109054 ⎘
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor; Recombinant DNA-technology; DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides against enzymes
IRAP INHIBITORS FOR USE IN THE TREATMENT OF INFLAMMATORY DISEASES
#302RNAI THERAPY FOR APBD AND LAFORA DISEASE
#303COMBINED TARGETING OF STAT3 AND ULKI TO TREAT GLIOBLASTOMA
#304Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
#305COMPOSITIONS AND METHODS FOR ENHANCING GENE SILENCING ACTIVITY OF OLIGONUCLEOTIDE COMPOUNDS
#306COMPOUND AND LIPID COMPOSITION
#307OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF MAKING THE SAME
#308ANTISENSE TRANSFER VECTORS AND METHODS OF USE THEREOF
#309METHODS AND COMPOSITIONS FOR TARGETING ALTERNATIVE METABOLISM ALONG WITH FLT3 INHIBITOR-MEDIATED ANTILEUKEMIC ACTIONS
#310NANOPARTICLE DRUG TARGETING JFK AND USE THEREOF
#311METHODS TO IDENTIFY AND TREAT SUBJECTS HAVING CORTICOSTEROID-RESISTANT INFLAMMATORY DISEASES
#312REGENERATION OR REJUVENATION OF TISSUES AND ORGANS
#313COMPOSITIONS AND METHODS FOR TREATMENT OF MIC60 DEPLETED CANCERS AND METASTASIS
#314NUCLEIC ACIDS CONTAINING ABASIC NUCLEOSIDES
#315Glioblastoma Multiforme Treatments
#316PARN AS A BIOMARKER AND THERAPEUTIC TARGET
#317METHODS FOR TREATING CELLULAR INFLAMMATION INDUCED BY AFB1
#318ANTISENSE OLIGONUCLEOTIDES HAVING ONE OR MORE ABASIC UNITS
#319USE OF PI3KC2B INHIBITORS FOR THE PRESERVATION OF VASCULAR ENDOTHELIAL CELL BARRIER INTEGRITY
#320SIRNA STRUCTURES FOR HIGH ACTIVITY AND REDUCED OFF TARGET
#321NUCLEIC ACID MOLECULE FOR TREATING BIETTI CRYSTALLINE DYSTROPHY AND USE THEREOF
#322OLIGONUCLEOTIDES FOR SARS-CoV-2 MODULATION
#323Compositions For and Methods of Improving Viral Vectors
#324INHIBITION OF GLUTARYL-COA DEHYDROGENASE FOR THE TREATMENT OF MELANOMA
#325MICRORNA TARGETING REGENERATIVE TREATMENTS FOR HEART FAILURE
#326RNAi TARGETING PPP2R5D MISSENSE MUTATIONS FOR TREATMENT OF JORDAN’S SYNDROME
#327COMPOSITIONS AND METHODS FOR THE INHIBITION OF TUMOR METASTASIS AND HORIZONTAL GENE TRANSFER
#328Atg8ylation coordinates stress granule formation and mTOR inactivation in response to lysosomal damage
#329METHOD OF MODULATING PCSK9 AND USES THEREOF
#330COMPOSITIONS AND METHODS FOR MODULATING PNPLA3 EXPRESSION
#331METHOD OF REGULATING PULMONARY ARTERY VASCULAR REMODELING
#332ADAMTS14 INHIBITION
#333FACTOR XII (F12) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#334INHIBITION OF NEUROFIBRILLARY TANGLES USING OLIGONUCLEOTIDES AGAINST CIRCULAR RNAS FROM THE MICROTUBULE ASSOCIATED PROTEIN TAU (MAPT) LOCUS
#335COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION
#336Muscle targeting complexes and uses thereof for treating myotonic dystrophy
#337COMPOSITIONS AND METHODS FOR GENERATING SYNTHETIC LETHALITY IN TUMORS
#338RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
#339Nucleic Acids and Methods for the Treatment of Pompe Disease
#340TREATMENT OF MST1R RELATED DISEASES AND DISORDERS
#341BIOLOGICALLY STABLE XNAZYME THAT EFFICIENTLY SILENCES GENE EXPRESSION IN CELLS
#342RNA THERAPEUTICS AND METHODS OF USE THEREOF
#343IONIZABLE CATIONIC LIPIDS FOR RNA DELIVERY
#344METHODS OF PRODUCING HUMAN MILK OLIGOSACCHARIDES AND COMPOSITIONS THEREOF
#345COMPOSITIONS FOR AND METHODS OF TREATING AND/OR PREVENTING GLUTARIC ACIDURIA TYPE-1
#346HSD17B13-RELATED DOUBLE STRANDED OLIGONUCLEOTIDE COMPOSITIONS AND METHODS RELATING THERETO
#347Oligonucleotides
#348CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#349PLANT VECTORS, COMPOSITIONS AND USES RELATING THERETO
#350Antisense Oligonucleotide (ASO) Gene Inhibition and Treatment
#351COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#352NUCLEIC ACID TARGETING PROPROTEIN CONVERTASE SUBTILISIN/KEXIN 9 AND USES THEREOF
#353Treatment of Liver Diseases With Cell Death Inducing DFFA Like Effector B (CIDEB) Inhibitors
#354TREATMENT AND DETECTION OF INHERITED NEUROPATHIES AND ASSOCIATED DISORDERS
#355TKTL1 INHIBITORS FOR ANTIVIRAL THERAPY
#356USE OF microRNA MIMICS TO INHIBIT OR TREAT LIVER DISEASE
#357USE OF SPLICE SWITCHING OLIGONUCLEOTIDES FOR EXON SKIPPING-MEDIATED KNOCKDOWN OF PIM2
#358SELECTIVE INHIBITORS OF ROCK2 FOR THE TREATMENT OF MUSCULAR DYSTROPHY
#359SYNTHETIC RNAS AND USES THEREOF
#360EXTRA-HEPATIC DELIVERY IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#361RENAL INJURY BIOMARKERS AS BIOMARKERS FOR ACUTE HEPATIC PORPHYRIA (AHP)
#362Compounds and methods for reducing LRRK2 expression
#363EXTRAHEPATIC DELIVERY
#364METHODS AND PHARMACEUTICAL COMPOSITIONS FOR TREATING LYMPHOID MALIGNANCY
#365RNA TARGETING OF MUTATIONS VIA SUPPRESSOR tRNAs AND DEAMINASES
#366Modified Short Interfering Nucleic Acid (siNA) Molecules and Uses Thereof
#367CONJUGATE
#368METHODS FOR MODULATING THE REGENERATIVE PHENOTYPE IN MAMMALIAN CELLS
#369DENDRITIC CONJUGATES FOR THE SKIN DELIVERY OF THERAPEUTIC OLIGONUCLEOTIDES
#370TREATMENT FOR TYROSINE DEGRADATION-ASSOCIATED DISORDERS
#371Products and Methods for Treatment of Amyotrophic Lateral Sclerosis
#372METHODS AND COMPOSITIONS FOR TREATING SUBJECTS HAVING OR AT RISK OF DEVELOPING A NON-PRIMARY HYPEROXALURIA DISEASE OR DISORDER
#373TARGETING 3-KETODIHYDROSPHINGOSINE REDUCTASE (KDSR) IN CANCER
#374Modulation of gene transcription using antisense oligonucleotides targeting regulatory RNAs
#375DNAZYMES TARGETING CELL WALL SYNTHESIS ENZYMES AND USES THEREOF
#376INHIBITORS OF EXPRESSION AND/OR FUNCTION
#3772'-MODIFIED NUCLEOSIDE BASED OLIGONUCLEOTIDE PRODRUGS
#378Compounds and Methods for Reducing DMPK Expression
#379A NON-CANONICAL SIGNALING ACTIVITY OF CGAMP TRIGGERS DNA DAMAGE RESPONSE SIGNALING
#380COMPOSITIONS AND METHODS FOR TREATMENT OF GAUCHER DISEASE
#381Method for Identification of Sensitivity of a Patient to Telomerase Inhibition Therapy
#382PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#383MODULATION OF PREKALLIKREIN (PKK) EXPRESSION
#384GENE THERAPY TREATMENT OF ATRIAL FIBRILLATION
#385NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS
#386MITOCHONDRIAL SUPERCOMPLEX FORMATION PROMOTER, COMPOSITION FOR MAINTAINING OR IMPROVING MUSCULAR STRENGTH, THERAPEUTIC OR PREVENTATIVE PHARMACEUTICAL COMPOSITION FOR DISEASES WHICH DECREASE MITOCHONDRIAL FUNCTION OR MUSCULAR FUNCTION, AND SCREENING METHOD FOR SUBSTANCES WHICH CONTRIBUTE TO FORMATION OF MITOCHONDRIAL SUPERCOMPLEX
#387TRACKING APOBEC MUTATIONAL SIGNATURES IN TUMOR CELLS
#388TMPRSS6 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#389COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#390COMPOSITIONS AND METHODS RELATED TO NUCLEIC ACID ANTICOAGULANTS AND ANTIDOTES
#391METABOLIC DISORDER-ASSOCIATED TARGET GENE IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#392OPTICAL TRANSMISSION SAMPLE HOLDER AND ANALYSIS, PARTICULARLY FOR HEMOGLOBIN
#393Inhibition of Diacylglycerol Kinase to Augment Adoptive T cell Transfer
#394Treatment for Skeletal Diseases Caused by Intracellular Protein Trafficking Defects
#395LEUCINE-RICH REPEAT KINASE 2 (LRRK2) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#396METHODS AND COMPOSITIONS FOR TREATING A PROPROTEIN CONVERTASE SUBTILISIN KEXIN (PCSK9) GENE-ASSOCIATED DISORDER
#397NATURAL KILLER CELLS ENGINEERED TO REDUCE OR ELIMINATE CBL-B AND USES THEREOF
#398METHODS AND COMPOSITIONS TO TREAT HUNTINGTON'S DISEASE BY TARGETING ALOX5- MEDIATED FERROPTOSIS
#399MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING POMPE DISEASE
#400MODULATION OF NOX4 EXPRESSION
#401Modulating Human Tyrosine Hydroxylase Expression Through Control of Specific G-Quadruplex Formation
#402FIDGETIN-LIKE 2 AS A TARGET TO ENHANCE WOUND HEALING
#403COMPOSITIONS FOR TREATING AND/OR PREVENTING CELL OR TISSUE NECROSIS SPECIFICALLY TARGETING CATHEPSIN C AND/OR CELA1 AND/OR CELA3A AND/OR STRUCTURALLY RELATED ENZYMES THERETO
#404Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of use thereof to target dystrophin and to treat Duchenne muscular dystrophy
#405COMPOSITIONS AND METHODS FOR TREATING CANCER
#406GLUCOKINASE (GCK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#407THERAPEUTIC TREATMENT OF TUMORS BY BLOCKING THE HEME SYNTHESIS-EXPORT AXIS
#408Conjugated antisense compounds and their use
#409POLYNUCLEIC ACID MOLECULES TARGETING PCSK9 AND USES THEREOF
#410NANOPARTICLE COMPRISING PEPTIDE-LIPID CONJUGATE FOR DELIVERING OLIGONUCLEOTIDE INTO TARGET CELL AND PHARMACEUTICAL COMPOSITION COMPRISING SAME
#411COMPOSITION FOR PREVENTING OR TREATING OF ISCHEMIC CEREBROVASCULAR DISEASE COMPRISING AN ARGINASE-1 INHIBITOR
#412NOVEL TARGETS FOR ENHANCING ANTI-TUMOR IMMUNITY
#413GENE EDITING SYSTEMS COMPRISING AN RNA GUIDE TARGETING HYDROXYACID OXIDASE 1 (HAO1) AND USES THEREOF
#414COMPOSITIONS AND METHODS FOR SILENCING CARBONIC ANHYDRASE 2 EXPRESSION
#415MODULATION OF COASY EXPRESSION
#416COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#417GENETICALLY MODIFIED CELL LINES FOR METABOLIC STUDIES
#418GENETICALLY MODIFIED CELL LINES FOR METABOLIC STUDIES
#419Modulators of Complement Factor B
#420Treatment Of Stroke With 1,4-Alpha-Glucan-Branching Enzyme (GBE1) Inhibitors
#421METHODS OF USING USP15 INHIBITORS
#422ANTISENSE OLIGONUCLEOTIDES TARGETING ADENOSINE KINASE
#423SIRNA TARGETING TMPRSS6 FOR THE TREATMENT OF MYELOPROLIFERATIVE DISORDERS
#424RNase Inhibitors
#425ALLELE-SPECIFIC SILENCING THERAPY FOR DYNAMIN 2-RELATED DISEASES
#426GENOME EDITING COMPOSITIONS AND METHODS FOR TREATMENT OF WILSON'S DISEASE
#427SIRNA TARGETING 17Beta-HYDROXYSTEROID DEHYDROGENASE TYPE 13 AND SIRNA CONJUGATE
#428COMPOSITION FOR TREATING DIABETIC RETINOPATHY CONTAINING scAAV2-shmTOR-SD (CRG-01)
#429METHOD OF TREATING BREAST CANCER IN A SUBJECT BY INHIBITING TUBB2B
#430Gene Vector
#431COMPOSITIONS FOR TREATING HEART DISEASE BY INHIBITING THE ACTION OF mAKAP-beta
#432METHODS AND USES OF PROTEIN DISULFIDE ISOMERASE INHIBITORY COMPOUNDS
#433iRNA COMPOSITIONS AND METHODS FOR SILENCING CHITINASE 3-LIKE PROTEIN 1/YKL-40 (CHI3L1/YKL-40) PROTEIN
#434ELONGATION OF VERY LONG CHAIN FATTY ACIDS PROTEIN 1 (ELOVL1) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#435PROLINE DEHYDROGENASE 2 (PRODH2) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#436COMPOSITIONS AND METHODS FOR TREATING CANCER
#437STEAROYL-COA DESATURASE 5 (SCD5) IRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#438NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#439Muscle targeting complexes and uses thereof for treating myotonic dystrophy
#440METHODS OF TREATING MYOTONIC DYSTROPHY TYPE 1 USING PEPTIDE-OLIGONUCLEOTIDE CONJUGATES
#441TRANSMEMBRANE PROTEASE, SERINE 6 (TMPRSS6) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#442OLIGONUCLEOTIDES TARGETING S6K1
#443TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
#444KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#445METHODS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF LDHA
#446Compositions and Methods for Treatment of Myotonic Dystrophy Type 1 with CRISPR/SACAS9
#447OPTICAL TRANSMISSION SAMPLE HOLDER AND ANALYSIS, PARTICULARLY FOR HEMOGLOBIN
#448COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF PCSK9
#449Ultrasound Responsive Nanobubble Compositions and Methods of Production and Use Thereof
#450LOSS OF LIPID KINASE PI5P4K GAMMA RESTRICTS TUMOR GROWTH
#451ANTISENSE OLIGONUCLEOTIDE
#452COMPOSITIONS AND METHODS FOR TREATING CANCER AND BIOMARKERS TO DETECT CANCER STEM CELL REPROGRAMMING AND PROGRESSION
#453NON-VIRAL GENE/CARRIER COMPLEX FOR PREVENTION OR TREATMENT OF ACUTE INFLAMMATORY DISEASE
#454ANTISENSE OLIGONUCLEOTIDES FOR THEIR USE IN AN ANTI-CANCER TREATMENT
#455KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#456RNase Inhibitors
#457KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#458METHODS FOR THE TREATMENT OF NASH WITH ADVANCED FIBROSIS AND/OR CIRRHOSIS
#459SUPEROXIDE DISMUTASE 1 (SOD1) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING SUPEROXIDE DISMUTASE 1- (SOD1-) ASSOCIATED NEURODEGENERATIVE DISEASES
#460OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF MAKING THE SAME
#461Lipid formulations for delivery of therapeutic agents
#462TREATING DISORDERS ASSOCIATED WITH DYRK1A DYSFUNCTION
#463PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#464GENE CIRCUIT, RNA DELIVERY SYSTEM AND USE THEREOF
#465METHODS AND COMPOSITIONS FOR MODULATING PLASMINOGEN
#466Driving Axon Regeneration by Activating STAT1 Signaling and cGAS-STING Pathway
#467MODULATION OF GYS1 EXPRESSION
#468HSD17B13 VARIANTS AND USES THEREOF
#469COMPOUND AND METHOD FOR AN ALLELE-SPECIFIC EDITING OF THE ELANE GENE
#470Factor XII (F12) iRNA compositions and methods of use thereof
#471GLYCOGEN SYNTHASE KINASE 3 ALPHA (GSK3 ALPHA) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#472OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#473METHODS FOR MODULATING A SRC-1 CONDENSATE
#474COMPOSITIONS FOR USE IN THE TREATMENT OF CHD2 HAPLOINSUFFICIENCY AND METHODS OF IDENTIFYING SAME
#475COMPOSITIONS AND METHODS FOR THE TARGETING OF PCSK9
#476COMPOSITIONS AND METHODS FOR THE TARGETING OF PCSK9
#477COMPOSITION AND METHOD FOR REDUCING OXALATE LEVELS IN PATIENTS RECEIVING MAINTENANCE DIALYSIS
#478METHODS AND COMPOSITIONS FOR DIAGNOSING AND TREATING PROSTATE CANCER BASED ON LONG NONCODING RNA OVERLAPPING THE LCK GENE THAT REGULATES PROSTATE CANCER CELL GROWTH
#479REGULATION OF METABOLISM AND OBESITY BY MITOCHONDRIAL MUL1 E3 UBIQUITIN LIGASE
#480MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR TREATING MYOTONIC DYSTROPHY
#481LIPOPROTEIN-MIMICKING SOLID LIPID NANOPARTICLES FOR DRUG DELIVERY AND USES THEREOF
#482COMPOSITIONS AND METHODS FOR MODULATING EXPRESSION OF GENES
#483A JMJD6 TARGETING AGENT FOR TREATING PROSTATE CANCER
#484PREVENTION AND/OR TREATMENT OF INFLAMMATORY SKIN DISEASE
#485COMPOSITIONS AND METHODS FOR INHIBITION OF EXPRESSION OF APOLIPOPROTEIN C-III (APOC3) GENES
#486METHODS FOR TREATING AND AMELIORATING CANCER
#487METHODS AND COMPOSITIONS RELATED TO T-CELL ACTIVITY
#488OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#489OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#490NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS
#491Method of Treating Corneal Opacities and Scarring
#492COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#493COMPOSITIONS AND METHODS FOR IMPROVING IMMUNE SYSTEM FUNCTION
#494COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE PCSK9 GENE
#495MODIFICATIONS OF MAMMALIAN CELLS USING ARTIFICIAL MICRO-RNA TO ALTER THEIR PROPERTIES AND THE COMPOSITIONS OF THEIR PRODUCTS
#496METHODS OF MODULATING NEURONAL AND OLIGODENDROCYTE SURVIVAL
#497COMPOSITIONS FOR THE TREATMENT OF EBV ASSOCIATED DISEASES OR CONDITIONS
#498OLIGONUCLEOTIDES FOR INDUCING PATERNAL UBE3A EXPRESSION
#499MODULATION OF CHITINASE PROTEIN EXPRESSION
#500FIBROTIC TREATMENT
#501ANTISENSE OLIGOMERS TARGETING PCSK9
#502METHODS AND COMPOSITIONS UTILIZING IDO1-DEPENDENT VASCULARIZING CELLS FOR THE TREATMENT OF PATHOLOGICAL CONDITIONS INVOLVING NEOVASCULARIZATION
#503COMPOUNDS AND METHODS FOR REDUCING ATXN3 EXPRESSION
#504MODULATORS OF PNPLA3 EXPRESSION
#505MODULATORS OF PNPLA3 EXPRESSION
#506Treatment Of Decreased Bone Mineral Density With Zinc And Ring Finger 3 (ZNRF3) Inhibitors
#507Oligonucleotides reducing the amount of CD73 mRNA and CD73 protein expression
#508RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS
#509FACTOR XII (HAGEMAN FACTOR) (F12), KALLIKREIN B, PLASMA (FLETCHER FACTOR) 1 (KLKB1), AND KININOGEN 1 (KNG1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#510ENGINEERED NK CELLS AND METHODS OF TREATING CANCER
#511METHODS AND COMPOSITIONS FOR CELL-PROLIFERATION-RELATED DISORDERS
#512Compositions and methods for producing tobacco plants and products having altered alkaloid levels with desirable leaf quality via manipulating leaf quality genes
#513METHOD OF ALLELE SPECIFIC SILENCING FOR THE TREATMENT OF AUTOSOMAL DOMINANT CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA (CPVT)
#514Oligonucleotide for reducing the expression of angiotensin-converting enzyme 2 (ACE2) and its use for treating viral infection
#515DIPEPTIDYL PEPTIDASE 4 (DPP4) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#516Muscle targeting complexes and uses thereof for treating dystrophinopathies
#517Lipid compositions
#518COMPOSITIONS AND METHODS FOR CHARACTERIZING AND TREATING DISEASES AND DISORDERS ASSOCIATED WITH MULTIPLE ORGAN FAILURE
#519ANTISENSE OLIGONUCLEOTIDES AND THEIR USE FOR THE TREATMENT OF PAIN
#520COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
#521FN3 Domain-siRNA Conjugates with Enzyme Replacement Therapy
#522TREATMENT FOR LYSINE DEGRADATION-ASSOCIATED DISORDERS
#523MUSCLE RETENTION IN AGING AND DUCHENNE MUSCULAR DYSTROPHY (DMD) THROUGH S1P INHIBITION
#524PREVENTION AND TREATMENT OF AGE-RELATED MACULAR DEGENERATION THROUGH SUPPRESSION OF CATHEPSIN S EXPRESSION
#525COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
#526RNAi agents for inhibiting expression of Superoxide Dismutase 1 (SOD1), compositions thereof, and methods of use
#527METHOD AND COMPOSITION FOR A TARGETED GENE KNOCKOUT
#528METHODS OF TREATING CANCER USING LSD1 INHIBITORS IN COMBINATION WITH IMMUNOTHERAPY
#529INHIBITION OF NGLY1 FOR THE TREATMENT OF CANCER
#530Method for preparing compound or biological drug enhancing CNPase activity for treating heart diseases
#531Extrahepatic delivery
#532DOUBLE STRANDED RNA TARGETING 17-BETA HYDROXYSTEROIDDEHYDROGENASE 13 (HSD17B13) AND METHODS OF USE THEREOF
#533ANTISENSE NUCLEIC ACID AND USE THEREOF
#534MEANS AND METHODS FOR MODIFYING MULTIPLE ALLELES
#535DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF LRRK2
#536APPROACH FOR TREATMENT OF CANCER VIA IMMUNOMODULATION BY USING TALABOSTAT
#537SOD1 dual expression vectors and uses thereof
#538MODULATION OF GENE EXPRESSION AND SCREENING FOR DEREGULATED PROTEIN EXPRESSION
#539RECOMBINANT MICROORGANISM HAVING IMPROVED ABILITY TO PRODUCE RECOMBINANT SILK PROTEIN AND METHOD FOR PRODUCING HIGH-MOLECULAR-WEIGHT RECOMBINANT SILK PROTEIN BY USING SAME
#540SINA MOLECULES, METHODS OF PRODUCTION AND USES THEREOF
#541SIRNA FOR TREATING HEPATIC FIBROSIS AND DELIVERY PREPARATION THEREOF
#542CHEMICAL MODIFICATIONS FOR INHIBITING EXPRESSION OF ALDH2
#543MATERIALS AND METHODS FOR TREATING CORNEAL DYSFUNCTION
#544CATALYTIC SEQUENCE BASED METHODS OF TREATING OR PREVENTING BACTERIAL INFECTIONS
#545APPLICATION OF HELZ2 IN DRUGS FOR PREVENTING AND TREATING TUMORS
#546KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#547METHODS AND COMPOSITIONS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#548NUCLEIC ACIDS CONTAINING ABASIC NUCLEOTIDES
#549TREATMENT OF OPTIC ATROPHY
#550ANTISENSE OLIGOMERS FOR TREATMENT OF DISEASE
#551IRON OXIDE NANOPARTICLE FOR SUPPRESSING DRUG-RESISTANT GENE FOR THE TREATMENT OF GLIOBLASTOMA
#552EXOSOMAL LOADING USING HYDROPHOBICALLY MODIFIED OLIGONUCLEOTIDES
#553COMPOSITIONS AND METHODS OF TREATING MUSCLE DYSTROPHY
#554METHODS AND COMPOSITIONS FOR TREATING PRIMARY HYPEROXALURIA
#555MAMMALIAN CELLS FOR PRODUCING A SECRETED PROTEIN
#556METHODS FOR PRODUCTION OF MSC-DERIVED EXOSOMES
#557SIRNA, MEDICAL COMPOSITIONS, AND METHODS FOR TREATING DIABETES USING THE SAME
#558Methods and Compositions for Reducing Immunosupression by Tumor Cells
#559NOVEL TARGETS FOR REACTIVATION OF PRADER-WILLI SYNDROME-ASSOCIATED GENES
#560MODIFIED GAPMER OLIGOMERS AND METHODS OF USE THEREOF
#561TARGETING XIST AND RNA METHYLATION FOR X REACTIVATION THERAPY
#562COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISEASE TYPE 1A
#563MARF/MFN MODULATORS AND USES THEREOF
#564MEANS AND METHODS FOR MODIFYING MULTIPLE ALLELES
#565BASE EDITING ENZYMES
#566COMPOSITIONS AND METHODS FOR TARGETING CORONAVIRUS USING LIPID VESICLES INCLUDING EXOSOMES
#567Methods of treating cholangiocarcinoma
#568SIRT1 INHIBITOR OR ANTAGONIST FOR USE IN PREVENTING AND/OR TREATING AN HPV INDUCED TUMOUR
#569LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#570COMPOSITIONS AND METHODS FOR INHIBITING GYS2 EXPRESSION
#571COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR B EXPRESSION
#572Methods of activating cytotoxic leukocytes using PTP1B and PTPN2 inhibitors
#573COMPOSITIONS AND METHODS FOR TREATMENT OF ACUTE LIVER FAILURE
#574METHODS TO RE-ENGAGE A FETAL WOUND HEALING PATHWAY FOR ADULT SKIN REPAIR
#575BASE EDITING ENZYMES
#576COMPOSITIONS AND METHODS FOR INHIBITING MITOCHONDRIA AMIDOXIME REDUCING COMPONENT 1 (MARC1) EXPRESSION
#577COMPOSITIONS AND METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS) WITH AAV-MIR-SOD1
#578TREATMENT OF SKIN DISEASES AND DISORDERS USING INHIBITORS OF KALLIKREIN-RELATED PEPTIDASES (KLK)
#579COMPOSITIONS AND METHODS FOR THE TREATMENT OF METABOLIC SYNDROME
#580IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF MONOGENIC DISEASES, INCLUDING MUCOPOLYSACCHARIDOSIS TYPE 1H AND OTHER DISORDERS
#581ANAPLASTIC LYMPHOMA KINASE (ALK) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#582METHODS OF TREATMENT OF INFLAMMATORY BOWEL DISEASES
#583METHODS FOR THE TREATMENT OF LUNG TUMORS
#584CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#585shRNA TARGETING UBE3A-ATS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
#586Compositions and methods of treating amyotrophic lateral sclerosis (ALS)
#587PHARMACEUTICAL COMPOSITION CONTAINING SIRT7 INHIBITOR FOR PREVENTING OR TREATING ALLERGIC DISEASES
#588shRNA TARGETING SNORD115 LOCATIONS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN ANGELMAN SYNDROME
#589COMPOSITIONS AND METHODS OF TREATING POMPE DISEASE
#590VESICLES COMPRISING A PTEN INHIBITOR AND USES OF SAME
#591Ketohexokinase (KHK) iRNA compositions and methods of use thereof
#592COMPLEMENT COMPONENT C1R INHIBITORS FOR TREATING A NEUROLOGICAL DISEASE, AND RELATED COMPOSITIONS, SYSTEMS AND METHODS OF USING SAME
#593Modulators of PCSK9 Expression
#594TARGETING OF LACTATE DEHYDROGENASE C, METHODS OF PREPARING SAME, AND METHODS OF USING SAME IN COMBINATION WITH ANTI-CANCER TREATMENTS
#595OLIGONUCLEOTIDES FOR MLH3 MODULATION
#596IN VIVO LYMPHOVENOUS ANASTOMOSIS
#597COMPOSITIONS AND METHODS FOR TREATING PAIN AND ANXIETY DISORDERS
#598NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#599MODULATORS OF HSD17B13 EXPRESSION
#600MIRNA-485 INHIBITOR FOR HUNTINGTON'S DISEASE