109170 ⎘
Structure or type of the nucleic acid; Chemical structure of the sugar 2'-O-R Modification
POLYNUCLEIC ACID MOLECULES TARGETING PCSK9 AND USES THEREOF
#602Asymmetric Short Duplex DNA as a Novel Gene Silencing Technology and Use Thereof
#603COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#604OLIGONUCLEOTIDES AND COMPOSITIONS THEREOF FOR NEUROMUSCULAR DISORDERS
#605MIRNA INHIBITORS FOR PREVENTING AND TREATING ANEURYSMS, HYPERTENSION, ARDS AND OTHER DISEASES ASSOCIATED WITH ENDOTHELIAL DYSFUNCTION
#606METHODS FOR THE TREATMENT OF NUCLEOTIDE REPEAT EXPANSION DISORDERS ASSOCIATED WITH MSH3 ACTIVITY
#607SERPINA1 SIRNAS: COMPOSITIONS OF MATTER AND METHODS OF TREATMENT
#608BETA-CATENIN (CTNNB1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#609ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES
#610COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#611SIRNAS TARGETING SLC10A1 TRANSCRIPTS, COMPOSITIONS AND USES THEREOF
#612SIGNAL TRANSDUCER AND ACTIVATOR OF TRANSCRIPTION FACTOR 6 (STAT6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#613ANTISENSE OLIGONUCLEOTIDE TARGETING CAV3.1 GENE AND USES THEREOF
#614RNA INTERFERENCE MEDIATED INHIBITION OF CATENIN (CADHERIN-ASSOCIATED PROTEIN), BETA 1 (CTNNB1) GENE EXPRESSION USING SHORT INTERFERING NUCLEIC ACID (SINA)
#615Modulators of Complement Factor B
#616METHODS AND COMPOSITIONS FOR TREATING AN ANGIOTENSINOGEN- (AGT-) ASSOCIATED DISORDER
#617NOVEL lncRNA CONTROLLING CARDIAC FIBROSIS
#618CONJUGATED ANTISENSE COMPOUNDS AND THEIR USE
#619TARGETED NUCLEIC ACID CONJUGATE COMPOSITIONS
#620ANTISENSE OLIGONUCLEOTIDES TARGETING ADENOSINE KINASE
#621Heteronucleic acid containing scpBNA or AmNA
#622HUMAN CHROMOSOME 9 OPEN READING FRAME 72 (C9ORF72) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#623COMPOSITIONS, SYSTEMS AND METHODS OF RNA EDITING USING DKC1
#624COMPOSITIONS AND METHODS FOR CD123 MODIFICATION
#625NUCLEIC ACID MEDICINE EXPRESSING SPLICING VARIANT OF MYOSTATIN
#626NUCLEIC ACID MOLECULES FOR PSEUDOURIDYLATION
#627SPLICE-SWITCHING OLIGONUCLEOTIDES AND METHODS OF USE
#628siRNA AND USE THEREOF
#629MODIFIED NUCLEIC ACIDS, HYBRID GUIDE RNAS, AND USES THEREOF
#630Programmed Cell Death Protein 1 (PD1) Compositions and Methods for Cell-Based Therapy
#631CORRECTION OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS WITH ALL-IN-ONE ADENO-ASSOCIATED VIRUS-DELIVERED SINGLE-CUT CRISPR
#632COMPOSITIONS OF MATTER FOR DETECTION ASSAYS
#633Nucleic Acid Compounds That Bind Coronavirus Proteins
#634OPTIMIZED 2'- MODIFIED RIBOSE DERIVATIVES AND METHODS OF USE
#635GUIDE RNAS WITH CHEMICAL MODIFICATION FOR PRIME EDITING
#636MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING FRIEDREICH'S ATAXIA
#637THERAPEUTIC COMPOSITIONS FOR TREATING PAIN VIA MULTIPLE TARGETS
#638Specific Oligonucleotide-Programmed Readthrough of Nonsense Codons
#639iRNA COMPOSITIONS AND METHODS FOR SILENCING CHITINASE 3-LIKE PROTEIN 1/YKL-40 (CHI3L1/YKL-40) PROTEIN
#640COMPOSITIONS AND METHODS FOR TREATING CANCER
#641Complement component C3 iRNA compositions and methods of use thereof
#642PHARMACEUTICAL COMPOSITION CONTAINING CELL-DERIVED NATURAL OR ARTIFICIAL NANOVESICLES LOADED WITH ANTISENSE OLIGONUCLEOTIDE-BASED DRUG
#643NUCLEIC ACID MOLECULE CONJUGATE HAVING PROLONGED IN VIVO HALF-LIFE
#644GENE THERAPY FOR INFLAMMATORY CONDITIONS
#645MICROGLIAL GENE SILENCING USING DOUBLE-STRANDED SIRNA
#646TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES
#647TRANSGENE CASSETTES DESIGNED TO EXPRESS A HUMAN MECP2 GENE
#648DEFINED MULTI-CONJUGATES OLIGONUCLEOTIDES
#649ANGIOTENSINOGEN (AGT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#650COMPOUNDS AND METHODS FOR REDUCING NLRP3 EXPRESSION
#651COMPOUNDS AND METHODS FOR REDUCING PLN EXPRESSION
#652RNA SILENCING AGENTS AND METHODS OF USE
#653MicroRNA (miRNA) and Downstream Targets for Diagnostic and Therapeutic Purposes
#654TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#655OLIGONUCLEOTIDES TARGETING S6K1
#656TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
#657KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#658METHODS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF LDHA
#659Non-liposomal systems for nucleic acid delivery
#660DOUBLE-STRANDED SIRNA HAVING PATTERNED CHEMICAL MODIFICATIONS
#661FN3 DOMAIN-SIRNA CONJUGATES AND USES THEREOF
#662TREATMENT OF MST1 RELATED DISEASES AND DISORDERS
#663ANGIOPOIETIN-LIKE 3 (ANGPTL3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#664RNA INHIBITOR FOR INHIBITING HEPATITIS B VIRUS GENE EXPRESSION AND APPLICATION THEREOF
#665SMALL INTERFERING RNA TARGETING C3 AND USES THEREOF
#666DOSAGES AND METHODS FOR DELIVERING LIPID FORMULATED NUCLEIC ACID MOLECULES
#667METHODS FOR MODULATING FMR1 EXPRESSION
#668COMPLEMENT COMPONENT C5 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#669OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#670EXTRACELLULAR VESICLE-NLRP3 ANTAGONIST
#671RNAi agents for inhibiting expression of complement component C3 (C3), pharmaceutical compositions thereof, and methods of use
#672Methods for Treatment of Alport Syndrome
#673MODIFIED GUIDE RNAS FOR CRISPR GENOME EDITING
#674TEMPLATE DIRECTED IMMUNOMODULATION FOR CANCER THERAPY
#675MODIFIED NUCLEIC ACID MOLECULES AND USES THEREOF
#676METHOD OF TREATING FATTY LIVER DISEASE
#677THERAPEUTIC SPLICE-SWITCHING OLIGONUCLEOTIDES FOR CANCER
#678Modified Guide RNAs Comprising an Internal Linker for Gene Editing
#679ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
#680Splicing Modulators for the Treatment of Timothy Syndrome
#681ANTISENSE OLIGONUCLEOTIDES TARGETING FOXG1
#682OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#683Lipid formulations for delivery of therapeutic agents
#684PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#685MODULATORS AND MODULATION OF THE RECEPTOR FOR ADVANCED GLYCATION END-PRODUCTS RNA
#686MODULATION OF GYS1 EXPRESSION
#687ALPHA-1 ANTITRYPSIN (AAT) RNAI AGENTS, COMPOSITIONS INCLUDING AAT RNAI AGENTS, AND METHODS OF USE
#688ALLELE SELECTIVE INHIBITION OF MUTANT C9ORF72 FOCI EXPRESSION BY DUPLEX RNAS TARGETING THE EXPANDED HEXANUCLEOTIDE REPEAT
#689TARGETED INHIBITION USING ENGINEERED OLIGONUCLEOTIDES
#690Selective Antisense Compounds and Uses Thereof
#691ANTISENSE OLIGONUCLEOTIDES FOR USE IN THE TREATMENT OF CORNEAL DYSTROPHIES
#692Oligonucleotide Comprising Inosine for Treating DMD
#693TARGETED RNA EDITING
#694NUCLEIC ACID MEDICINE EXPRESSING SPLICING VARIANT OF MYOSTATIN
#695NUCLEIC ACID MOLECULES FOR PSEUDOURIDYLATION
#696MODULATION OF TRANSTHYRETIN EXPRESSION
#697Compositions and methods of treating facioscapulohumeral muscular dystrophy
#698COMPOSITIONS FOR USE IN THE TREATMENT OF CHD2 HAPLOINSUFFICIENCY AND METHODS OF IDENTIFYING SAME
#699RNAI AGENT FOR INHIBITING HBV EXPRESSION AND USE THEREOF
#700Compositions and methods of treating facioscapulohumeral muscular dystrophy
#701METHODS AND COMPOSITIONS FOR PRODUCTION OF GENETICALLY MODIFIED PRIMARY CELLS
#702MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING MYOTONIC DYSTROPHY
#703COMPOSITIONS FOR TREATMENT OF CONDITIONS AND DISEASES ASSOCIATED WITH POLYCYSTIN EXPRESSION
#704COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION IN THE CENTRAL NERVOUS SYSTEM
#705MODIFIED HETERO NUCLEIC ACIDS
#706METHODS FOR PERFORMING ANTISENSE OLIGONUCLEOTIDE-MEDIATED EXON SKIPPING IN THE RETINA OF A SUBJECT IN NEED THEREOF
#707COMPOSITIONS AND METHODS FOR TREATING PULMONARY ARTERIAL HYPERTENSION (PAH) AND OTHER DISORDERS
#708METHODS FOR PROPHYLACTIC AND THERAPEUTIC TREATMENT OF 2019-nCoV USING siRNAs AGAINST ORF1AB and N-Protein
#709SUBSTANCES AND METHODS FOR TREATING DYSTROPHIC EPIDERMOLYSIS BULLOSA
#710COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) GENES
#711COMBINATION siRNA AND PEPTIDE DOCKING VEHICLE (PDoV) COMPOSITIONS AND METHODS OF THEIR USE FOR IMPROVED REGULATION AND FUNCTIONALITY
#712POLYNUCLEOTIDE AGENTS TARGETING PROGRAMMED CELL DEATH 1 LIGAND 1 (PD-L1) AND METHODS OF USE THEREOF
#713OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#714OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#715Antisense Oligonucleotides Targeting SARS-CoV-2
#716OPA1 ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
#717NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS
#718MODULATION OF AAV-BASED GENE EXPRESSION
#719STABLE TARGET-EDITING GUIDE RNA TO WHICH CHEMICALLY MODIFIED NUCLEIC ACID IS INTRODUCED
#720COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#721COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
#722IL-34 antisense agents and methods of using same
#723ANTISENSE OLIGONUCLEOTIDES INCREASING FOXG1 EXPRESSION
#724CHEMICALLY REVERSIBLE 2`-OH ACYLATION PROTECTS RNA FROM HYDROLYTIC AND ENZYMATIC DEGRADATION
#725OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#726Methods and Systems for Guide RNA Design and Use
#727RNAI CONSTRUCTS AND METHODS FOR INHIBITING FAM13A EXPRESSION
#728COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#729Oligonucleotides reducing the amount of CD73 mRNA and CD73 protein expression
#730FACTOR XII (HAGEMAN FACTOR) (F12), KALLIKREIN B, PLASMA (FLETCHER FACTOR) 1 (KLKB1), AND KININOGEN 1 (KNG1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#731COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN (TTR) MEDIATED AMYLOIDOSIS
#732METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL DOMINANT CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA (CPVT)
#733Target Ligand
#734Compositions, systems, and methods for regulation of hepatitis B virus through targeted gene repression
#735Compositions, systems, and methods for regulation of hepatitis b virus through targeted gene repression
#736OPTIMIZED ANTI-FLT1 OLIGONUCLEOTIDE COMPOUNDS FOR TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS
#737METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
#738ANTISENSE COMPOUNDS
#739Bispecific Aptamer Compositions for the Treatment of Retinal Disorders
#740ANTISENSE OLIGONUCLEOTIDES AND THEIR USE FOR THE TREATMENT OF PAIN
#741OLIGONUCLEOTIDE AND USE THEREOF AGAINST HEPATITIS B VIRUS AND HEPATITIS D VIRUS
#742DOUBLE STRANDED RNA TARGETING ANGIOTENSINOGEN (AGT) AND METHODS OF USE THEREOF
#743PHARMACEUTICAL COMPOSITION
#744OLIGONUCLEOTIDES FOR PMS1 MODULATION
#745OPTIMIZED TLR7 LIGANDS AND USES THEREOF
#746APPLICATION OF CRISPR/CAS13 FOR THERAPY OF RNA VIRUS AND/OR BACTERIUM INDUCED DISEASES
#747ANGIOTENSINOGEN-MODULATING COMPOSITIONS AND METHODS OF USE THEREOF
#748Methods and compositions for modulating a genome
#749ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES
#750Compositions of matter for detection assays
#751COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
#752OLIGONUCLEOTIDES FOR MODULATING TAU EXPRESSION
#753OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#754OLIGONUCLEOTIDES USEFUL FOR MODULATION OF SPLICING
#755Stabilized saRNA Compositions and Methods of Use
#756SNCA IRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING SNCA-ASSOCIATED NEURODEGENERATIVE DISEASES
#757MODIFIED OLIGONUCLEOTIDES FOR USE IN TREATMENT OF TAUOPATHIES
#758DOUBLE STRANDED RNA TARGETING 17-BETA HYDROXYSTEROIDDEHYDROGENASE 13 (HSD17B13) AND METHODS OF USE THEREOF
#759Nucleic Acid Molecule Binding to YB-1 Protein
#760COMPOUNDS FOR MODULATING CHMP7
#761LINKAGE MODIFIED OLIGOMERIC COMPOUNDS AND USES THEREOF
#762TREATMENT OF FUCHS' ENDOTHELIAL CORNEAL DYSTROPHY
#763COMPOSITIONS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
#764MODULATION OF GENE EXPRESSION AND SCREENING FOR DEREGULATED PROTEIN EXPRESSION
#765CHEMICAL MODIFICATIONS FOR INHIBITING EXPRESSION OF ALDH2
#766COMPOSITIONS AND METHODS FOR INHIBITING SNCA EXPRESSION
#767COMPOSITIONS AND METHODS FOR INHIBITING MAPT EXPRESSION
#768UMLILO ANTISENSE TRANSCRIPTION INHIBITORS
#769GUIDE RNA WITH CHEMICAL MODIFICATIONS
#770METHODS AND COMPOSITIONS RELATING TO GENETICALLY ENGINEERED CELLS EXPRESSING CHIMERIC ANTIGEN RECEPTORS
#771METHODS AND COMPOSITIONS FOR INCREASING EFFICIENCY OF TARGETED GENE MODIFICATION USING OLIGONUCLEOTIDE-MEDIATED GENE REPAIR
#772METHODS AND COMPOSITIONS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#773TREATMENT OF OPTIC ATROPHY
#774MODIFIED ANTISENSE OLIGONUCLEOTIDES TARGETING SPLICING FACTORS
#775NOVEL EFFICACIOUS MICRORNA-30C ANALOGS REDUCE APOLIPOPROTEIN B SECRETION IN HUMAN LIVER CELLS
#776TREATMENT OF SOS2 RELATED DISEASES AND DISORDERS
#777PROTECTING OLIGONUCLEOTIDES FOR CRISPR GUIDE RNA
#778Compositions of matter for detection assays
#779EXOSOMAL LOADING USING HYDROPHOBICALLY MODIFIED OLIGONUCLEOTIDES
#780COMPOSITIONS AND METHODS OF TREATING MUSCLE DYSTROPHY
#781COMPOSITIONS AND METHODS FOR CD38 MODIFICATION
#782Huntingtin (HTT) iRNA agent compositions and methods of use thereof
#783NUCLEIC ACID MOLECULES WITH INCREASED GENE SILENCING ACTIVITY AND USES THEREOF
#784NOVEL RNA COMPOSITIONS AND METHODS FOR INHIBITING ANGPTL3
#785MODIFIED FUNCTIONAL NUCLEIC ACID MOLECULES
#786Acetylated ribonucleic acids and uses thereof
#787COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISEASE TYPE 1A
#788COMPOSITIONS AND METHODS FOR MODULATING SCAP ACTIVITY
#789COMPOUNDS AND METHODS FOR MODULATING PMP22
#790METHODS AND COMPOSITIONS FOR MACROPHAGE POLARIZATION
#791Modified short interfering nucleic acid (siNA) molecules and uses thereof
#792SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#793COMPOSITIONS AND METHODS FOR CD6 MODIFICATION
#794Methods for treating or preventing ophthalmological conditions
#795METHODS FOR TREATING OR PREVENTING OPHTHALMOLOGICAL CONDITIONS
#796GENE CORRECTION FOR X-CGD IN HEMATOPOIETIC STEM AND PROGENITOR CELLS
#797COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
#798OLIGONUCLEOTIDE TREATMENT OF HEPATITIS B PATIENTS
#799COMPOUNDS AND METHODS FOR REDUCING APOE EXPRESSION
#800Nucleic acid, composition and conjugate containing same, preparation method, and use thereof
#801RNAI AGENTS FOR HEPATITIS B VIRUS INFECTION
#802ANTISENSE OLIGONUCLEOTIDES FOR MODULATION OF LONG NONCODING RNAS
#803DYSTROPHIN EXON SKIPPING OLIGONUCLEOTIDES
#804SHORT INTERFERING NUCLEIC ACID (siNA) COMPOSITIONS
#805OLIGONUCLEOTIDES
#806Compositions of matter for detection assays
#807COMPOSITIONS AND METHODS FOR INHIBITING MITOCHONDRIA AMIDOXIME REDUCING COMPONENT 1 (MARC1) EXPRESSION
#808COMPOSITIONS AND METHODS FOR THE TREATMENT OF METABOLIC SYNDROME
#809USES FOR PREVENTION OR TREATMENT OF BRAIN DISEASES USING MICRORNA
#810Compositions and methods of treating facioscapulohumeral muscular dystrophy
#811SIRNA THERAPY FOR TRANSTHYRETIN (TTR) RELATED OCULAR AMYLOIDOSIS
#812METHODS FOR USING GUIDE RNAS WITH CHEMICAL MODIFICATIONS
#813RNA-editing oligonucleotides and uses thereof
#814Base editing of ANGPTL3 and methods of using same for treatment of disease
#815Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
#816DISCONTINUOUS OLIGONUCLEOTIDE LIGANDS
#817IRNA COMPOSITIONS AND METHODS FOR SILENCING GROWTH FACTOR RECEPTOR BOUND PROTEIN 10 (GRB10) OR GROWTH FACTOR RECEPTOR BOUND PROTEIN 14 (GRB14) IN THE LIVER
#818RNA-editing oligonucleotides and uses thereof
#819OLIGOMERS HAVING BICYCLIC SCAFFOLD MOIETIES
#820OLIGONUCLEOTIDES TARGETING XBP1
#821TUMOR NECROSIS FACTOR RECEPTOR ASSOCIATED FACTOR 6 (TRAF6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#822ANTIVIRAL SILENCING RNA MOLECULES, CHEMICALLY MODIFIED ANTIVIRAL SILENCING RNA MOLECULES WITH ENHANCED CELL PENETRATING ABILITIES, PHARMACEUTICAL COMPOSITIONS COMPRISING SAME AND USES THEREOF FOR TREATMENT OF VIRAL INFECTIONS
#823RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
#824UNA OLIGOMERS FOR THE TREATMENT OF POLYGLUTAMINE DISEASES
#825Polynucleotides containing a morpholino linker
#826MONOCLONAL ANTIBODIES TO CHEMICALLY-MODIFIED NUCLEIC ACIDS AND USES THEREOF
#827OLIGONUCLEOTIDES FOR MLH3 MODULATION
#828MUSCLE REGENERATION AND GROWTH
#829Modified oligonucleotides
#830TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES
#831Oligonucleotides for inducing paternal UBE3A expression
#832METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF LEUCINE RICH REPEAT KINASE 2 (LRRK2)
#833ANTI-C9ORF72 OLIGONUCLEOTIDES AND RELATED METHODS
#834Targeted inhibition using engineered oligonucleotides
#835OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#836COMPOSITIONS AND METHODS FOR INHIBITING ANGPTL3 EXPRESSION
#837RNAi COMPOSITIONS AND METHODS OF USE THEREOF FOR DELIVERY BY INHALATION
#838Compositions and methods of treating facioscapulohumeral muscular dystrophy
#839OPA1 ANTISENSE OLIGOMERS FOR TREATMENT OF CONDITIONS AND DISEASES
#840RNAI CONSTRUCTS FOR INHIBITING HSD17B13 EXPRESSION AND METHODS OF USE THEREOF
#841Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#842Compositions and methods of treating Facioscapulohumeral muscular dystrophy
#843Single-stranded RNA-editing oligonucleotides
#844METHODS AND COMPOSITIONS FOR MODULATING A GENOME
#845ANTISENSE OLIGONUCLEOTIDE INDUCING EXON SKIPPING OF ANGIOTENSIN CONVERTING ENZYME 2 GENE
#846Compositions and methods for modulating APOC3 expression
#847ADAR DEPENDENT EDITING COMPOSITIONS AND METHODS OF USE THEREOF
#848siRNAs WITH VINYLPHOSPHONATE AT THE 5' END OF THE ANTISENSE STRAND
#849METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF RETINOCHISIN 1 (RS1)
#850METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF GAP JUNCTION PROTEIN BETA 2 (GJB2)
#851COMPLEMENT COMPONENT C3 iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING COMPLEMENT COMPONENT C3-ASSOCIATED DISEASES
#852METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF TRANSMEMBRANE CHANNEL-LIKE PROTEIN 1 (TMC1)
#853METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF METHYL-CPG BINDING PROTEIN 2 (MECP2)
#854Compositions and methods for targeting, editing, or modifying genes
#855TARGETING THE HUMAN CCR5 LOCUS AS A SAFE HARBOR FOR THE EXPRESSION OF THERAPEUTIC PROTEINS
#856SMAC/Diablo inhibitors useful for treating cancer
#857RNAi agents for inhibiting expression of DUX4, compositions thereof, and methods of use
#858Modulators of APOL1 expression
#859Genetically engineered T cells with Regnase-1 and/or TGFBRII disruption have improved functionality and persistence
#860MODULATION OF FACTOR 11 EXPRESSION
#861METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF ARGININOSUCCINATE SYNTHETASE (ASS1)
#862SIRNA OF ANGPTL3 AND USE THEREOF
#863Compositions and methods for TTR gene editing and treating ATTR amyloidosis
#864Circular siRNAs
#865Compositions and methods for inhibiting expression of transthyretin
#866Modified messenger RNA comprising functional RNA elements
#867Nucleic acid oligomers and uses therefor
#868PCSK9 TARGETING OLIGONUCLEOTIDES FOR TREATING HYPERCHOLESTEROLEMIA AND RELATED CONDITIONS
#869RESPIRATORY SYNCYTIAL VIRUS (RSV) POLYANHYDRIDE NANOPARTICLE VACCINE
#870OPA1 antisense oligomers for treatment of conditions and diseases
#871Lipid formulations for delivery of therapeutic agents
#872Compositions of matter for detection assays
#873Compositions and methods for targeting, editing, or modifying genes
#874TREATMENT OF NEUROLOGICAL DISEASES USING MODULATORS OF GENE TRANSCRIPTS
#875COMPOUNDS AND METHODS FOR REDUCING MSH3 EXPRESSION
#876ANTISENSE OLIGONUCLEOTIDES (ASO) FOR EFFICIENT AND PRECISE RNA EDITING WITH ENDOGENOUS ADENOSINE DEAMINASE ACTING ON RNA (ADAR)
#877NUCLEASE SYSTEMS FOR GENETIC ENGINEERING
#878RNA EDITING INHIBITORS AND METHODS OF USE
#879Reactions with non-retroviral reverse transcriptase
#880Transmembrane protease, serine 6 (TMPRSS6) iRNA compositions and methods of use thereof
#881METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#882OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#883INHIBITION OF POLYOMAVIRUS REPLICATION
#884COMPOSITIONS AND METHODS FOR TREATING HYPERTRIGLYCERIDEMIA
#885METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF OTOFERLIN (OTOF)
#886THERAPEUTICS FOR HAPLOINSUFFICIENCY CONDITIONS
#887METHODS AND COMPOSITIONS FOR MODULATING ALPHA-1-ANTITRYPSIN EXPRESSION
#888ANTISENSE THERAPEUTICS FOR BETACORONAVIRUS TREATMENT
#889TGF-BETA INHIBITION, AGENTS AND COMPOSITION THEREFOR
#890CYTOTOXIC T-LYMPHOCYTE BINDING APTAMERS
#891BISPECIFIC PERSONALIZED APTAMERS
#892OLIGONUCLEOTIDES FOR SOD1 MODULATION
#893PRODUCTS FOR SUPPRESSING OR REDUCING THE EXPRESSION OR ACTIVITY OF A SNORNA AND USES THEREOF IN THE TREATMENT OF CANCER
#894Reducing intron retention
#895Methods and Compositions for the ADAR-Mediated Editing of ABCA4
#896Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
#897COMPOSITIONS AND METHODS FOR TREATING DISORDERS ASSOCIATED WITH LOSS-OF-FUNCTION MUTATIONS IN SYNGAP1
#898IMMUNOSUPPRESSANT-RESISTANT T-CELLS FOR ADOPTIVE IMMUNOTHERAPY
#899COMPOSITIONS AND METHODS FOR CORRECTING LIMB GIRDLE MUSCULAR DYSTROPHY TYPE 2C USING EXON SKIPPING
#900Compositions for FNIP1/FNIP2 Gene Modulation and Methods Thereof