109162 ⎘
Structure or type of the nucleic acid; Chemical structure of the backbone Phosphorothioates
METHODS AND COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASES AND DISORDERS AND CANCERS
#2MICROEXON TARGETING FOR MODULATION OF RYR2 CHANNEL ACTIVITY
#3ANTI-CANCER TGFB2 AGENTS WITH IMMUNOTHERAPEUTICS
#4CORONAVIRUS IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#5OLIGONUCLEOTIDE-MEDIATED KNOCKDOWN OF PLN
#6OLIGONUCLEOTIDE-MEDIATED KNOCKDOWN OF DUX4
#7OLIGONUCLEOTIDES
#8RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#9COMPOSITIONS AND METHODS FOR EMR2 MODIFICATION
#10Compositions and Methods for Treating and Preventing Prekallikrein-Associated Conditions
#11Compositions for Modulating C9ORF72 Expression
#12APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#13METHOD FOR USING GUIDE RNAS WITH CHEMICAL MODIFICATIONS
#14COMPOSITIONS AND METHODS FOR THE TARGETING OF LPA
#15METHODS FOR TREATING HEPATITIS B INFECTION
#16SMALL RNA-BASED PROGNOSTIC SIGNATURES AND THERAPEUTIC COMPOSITIONS FOR IDIOPATHIC PULMONARY FIBROSIS
#17METHODS FOR REPROGRAMMING AND GENE EDITING CELLS
#18Enhancing Oligonucleotide Immunomodulatory Activity through Dianophore Long-Lasting Modification: Methods and Applications
#19COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#20DOUBLE STRANDED RNAi AGENTS, COMPOSITIONS AND METHODS OF USE
#21ANTISENSE OLIGONUCLEOTIDES FOR TREATING A DISEASE OR CONDITION ASSOCIATED WITH AN ABNORMAL PROCESSING OF APP
#22GENE EDITING SYSTEMS AND USES THEREOF
#23RNAi Agent Targeting PCSK9 and Use Thereof
#24INTERFERING RNA FOR INHIBITING TOP1 GENE EXPRESSION AND USE THEREOF
#25ANTI-CANCER TGFB2 AGENTS WITH IMMUNOTHERAPEUTICS
#26RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use
#27COMPOSITIONS AND METHODS FOR DELIVERING A MACROMOLECULE TO A CELL
#28Modulation of Apolipoprotein C-III (ApoCIII) Expression in Lipoprotein Lipase Deficient (LPLD) Populations
#29MODIFIED OLIGONUCLEOTIDES AND DOUBLE-STRANDED RNAS
#30ANTISENSE OLIGONUCLEOTIDES FOR TARGETING PROGRANULIN
#31Compounds and Methods for Modulating UBE3A-ATS
#32RNAi AGENTS FOR MODULATING SNCA
#33STABILIZED RNA AGENTS
#34NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#35DOUBLE-STRANDED RNAi AGENTS FOR TARGETING AND REGULATING HBV GENE EXPRESSION, AND A USE THEREOF
#36OLIGONUCLEOTIDES CONJUGATED TO OLEIC ACID AND USES THEREOF
#37RUNX1 MODULATORS
#38ANTISENSE OLIGONUCLEOTIDE, COMPOSITIONS AND PHARMACEUTICAL FORMULATIONS THEREOF FOR EXON SKIPPING
#39COMPOSITIONS AND METHODS FOR TREATING PRIMARY CILIARY DYSKINESIA
#40Prime Editing Systems having pegRNA with Reduced Auto-inhibitory Interaction
#41Compounds and Methods for Reducing ATXN3 Expression
#42COMPOSITIONS AND METHODS TO MODULATE TUMOR MICROENVIRONMENT ASSOCIATED LONG NONCODING RNAS
#43OLIGONUCLEOTIDES FOR MAPT MODULATION
#44IMMUNOMODULATING POLYNUCLEOTIDES, ANTIBODY CONJUGATES THEREOF, AND METHODS OF THEIR USE
#45Methods for Preventing Cardiovascular Events Through Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Reduction
#46POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#47ANTI-FIBROTIC MICRORNA COMPOSITION
#48Antisense oligonucleotides for treatment of USHER 2A. Exon 68
#49siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS
#50COMPOSITIONS AND METHODS FOR TREATMENT OF NEUROINFLAMMATORY DISEASES
#51INDUCTION OF STEM-LIKE ACTIVATED T CELLS
#52ANTI-C5 ANTIBODY/C5 IRNA DOSING REGIMENS FOR TREATING C5-ASSOCIATED DISEASES
#53DNA-RNA HETERODUPLEX TARGETING ANDROGEN RECEPTOR
#54METHODS FOR TREATMENT OF ANEMIA
#55NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#56DOUBLE-STRANDED RNAI AGENTS AND COMPOSITIONS FOR REDUCING EXPRESSION OF ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND METHODS OF USE THEREOF
#57PRODUCTS AND COMPOSITIONS
#58TREATMENT OF THYMIC STROMAL LYMPHOPOIETIN (TSLP) RELATED DISEASES BY INHIBITION OF LONG-FORM TSLP TRANSCRIPTS
#59THERAPEUTIC AGENTS FOR METABOLISM-ASSOCIATED DISEASES
#60GALNAC MONOMER COMPRISING RIBOSE RING OR DERIVATIVE STRUCTURE THEREOF AND USE THEREOF IN LIVER-TARGETED DELIVERY OF SMALL NUCLEIC ACID DRUGS
#61SIRNA FOR TARGETED INHIBITION OF AGT GENE EXPRESSION AND USE THEREOF IN TREATING HYPERTENSION
#62POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF CANCER
#63CONJUGATED OLIGONUCLEOTIDE COMPOUNDS, METHODS OF MAKING AND USES THEREOF
#64COMPOUNDS AND METHODS FOR MODULATING SMN2
#65G-QUADRUPLEX-CONTAINING OLIGONUCLEOTIDES
#66Use of a Janus Kinase Inhibitor and a Telomerase Inhibitor for the Treatment of Myeloproliferative Neoplasms
#67Cyclic Antisense Therapeutics
#68NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF LPA IN A CELL
#69DSRNA, USE THEREOF AND PREPARATION METHOD THEREFOR
#70OLIGONUCLEOTIDES, COMPOSITIONS AND METHODS THEREOF
#71SPLICE-SWITCHING OLIGONUCLEOTIDES TARGETING IL-4RA
#72COMPOSITIONS AND METHODS FOR INHIBITING LPA EXPRESSION
#73Oral Delivery of Antisense Conjugates Targeting PCSK9
#74GalNAc conjugated oligonucleotides for RNA editing
#75Genome Editing Compositions and Methods for Treatment of Friedreich's Ataxia
#76Delivery of RNA Therapeutics Using Circular Prodrug Nucleic Acids
#77COMPOSITIONS AND METHODS FOR TREATING MENINGIOMA
#78METHODS AND COMPOSITIONS FOR MODULATING SPLICING AT ALTERNATIVE SPLICE SITES
#79OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#80NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#81COMPOSITIONS AND METHODS FOR INHIBITING XDH EXPRESSION
#82REVERSIR MOLECULES AND METHODS OF USE THEREOF
#83SIRNA TARGETING ANGPTL4, AND CONJUGATES AND USES THEREOF
#84TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#85COMPOUNDS AND METHODS FOR TREATING HUMAN SUBJECTS
#86OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#87ANTISENSE OLIGONUCLEOTIDE-BASED ANTI-FIBROTIC THERAPEUTICS
#88COMPLEMENT COMPONENT C3 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#89REGULATION OF ACTIVITY OF RNAI MOLECULES
#90METHODS FOR DECREASING OR INHIBITING HUMAN L1 RETROTRANSPOSON RNA AND COMPOSITIONS FOR USE THEREIN
#91AMBIENT TEMPERATURE NUCLEIC ACID AMPLIFICATION AND DETECTION
#92LINKER, COMPOUND INCLUDING LINKER, METHOD OF PREPARING COMPOUND, USE OF LINKER, AND METHOD OF DELIVERING OLIGONUCLEOTIDES THROUGH LINKER
#93RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
#94Compositions and Methods for Correcting Limb Girdle Muscular Dystrophy Type 2C Using Exon Skipping
#95COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF INHIBIN SUBUNIT BETA E (INHBE) GENES
#96OLIGONUCLEOTIDES FOR PRNP MODULATION
#97Compositions and Methods for Lactate Dehydrogenase (LDHA) Gene Editing
#98COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#99SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
#100AGENTS FOR MODULATING EXPRESSION
#101COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF INHIBIN SUBUNIT BETA E (INHBE) GENES
#102RNAi AGENTS OF PRION EXPRESSION
#103EXTRACELLULAR VESICLE-ASO CONSTRUCTS TARGETING CEBP/BETA
#104COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#105Compositions and Methods for Modulating Complement Factor B Expression
#106RNAi Agents for Inhibiting Expression of Complement Factor B (CFB), Pharmaceutical Compositions Thereof, and Methods of Use
#107POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASES
#108DOUBLE STRANDED RNAi AGENTS, COMPOSITIONS AND METHODS OF USE
#109BRANCHED LIPID CONJUGATES OF siRNA FOR SPECIFIC TISSUE DELIVERY
#110Modified Guide RNAs
#111COMPOSITIONS AND METHODS OF TREATING MUSCLE DYSTROPHY
#112RNAi Agents for Inhibiting Expression of Mitochondrial Amidoxime Reducing Component 1 (MARC1), Pharmaceutical Compositions Thereof, and Methods of Use
#113INHIBITORS OF EXPRESSION AND/OR FUNCTION
#114ANTI-C9ORF72 OLIGONUCLEOTIDES AND RELATED METHODS
#115COMPOUND FOR INHIBITING AGT GENE EXPRESSION, AND PHARMACEUTICAL COMPOSITION AND USE THEREOF
#116COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
#117COMPOSITIONS AND METHODS FOR TREATING PANCREATIC CANCER
#118OLIGONUCLEOTIDES FOR MODULATING RTEL1 EXPRESSION
#119MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA) MOLECULES AND USES THEREOF
#120COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
#121OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#122COMPOSITIONS AND METHODS FOR TARGETING, EDITING, OR MODIFYING GENES
#123IMMUNE CELLS DEFECTIVE FOR SUV39H1
#124Methods and Compositions for Adar-Mediated Editing
#125NUCLEIC ACID COMPOUNDS
#126ASS1 Gene Insertion For The Treatment Of Citrullinemia Type I
#127DETECTION ASSAYS
#128SIRNA TARGETING RECQL1 HELICASE GENE
#129METHODS AND COMPOSITIONS FOR INHIBITING VIRUS
#130SMALL INTERFERING RNA TARGETING CFB AND USES THEREOF
#131NOVEL RNA THERAPEUTICS AND USES THEREOF
#132COMPOSITIONS AND METHODS FOR TREATING DISEASES ASSOCIATED WITH PATHOGENIC FUS VARIANTS
#133RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#134MUSCLE-TARGETING COMPLEXES AND USES THEREOF
#135CRISPR INTERFERENCE THERAPEUTICS FOR C9ORF72 REPEAT EXPANSION DISEASE
#136NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#137COMPOSITIONS AND METHODS OF TREATING MUSCLE DYSTROPHY
#138RNAi Agents for Inhibiting Expression of Proprotein Convertase Subtilisin Kexin 9 (PCSK9), Pharmaceutical Compositions Thereof, and Methods of Use
#139THREOSE NUCLEIC ACID ANTISENSE OLIGONUCLEOTIDES AND METHODS THEREOF
#140Selective Antisense Compounds and Uses Thereof
#141COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TRANSTHYRETIN
#142NUCLEIC ACID COMPOUNDS
#143METHODS AND COMPOSITIONS FOR INHIBITION OF IRF4
#144METHOD OF GENETICALLY RECOMBINING LACTIC ACID BACTERIA AND GRAM-POSITIVE BACTERIA USING CRISPR/CAS SYSTEM
#145NUCLEOTIDE ANALOGUES, AND PREPARATION AND APPLICATION THEREOF
#146MODIFIED ANTISENSE OLIGONUCLEOTIDES FOR TREATING HEPATITIS B VIRUS
#147Linkage Modified Oligomeric Compounds and Uses Thereof
#148COMPOSITIONS AND METHODS FOR CONTROL OF PSEUDOGYMNOASCUS DESTRUCTANS
#149Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#150OLIGONUCLEOTIDES
#151TREATMENT OF MS4A4E RELATED DISEASES AND DISORDERS
#152Compositions and Methods for Treating FUS Associated Diseases
#153MUSCLE TARGETING COMPLEXES AND FORMULATIONS FOR TREATING MYOTONIC DYSTROPHY
#154Compositions and Methods Using CPG Oligonucleotides
#155POLYNUCLEIC ACID MOLECULES TARGETING AGT AND USES THEREOF
#156ANTISENSE OLIGONUCLEOTIDES FOR TARGETING PROGRANULIN
#157NANOPARTICLE FORMULATIONS FOR DELIVERY OF NUCLEIC ACID COMPLEXES
#158Compositions and Methods for Treating Cancer
#159METHODS OF TREATING X-LINKED GENETIC DISEASES
#160DUAL-ACTING siRNA BASED MODULATION OF C9orf72
#161Antisense Compounds Targeted to CLN3 and Uses Thereof
#162MODIFIED ANTISENSE OLIGONUCLEOTIDES TARGETING FOXG1
#163Modified Compounds and Uses Thereof
#164COMPOSITIONS COMPRISING PLN-TARGETING ANTI-TRANSFERRIN RECEPTOR ANTIBODY-POLYNUCLEOTIDES AND METHODS OF USE THEREOF TO TREAT CARDIOMYOPATHY
#165COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF CD274/PD-L1 GENE
#166CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
#167COMPOSITIONS AND METHODS FOR TREATMENT OF MICROSATELLITE DNA EXPANSION DISORDERS
#168Antisense Oligonucleotide for Targeting Progranulin
#169ANTISENSE OLIGONUCLEOTIDES
#170RNAi Agents for Inhibiting Expression of Complement Component C3 (C3), Pharmaceutical Compositions Thereof, and Methods of Use
#171RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#172COMPOUNDS AND METHODS FOR REDUCING PCDH19 EXPRESSION
#173COMPOUNDS AND METHODS FOR REDUCING TAU EXPRESSION
#174METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
#175Conjugated Antisense Compounds and Their Use
#176OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#177COMPOSITION FOR REGULATING SPLICING OF POLYBROMO 1 GENE COMPRISING SPLICING-SWITCH OLIGONUCLEOTIDE AS EFFECTIVE COMPONENT
#17817ß-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#179Antisense oligonucleotides for the treatment of Stargardt disease
#180TREATMENT OF SOS2 RELATED DISEASES AND DISORDERS
#181SIRNA FOR INHIBITING ANGPTL3 GENE EXPRESSION AND USE THEREOF
#182COMPOSITIONS AND METHODS OF TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#183Targeting Skin-Related Conditions Using Bottlebrush Polymer-Conjugated Oligonucleotides
#184LPA-TARGETING SIRNA AND CONJUGATE
#185ANTISENSE MODULATION OF SETBP1 EXPRESSION
#186CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES (ASOS) AND COMPOSITIONS FOR RNA EDITING
#187OLIGONUCLEOTIDES FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#188ANTISENSE OLIGONUCLEOTIDES TARGETING ALPHA-SYNUCLEIN AND USES THEREOF
#189COMPOSITIONS AND METHODS FOR EDITING A TRANSTHYRETIN GENE
#190APTAMER, CONJUGATE, COMPOSITION, METHOD FOR PREPARING SAME, AND USE THEREOF
#191Compounds and Methods for Reducing KCNT1 Expression
#192RNAI CONSTRUCTS FOR INHIBITING PNPLA3 EXPRESSION AND METHODS OF USE THEREOF
#193NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND SIRNA CONJUGATE CONTAINING THE NUCLEIC ACID, PREPARATION METHOD THEREFOR, AND USE THEREOF
#194COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#195COMPOSITIONS AND METHODS FOR TREATING HEPATITIS B VIRUS (HBV) INFECTION AND HBV-ASSOCIATED DISEASES
#196MODULATION OF ABCB11 GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
#197INHIBITORS OF EXPRESSION AND/OR FUNCTION
#198METHODS FOR REDUCING TAU EXPRESSION
#199NUCLEIC ACID MOLECULE FOR REDUCTION OF PAPD5 AND PAPD7 mRNA FOR TREATING HEPATITIS B INFECTION
#200Compounds and Methods for Reducing LRRK2 Expression
#201TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#202MUCIN-5B (MUC5B) TARGETED SIRNA AND ANTISENSE OLIGONUCLEOTIDES AND METHODS OF USE THEREOF
#203CONJUGATES FOR TISSUE-SPECIFIC OLIGONUCLEOTIDE DELIVERY
#204CHIRAL DESIGN
#205RNAI CONSTRUCTS FOR INHIBITING SCAP EXPRESSION AND METHODS OF USE THEREOF
#206BASE EDITING OF TRANSTHYRETIN GENE
#207ENHANCING GENE TARGETING EFFICIENCY IN HUMAN CELLS WITH DNA-PK INHIBITOR TREATMENT
#208TLR9-TARGETED SPHERICAL NUCLEIC ACIDS HAVING POTENT ANTITUMOR ACTIVITY
#209Methods for Reducing Ataxin-2 Expression
#210COMPOSITIONS AND METHODS FOR SPLICING MODULATION OF UNC13A
#211STEREOSPECIFIC LINKAGES IN RNA EDITING OLIGONUCLEOTIDES
#212EDITING OLIGONUCLEOTIDE
#213RNAi Agents for Inhibiting Expression of Microtubule Associated Protein Tau (MAPT), Compositions Thereof, and Methods of Use
#214TRANSGENE CASSETTES DESIGNED TO EXPRESS A HUMAN MECP2 GENE
#215OLIGONUCLEOTIDE-BASED DELIVERY VEHICLE FOR OLIGONUCLEOTIDES AGENTS AND METHODS OF USE THEREOF
#216SPLICE-SWITCHING OLIGONUCLEOTIDES
#217COMPOUNDS AND METHODS FOR MODULATING ATXN1
#218CERTAIN DUX4 INHIBITORS AND METHODS OF USE THEREOF
#219METHODS AND COMPOSITIONS FOR INHIBITING MISMATCH REPAIR
#220MUC5AC-TARGETED ANTISENSE OLIGONUCLEOTIDES AND RELATED METHODS FOR MODULATING MUCIN EXPRESSION
#221MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING DYSTROPHINOPATHIES
#222TARGETED TREATMENT OF SPLICEOPATHY-INDUCED NEUROLOGICAL DISORDERS
#223CHEMICALLY MODIFIED ANTISENSE OLIGONUCLEOTIDES (ASOS) AND COMPOSITIONS COMPRISING THE SAME FOR RNA EDITING
#224MODULATION OF SYNGAP1 GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
#225IMPROVED OLIGONUCLEOTIDES TARGETING RNA BINDING PROTEIN SITES
#226COMPOSITIONS FOR THE SILENCING OF snoRNAS AND METHODS OF USING SAME
#227METHODS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF LDHA
#228MODULATORS OF DIACYGLYCEROL ACYLTRANSFERASE 2 (DGAT2)
#229MAPT ANTISENSE OLIGONUCLEOTIDE
#230COMPOSITIONS AND METHODS FOR SPLICING MODULATION OF UNC13A
#231Compositions for Treatment of Polycystic Kidney Disease
#232Compounds and Methods for Modulating UBE3A-ATS
#233ACTIN-BINDING LIM PROTEIN 3 (ABLIM3) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#234TREATMENT OF CARDIOVASCULAR DISEASE
#235COMPOSITIONS FOR TARGETING FLI-1 AND METHODS OF USE THEREOF
#236MODIFIED DOUBLE STRANDED OLIGONUCLEOTIDES
#237FN3 DOMAIN-siRNA CONJUGATES AND USES THEREOF
#238THERAPEUTIC COMPOSITIONS FOR TREATING PAIN VIA MULTIPLE TARGETS
#239OLIGONUCLEOTIDES FOR PRNP MODULATION
#240METHODS AND COMPOSITIONS FOR TREATING A PROPROTEIN CONVERTASE SUBTILISIN KEXIN (PCSK9) GENE-ASSOCIATED DISORDER
#241SPLICE SWITCHING OLIGONUCLEOTIDES TARGETING PSEUDOEXONS
#242INHIBITORS OF MIR-221 FOR THE TREATMENT OF SOLID NEOPLASMS AND PERIPHERAL NEUROTOXICITY INDUCED BY ANTICANCER DRUGS AND OTHER NEUROLOGICAL DISEASES RELATED TO MIR-221
#243LPA inhibitors and uses thereof
#244Novel CRISPR gRNAs
#245BIODIFFUSION CHAMBER
#246MUSCLE-TARGETING COMPLEXES AND USES THEREOF
#247SERUM AMYLOID P COMPONENT (APCS) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#248TARGETED AUGMENTATION OF NUCLEAR GENE OUTPUT
#249ANTI-CANCER AGENTS
#250COMPOSITIONS, SYSTEMS, AND METHODS FOR REGULATION OF HEPATITIS B VIRUS THROUGH TARGETED GENE REPRESSION
#251COMPOUNDS AND METHODS FOR MODULATING SCN2A
#252Selective Antisense Compounds and Uses Thereof
#253COMPOSITIONS AND METHODS FOR TREATING TARDBP ASSOCIATED DISEASES
#254CONJUGATE, PHARMACEUTICAL COMPOSITION COMPRISING SAME, METHOD FOR PREPARING SAME, AND USE THEREOF
#255PHARMACEUTICAL COMBINATIONS FOR TREATMENT OF HBV
#256Linkage Modified Oligomeric Compounds
#257COMPOSITIONS AND METHODS FOR MODULATING APOC3 EXPRESSION
#258COMPOSITIONS AND METHODS OF MODULATING RNA AND PROTEIN INTERACTIONS
#259KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#260COMPOSITION AND METHOD FOR INHIBITING EXPRESSION OF PROTEIN LPA(APO(A))
#261Antisense oligonucleotides for treatment of USHER 2A. Exons 30-31
#262SIGNAL ACTIVATABLE NUCLEIC ACID COMPLEXES
#263REVERSIR TM COMPOUNDS
#264PRIMARY CELL GENE EDITING
#265MODULATION OF GENE TRANSCRIPTION USING ANTISENSE OLIGONUCLEOTIDES TARGETING REGULATORY RNAS
#266SIRNA OR SALT THEREOF AND MEDICAMENT FOR INHIBITING EXPRESSION OF TMPRSS6 GENE, AND USE THEREOF
#267POLYNUCLEIC ACID MOLECULES FOR INHIBITING EXPRESSION OF FXI, PHARMACEUTICAL COMPOSITIONS, AND USES THEREOF
#268XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#269DOUBLE-STRANDED RNA MOLECULE FOR ADMINISTRATION TO THE EYE
#270TRANSLATION ENHANCING NUCLEIC ACID COMPOUNDS: ASO COUPLED TRANSLATION – UPREGULATION 1 (ACT-UP1) AND USES THEREOF
#271MODIFIED OLIGONUCLEOTIDES
#272OLIGONUCLEOTIDES CONJUGATES COMPRISING 7'-5'-ALPHA-ANOMERIC-BICYCLIC SUGAR NUCLEOSIDES
#273COMPOSITIONS COMPRISING THERAPEUTIC NUCLEIC ACID AND SAPONIN FOR THE TREATMENT OF MUSCLE-WASTING DISORDERS
#274SARM1 RNAi AGENTS
#275Compounds and Methods for Reducing APOCIII Expression
#276COMPOSITIONS AND METHODS FOR INHIBITING STAG1 EXPRESSION AND USES THEREOF
#277Materials and Methods for Treatment of Hemoglobinopathies
#278MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING MYOTONIC DYSTROPHY
#279METHODS OF TREATMENT OF GNAQ AND GNA11 DRIVEN DISEASE
#280ANTISENSE NUCLEIC ACID CAPABLE OF INHIBITING BIOSYNTHESIS OF CHONDROITIN SULFATE
#281SYF2 ANTISENSE OLIGONUCLEOTIDES
#282Selective Reduction of Allelic Variants
#283OLIGONUCLEOTIDES WITH 2'-DEOXY-2'-F-2'-C-METHYL NUCLEOTIDES
#284IMMUNOSTIMULATORY COMPOSITIONS
#285RNAi Agents for Inhibiting Expression of Complement Factor B (CFB), Pharmaceutical Compositions Thereof, and Methods of Use
#286BRANCHED TRIPLE LIPID-MODIFIED NUCLEIC ACID COMPOUNDS
#287TUNING CASCADE ASSAY KINETICS VIA MOLECULAR DESIGN
#288MODIFIED RNAi AGENTS
#289RNAI CONSTRUCTS FOR INHIBITING PNPLA3 EXPRESSION AND METHODS OF USE THEREOF
#290COMPOUNDS AND COMPOSITIONS INCLUDING PHOSPHOROTHIOATED OLIGODEOXYNUCLEOTIDE, AND METHODS OF USE THEREOF
#291PHYTOECDYSONES AND/OR 20-HYDROXYECDYSONE DERIVATIVES IN COMBINATION WITH AN ACTIVE INGREDIENT FOR RESTORING SMN EXPRESSION, FOR USE IN THE TREATMENT OF SPINAL MUSCULAR ATROPHY
#292GALNAC COMPOSITIONS FOR IMPROVING SIRNA BIOAVAILABILITY
#293COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF ALPHA-1 ANTITRYPSIN
#294COMPOSITIONS AND METHODS FOR THE TREATMENT OF ANGIOPOIETIN LIKE 7 (ANGPTL7) RELATED DISEASES
#295RNAi Agents of Modulating PLP1
#296OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF FOR EXON SKIPPING
#297METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#298Multiplexed Analysis of Materials for Tissue Delivery
#299COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
#300METHODS OF DESIGNING CONDITIONAL-ACTIVATABLE SMALL INTERFERING RNA SENSORS