE3A expression
#1958SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
#1959COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION
#1960Post-selex modification methods
#1961Compounds and methods for reducing ATXN2 expression
#1962MODULATION OF APOLIPOPROTEIN CIII (APOCIII) EXPRESSION
#1963COMPOSITIONS AND METHODS FOR INHIBITIION OF EXPRESSION OF PROTEIN C (PROC) GENES
#1964Oligonucleotide compositions and methods thereof
#1965Modified nucleic acids, hybrid guide RNAs, and uses thereof
#1966Compositions and methods for gene editing in T cells using CRISPR/Cpf1
#1967COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
#1968Oligonucleotides for reduction of PD-L1 expression
#1969Compositions and methods for the treatment of hypercholesterolemia
#1970Compositions for modulating SOD-1 expression
#1971Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#1972Compositions and methods for modulating SMN gene family expression
#1973Treatment of CNS injury with RNAi therapeutics
#1974METHODS FOR DOSING AND MONITORING SMAD7 ANTISENSE OLIGONUCLEOTIDE TREATMENT USING BIOMARKER LEVELS
#1975Compositions and methods for modulation of IKBKAP splicing
#1976Complement component C5 iRNA compositions and methods of use thereof
#1977COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#1978Methods for treating Alzheimer's disease
#1979OLIGONUCLEOTIDES CONTAINING 2'-DEOXY-2'FLUORO-BETA-D-ARABINOSE NUCLEIC ACID (2'-FANA) FOR TREATMENT AND DIAGNOSIS OF RETROVIRAL DISEASES
#1980Oligomers and oligomer conjugates
#1981Oligonucleotides for modulating ATXN2 expression
#1982Targeting human satellite II (HSATII)
#1983MODULATION OF APOLIPOPROTEIN C-III (APOCIII) EXPRESSION IN LIPOPROTEIN LIPASE DEFICIENT (LPLD) POPULATIONS
#1984Oligonucleotides for modulating Tau expression
#1985Antisense oligonucleotides for inhibition of PD-L1 expression and treating cancer
#1986Reduced size self-delivering RNAI compounds
#1987Modulation of Factor 11 expression
#1988OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#1989MODIFIED GUIDE RNAS, METHODS AND USES
#1990Methods and systems for guide RNA design and use
#1991Antisense oligonucleotides for use in treating Alzheimer's disease
#1992Chemically-modified siRNA
#1993MODIFIED CRISPR RNA AND USES THEREOF
#1994Oligonucleotides to treat eye disease
#1995Anti-IL-17 aptamer having improved retention in blood
#1996Primary cell gene editing
#1997Primary cell gene editing
#1998Antisense Compositions and Methods of Making and Using Same
#1999Treatment of idiopathic pulmonary fibrosis using RNA complexes that target connective tissue growth factor
#2000Antisense oligonucleotides that inhibit influenza virus replication and uses thereof
#2001ANTISENSE OLIGONUCLEOTIDES FOR MODULATING NFKB1 EXPRESSION
#2002Antisense oligonucleotides for the treatment of Stargardt disease
#2003Compositions for modulating C9ORF72 expression
#2004Conjugated antisense compounds and their use
#2005OLIGONUCLEOTIDES FOR MODULATING ATXN2 EXPRESSION
#2006Compositions and methods for inhibiting gene expression of factor XII
#2007Modulators of APOL1 expression
#2008OLIGONUCLEOTIDE COMPOSITIONS AND USES THEREOF
#2009Oligomeric compounds comprising α-β-constrained nucleic acid
#2010Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#2011Oligonucleotide complexes for use in RNA editing
#2012Sterile formulation comprising a stable phosphorothioate oligonucleotide
#2013METHODS FOR TREATING OR PREVENTING TTR-ASSOCIATED DISEASES USING TRANSTHYRETIN (TTR) iRNA COMPOSITIONS
#2014Modified iRNA agents
#2015ANTISENSE OLIGONUCLEOTIDES FOR MODULATING RELB EXPRESSION
#2016ANTISENSE OLIGONUCLEOTIDES FOR MODULATING NFKB2 EXPRESSION
#2017Transthyretin (TTR) iRNA compositions and methods of use thereof for treating or preventing TTR-associated diseases
#2018Bicyclic nucleosides and oligomers prepared therefrom
#2019Modulation of Lnc05 expression
#2020MODULATION OF TMPRSS6 EXPRESSION
#2021ANTISENSE OLIGONUCLEOTIDES FOR MODULATING REL EXPRESSION
#2022METHODS AND COMPOSITIONS FOR TREATING A SUBJECT WITH A SMAD7 ANTISENSE OLIGONUCLEOTIDE
#2023SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
#2024RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#2025Method for generating aptamers with improved off-rates
#2026Endosomal cleavable linkers
#2027Methods for intracellular delivery and enhanced gene targeting
#2028COMPOUNDS AND METHODS FOR USE IN DYSTROPHIN TRANSCRIPT
#2029Therapeutic inhibition of lactate dehydrogenase and agents therefor
#2030ANTISENSE OLIGONUCLEOTIDES DIRECTED AGAINST CONNECTIVE TISSUE GROWTH FACTOR AND USES THEREOF
#2031Antisense oligonucleotides for inducing exon skipping and methods of use thereof
#2032Piston arrangement
#2033COMPOUNDS AND METHODS FOR MODULATION OF SMN2
#2034Pharmaceutical compositions and methods for potentiating gene silencing
#2035COMPOUNDS AND METHODS FOR MODULATION OF TRANSCRIPT PROCESSING
#2036Oligonucleotide compositions with enhanced efficiency
#2037Angiopoietin-like 3 (ANGPTL3) iRNA compositions and methods of use thereof
#2038Serpina1 iRNA compositions and methods of use thereof
#2039Compositions comprising eicosapentaenoic acid and mipomersen and methods of use thereof
#2040Modified guide RNAs
#2041Antisense nucleic acids
#2042Oligonucleotides for inducing paternal UBE3A expression
#2043RNAi constructs for inhibiting ASGR1 expression and methods of use thereof
#2044Antisense compounds targeting Leucine-Rich repeat kinase 2(LRRK2) for the treatment of Parkinsons disease
#2045Treatment of tumor suppressor gene related diseases by inhibition of natural antisense transcript to the gene
#2046Antisense oligonucleotides
#2047Treatment of collagen gene related diseases by inhibition of natural antisense transcript to a collagen gene
#2048Compositions and Methods for Inhibiting Expression of Transthyretin
#2049Compounds and methods for enhanced cellular uptake
#2050CCCTC-binding factor (CTCF) RNA interactome
#2051Soluble TNF Receptors and Their Use in Treatment of Disease
#2052Methods for enhancing utrophin production via inhibition of microRNA
#2053METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#2054Compositions and methods of treating muscle atrophy and myotonic dystrophy
#2055RNAi agents for Hepatitis B virus infection
#2056METHOD FOR EFFICIENT EXON (44) SKIPPING IN DUCHENNE MUSCULAR DYSTROPHY AND ASSOCIATED MEANS
#2057SPINAL MUSCULAR ATROPHY (SMA) TREATMENT VIA TARGETING OF SMN2 SPLICE SITE INHIBITORY SEQUENCES
#2058Compositions and methods for inhibiting gene expression of alpha-1 AntiTrypsin
#2059DOSAGES AND METHODS FOR DELIVERING LIPID FORMULATED NUCLEIC ACID MOLECULES
#2060MicroRNAs and methods of their use
#2061Materials and methods for treatment of hemoglobinopathies
#2062SELECTIVE LOCAL INHIBITION OF TNFR1-MEDIATED FUNCTIONS AT THE SITE OF ANTIGEN/ALLERGEN PRESENTATION
#2063Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression
#2064Immunostimulatory sequence oligonucleotides and methods of using the same
#2065SELECTIVE ANTISENSE COMPOUNDS AND USES THEREOF
#2066CAMK2D antisense oligonucleotides and uses thereof
#2067FHSS hotspot device and methods
#2068Antisense molecules and methods for treating pathologies
#2069Therapeutic targeting of a microRNA to treat Duchenne muscular dystrophy
#2070Compositions for delivery of cargo to cells
#2071Antisense compounds and uses thereof
#2072Methods and compositions for adaptive immune modulation
#2073COMPOSITIONS FOR DELIVERY OF CARGO TO CELLS
#2074Modulation of gene expression and screening for deregulated protein expression
#2075Method for Generating Aptamers with Improved Off-Rates
#2076Therapeutic compositions
#2077Compositions for modulating C9ORF72 expression
#2078GLYCOCONJUGATES OF RNA INTERFERENCE AGENTS
#2079TGF-beta oligonucleotide for use in treatment of ophthalmic diseases
#2080Targeting ligands
#2081Antisense oligomers targeting HOXB-AS3 long non-coding RNA
#2082COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
#2083Materials and methods for treatment of hemoglobinopathies
#2084Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping
#2085Compositions and methods for treatment of cardiac diseases
#2086Modulation of huntingtin expression
#2087METHODS AND COMPOSITIONS OF BIOLOGICALLY ACTIVE AGENTS
#2088Compounds and methods for reducing ATXN3 expression
#2089Engineered guide RNA and uses thereof
#2090Modulators of DNM2 expression
#2091MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#2092Modulation of prekallikrein (PKK) expression
#2093RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#2094MODULATORS OF GROWTH HORMONE RECEPTOR
#2095SPHERICAL NUCLEIC ACID TLR9 AGONISTS
#2096Oligonucleotide compounds for targeting huntingtin mRNA
#2097MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#2098Nucleic acid conjugates and uses thereof
#2099INHIBITION OF PCSK9 THROUGH RNAI
#2100Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof