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
MUSCLE REGENERATION AND GROWTH
#602Oligonucleotides for inducing paternal UBE3A expression
#603COMPOSITIONS AND METHODS FOR THE TREATMENT OF DISEASES BY ENHANCING ARGINASE 2 IN MACROPHAGES
#604MODIFICATIONS OF MAMMALIAN CELLS USING ARTIFICIAL MICRO-RNA TO ALTER THEIR PROPERTIES AND THE COMPOSITIONS OF THEIR PRODUCTS
#605TREATMENT AND DETECTION OF INHERITED NEUROPATHIES AND ASSOCIATED DISORDERS
#606USE OF PI3KC2B INHIBITORS FOR THE PRESERVATION OF VASCULAR ENDOTHELIAL CELL BARRIER INTEGRITY
#607BIALLELIC KNOCKOUT OF SARM1
#608VIRAL VECTORS FOR TREATING PARKINSON'S DISEASE
#609Compositions and methods for treatment of sepsis-related disorders
#610COMPOSITIONS AND METHODS FOR TRANSDIFFERENTIATING CELLS
#611Compositions and methods for controlling food intake of animal
#612RNAI CONSTRUCTS FOR INHIBITING HSD17B13 EXPRESSION AND METHODS OF USE THEREOF
#613Oligomeric Compounds Comprising Bicyclic Nucleotides and Uses Thereof
#614METHODS AND COMPOSITIONS FOR THE ACTIVATION OF GAMMA-DELTA T-CELLS
#615ANTISENSE OLIGONUCLEOTIDE INDUCING EXON SKIPPING OF ANGIOTENSIN CONVERTING ENZYME 2 GENE
#616ADAR DEPENDENT EDITING COMPOSITIONS AND METHODS OF USE THEREOF
#617Long Non-Coding RNA-Expression In Fungal Hosts
#618COMPOSITIONS USEFUL FOR TREATMENT OF CHARCOT-MARIE-TOOTH DISEASE
#619Muscle targeting complexes and uses thereof for treating dystrophinopathies
#620Compositions and methods for treating hyperphagia in prader-willi syndrome
#621Compositions and methods for reducing skin fat
#622RNAi Agents for Inhibiting Expression of Matrix Metalloproteinase 7 (MMP7), Compositions Thereof, and Methods of Use
#623THERAPEUTICS FOR SYNGAP HAPLOINSUFFICIENCY
#624MODULATION OF FACTOR 11 EXPRESSION
#625METHOD FOR ENHANCING RECOVERY OF COSMETIC LASER-TREATED SKIN
#626PRODUCTS AND COMPOSITIONS
#627METHODS AND COMPOSITIONS FOR THE ADAR-MEDIATED EDITING OF ARGININOSUCCINATE SYNTHETASE (ASS1)
#628METHODS FOR PROPHYLACTIC AND THERAPEUTIC TREATMENT OF 2019-nCoV USING siRNAs AGAINST TGFB1 AND COX2
#629METHODS FOR TREATING GLIOBLASTOMA
#630PCSK9 TARGETING OLIGONUCLEOTIDES FOR TREATING HYPERCHOLESTEROLEMIA AND RELATED CONDITIONS
#631Treatment of uveitis with endoplasmic reticulum aminopeptidase 1 (ERAP1) inhibitors
#632METHODS AND COMPOSITIONS FOR TREATMENT OF APC-DEFICIENT CANCER
#633siRNA-Copolymer Compositions And Methods Of Use For Treatment Of Liver Cancer
#634Lipid formulations for delivery of therapeutic agents
#635METHOD AND DRUG FOR TREATING HURLER SYNDROME
#636SMALL INTERFERING DEOXYRIBONUCLEIC ACID (SIDNA) AGAINST METALLO-BETA LACTAMASE GENE
#637Composition, Comprising Rip Kinase Inhibitor, For Preventing Hair Loss Or Promoting Hair Regrowth
#638CANCER THERAPEUTIC COMPOSITIONS AND METHODS TARGETING DNASE1L3
#639Transmembrane protease, serine 6 (TMPRSS6) iRNA compositions and methods of use thereof
#640METHODS AND COMPOSITIONS FOR TREATMENT OF AGE-RELATED MACULAR DEGENERATION
#641ANTISENSE THERAPEUTICS FOR THE TREATMENT OF CORONAVIRUS
#642NUCLEIC ACID MOLECULES FOR REDUCTION OF PAPD5 OR PAPD7 MRNA FOR TREATING HEPATITIS B INFECTION
#643ISOTHERMAL METHODS, COMPOSITIONS, KITS, AND SYSTEMS FOR DETECTING NUCLEIC ACIDS
#644SINGLE-STRANDED OLIGONUCLEOTIDE
#645DYNAMIN 2 INHIBITOR FOR THE TREATMENT OF CENTRONUCLEAR MYOPATHIES
#646SIRNA COMPOSITIONS AND METHODS FOR SILENCING GPAM (GLYCEROL-3-PHOSPHATE ACYLTRANSFERASE 1, MITOCHONDRIAL) EXPRESSION
#647FIDGETIN-LIKE 2 AS A TARGET TO ENHANCE SKIN GRAFT HEALING
#648MUSCLE TARGETING COMPLEXES AND USES THEREOF FOR MODULATION OF GENES ASSOCIATED WITH MUSCLE HEALTH
#649COMPOSITIONS AND METHODS FOR TREATING VIRUS INFECTION
#650OLIGONUCLEOTIDES FOR SOD1 MODULATION
#651METHOD FOR TREATING CYCLOPHILIN A ASSOCIATED DISEASES
#652Compositions and Methods for Modifying Target RNAs
#653MULTI-TARGETED siRNA FOR TREATING CANCERS
#654NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#655Composition of drug targets and method of using thereof
#656Human antibodies to the human glucagon receptor (HGCGR) and methods of use thereof to lower blood glucose or ketone levels
#657COMPOSITIONS AND METHODS FOR FUNGAL INHIBITION USING MINICELL-BASED RNAi
#658COMPOSITIONS FOR TRANSFER OF CARGO TO CELLS
#659COMPOSITIONS AND METHODS FOR INHIBITING MARC1 GENE EXPRESSION
#660COMPOSITIONS AND METHODS FOR TREATING SUBJECTS HAVING A HETEROZYGOUS ALANINE-GLYOXYLATE AMINOTRANSFERASE GENE (AGXT) VARIANT
#661AMINE-CONTAINING TRANSFECTION REAGENTS AND METHODS FOR MAKING AND USING SAME
#662COMPOSITIONS AND METHODS FOR TREATING NEOPLASIA
#663Modulation of Dystrophia Myotonica-Protein Kinase (DMPK) Expression
#664Guide RNA that targets a mutant human inosine monophosphate deyhydrogenase I allele
#665Guide RNA that targets a mutant human guanylate cyclase 2A allele
#666COMPOSITIONS AND METHODS FOR TREATMENT OF FAMILIAL HYPERCHOLESTEROLEMIA AND ELEVATED LOW-DENSITY LIPOPROTEIN CHOLESTEROL
#667Manipulating Glycogen in Alzheimer’s Disease, Epilepsy, Traumatic Brain Injury, and ALS as a Treatment
#668COMBINATION THERAPY OF LYMPHOMA
#669BIFUNCTIONAL MOLECULES AND METHODS OF USING THEREOF
#670DEPLETION OF EXT1 EXPRESSION AND/OR ACTIVITY IMPROVES CELLULAR PRODUCTION OF BIOLOGICAL ENTITIES
#671Hexokinase 2-specific inhibitors for use in acute central nervous system injury
#672METHODS AND COMPOSITIONS FOR TREATING INFLAMMATORY CONDITIONS ASSOCIATED WITH INFECTIOUS DISEASE
#673TARGETING THE PALMOTYLATION/DEPALMOTYLATION CYCLE TO TREAT INFLAMMATORY DISEASES
#674EXPRESSION OF FC-CONTAINING PROTEINS
#675COMPOUND COMPRISING A NUCLEIC ACID AND A HALF-LIFE EXTENSION MOTIF
#676EXTRACELLULAR VESICLE SECRETION REDUCING AGENT FOR REDUCING EXTRACELLULAR VESICLE SECRETION, AND USE OF THE SAME
#677ARTC1 LIGANDS FOR CANCER TREATMENT
#678COMPOSITIONS AND METHODS FOR TREATING AND PREVENTING PREKALLIKREIN-ASSOCIATED CONDITIONS
#679METHODS FOR ENHANCING VASCULAR DENSITY
#680METHODS AND COMPOSITIONS FOR TREATING ATHEROSCLEROSIS
#681MULTIMERIC OLIGONUCLEOTIDES WITH DIVIDED STRANDS
#682COMPOSITIONS AND METHODS FOR INHIBITION AND TARGETING OF P97
#683METHODS AND COMPOSITIONS FOR INACTIVATING INTERLEUKIN-2-INDUCIBLE T-CELL KINASE (ITK)
#684COMPOSITION AND METHODS FOR IMPROVING HEART FUNCTION AND TREATING HEART FAILURE
#685CATIONIC LIPIDS FOR LIPID NANOPARTICLE DELIVERY OF THERAPEUTICS TO HEPATIC STELLATE CELLS
#686SiRNA molecule inhibiting the expression of the PCSK9 gene and use thereof
#687RNAi Agents for Inhibiting Expression of Xanthine Dehydrogenase (XDH), Pharmaceutical Compositions Thereof, and Methods of Use
#688METHODS FOR TREATING HYPERCHOLESTEROLEMIA
#689ANGIOTENSIN-CONVERTING ENZYME 2 (ACE2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#690LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#691MIRNA-485 INHIBITOR FOR GENE UPREGULATION
#692MANNAN BINDING LECTIN SERINE PEPTIDASE 2 (MASP2) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#693RNAi Agents for Inhibiting Expression of Xanthine Dehydrogenase (XDH), Pharmaceutical Compositions Thereof, and Methods of Use
#694N6-Methyladenosine Regulators in Uterine Fibroids
#695METHODS AND COMPOSITIONS FOR TREATMENT OF ATHEROSCLEROSIS
#696USP10 TARGETED SELF-DELIVERABLE SIRNA COMPOSITIONS AND METHODS FOR PREVENTING OR INHIBITING FIBROSIS AND/OR SCARRING
#697USE OF COPS3 INHIBITORS FOR TREATING HEPATITIS B VIRUS INFECTION
#698COMPOSITIONS AND METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#699DIAGNOSTIC METHODS USING PCG-1A EXPRESSION
#700MUSCLE-TARGETING COMPLEXES AND USES THEREOF IN TREATING MUSCLE ATROPHY
#701NANOPARTICLES FOR EXPRESSION OF GENES OF INTEREST AND/OR REGULATION OF SIGNALING PATHWAYS
#702Compounds and methods for reducing LRRK2 expression
#703Oligomeric compounds for reducing DMPK expression
#704Method for Identification of Sensitivity of a Patient to Telomerase Inhibition Therapy
#705Nucleic acid molecule for reduction of PAPD5 and PAPD7 mRNA for treating hepatitis B infection
#706GENETICALLY MODIFIED CLOSTRIDIUM BACTERIA, PREPARATION AND USES OF SAME
#707Methods Of Treating Chronic Kidney Disease (CKD) With Inhibitors Of Protective Loss-Of-Function Genes
#708CONDITIONAL-siRNAS AND USES THEREOF IN TREATING CARDIAC HYPERTROPHY
#709METHODS AND COMPOSITIONS FOR TREATING A PROPROTEIN CONVERTASE SUBTILISIN KEXIN (PCSK9) GENE-ASSOCIATED DISORDER
#710NANOMETRIC PHARMACEUTICAL COMPOSITION IN THE FORM OF LIPOSOMES OR NANOEMULSION CONTAINING SPECIFIC SEQUENCES OF INTERFERENCE RNA
#711COMPOSITIONS AND METHODS TO TREAT NEUROLOGICAL DISEASES
#712ANTI-ADAM33 OLIGONUCLEOTIDES AND RELATED METHODS
#713Methods of treating cancers having a BRCA1 and/or BRCA2 mutation(s)
#714COMPOSITIONS AND METHODS FOR INHIBITING ASAH1 GENE EXPRESSION
#715DRUG FOR CURATIVE THERAPY OF INTRACTABLE HEREDITARY RENAL ALPORT SYNDROME
#716ANTISENSE OLIGOMERS AND METHODS FOR TREATING PARKIN-RELATED PATHOLOGIES
#717SHORT HAIRPIN RNA (SHRNA734) AND USE OF SAME TO POSITIVELY SELECT AND ELIMINATE GENETICALLY MODIFIED CELLS
#718COMPOSITIONS AND METHODS FOR TREATING OR AMELIORATING A MYCOBACTERIUM TUBERCULOSIS INFECTION
#719METHODS AND USES FOR PROPROTEIN CONVERTASE SUBTILISIN KEXIN 9 (PCSK9) INHIBITORS
#720FORMULATIONS FOR DELIVERY OF OLIGONUCLEOTIDES TO LUNG CELLS
#721Compositions and methods for modulating complement factor B expression
#722ATAXIN3 (ATXN3) RNAI AGENT COMPOSITIONS AND METHODS OF USE THEREOF
#723NOVEL DRUGGABLE TARGETS FOR THE TREATMENT OF INFLAMMATORY DISEASES SUCH AS SYSTEMIC LUPUS ERYTHEMATOSUS (SLE) AND METHODS FOR DIAGNOSIS AND TREATMENT USING THE SAME
#724PLATELETS TRANSFECTED BY EXOGENOUS GENETIC MATERIAL AND PLATELET MICROPARTICLES OBTAINED BY SAID TRANSFECTED PLATELETS, METHOD FOR THE PREPARATION AND USES THEREOF
#725Modulators of diacyglycerol acyltransferase 2 (DGAT2)
#726COMPOSITIONS AND METHODS FOR HEMOGLOBIN PRODUCTION
#727Compositions and methods for inhibiting mitochondria amidoxime reducing component 1 (MARC1) expression
#728Methods and Compositions for Modulationg Heterochromatin Dysfunction, Genomic Instability, and Associate Conditions
#729METHODS FOR THE TREATMENT OF TRINUCLEOTIDE REPEAT EXAPNSION DISORDERS ASSOCIATED WITH OGG1 ACTIVITY
#730COMPOSITIONS AND METHODS FOR SILENCING DNAJB1-PRKACA FUSION GENE EXPRESSION
#731INHIBITORS OF KDM5A FOR USE IN TREATMENT OF IDIOPATHIC INFLAMMATORY MYOPATHIES
#732HSD17B13 VARIANTS AND USES THEREOF
#733COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#734RNAi-BASED TARGETING COMPOUNDS AND USES THEREOF TO PREVENT ACQUIRED HEARING LOSS
#735Oligonucleotides for SARS-CoV-2 modulation
#736INHIBITING THE RNA METHYLTRANSFERASE METTL3 OR ITS INTERACTION WITH EIF3H TO SUPPRESS ONCOGENE TRANSLATION AND TUMORIGENESIS
#737AN UBIQUITIN LIGASE INHIBITOR FOR USE FOR PREVENTING AND/OR TREATING A DISEASE LINKED WITH CEREBRAL HYPOPERFUSION
#738ANTISENSE OLIGONUCLEOTIDES AND THIER USE FOR THE TREATMENT OF CANCER
#739METHODS AND COMPOSITIONS TO INCREASE SOMATIC CELL NUCLEAR TRANSFER (SCNT) EFFICIENCY BY REMOVING HISTONE H3-LYSINE TRIMETHYLATION
#740METHODS AND COMPOSTIONS FOR INHIBITING P97
#741METHODS AND COMPOSTIONS FOR INHIBITING CORONAVIRAL REPLICATION
#742RNAi agents for inhibiting expression of xanthine dehydrogenase (XDH), pharmaceutical compositions thereof, and methods of use
#743PCSK9 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#744Compounds and methods for modulating factor XII
#745CELA-1 INHIBITION FOR TREATMENT OF LUNG DISEASE
#746Muscle targeting complexes and uses thereof for treating dystrophinopathies
#747COMPOUNDS AND METHODS OF PROMOTING MYELINATION
#748PLATELETS TRANSFECTED BY EXOGENOUS GENETIC MATERIAL AND PLATELET MICROPARTICLES OBTAINED BY SAID TRANSFECTED PLATELETS, METHOD FOR THE PREPARATION AND USES THEREOF
#749PIKFYVE antisense oligonucleotides
#750ANTISENSE OLIGONUCLEOTIDE SEQUENCES FOR SILENCING THE HUMAN L1-MET TRANSCRIPT IN TUMORS
#751METHODS OF CANCER TREATMENT BY DELIVERY OF siRNAs AGAINST NSD3
#752XANTHINE DEHYDROGENASE (XDH) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#753Compositions and methods of treating muscle dystrophy
#754METHODS FOR INHIBITION OF HAO1 (HYDROXYACID OXIDASE 1 (GLYCOLATE OXIDASE)) GENE EXPRESSION
#755COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF THE ALAS1 GENE
#756THERAPEUTIC INHIBITION OF LACTATE DEHYDROGENASE AND AGENTS THEREFOR
#757CRISPR-RELATED METHODS AND COMPOSITIONS WITH GOVERNING gRNAS
#758CHEMICAL MODIFICATIONS OF SMALL INTERFERING RNA WITH MINIMAL FLUORINE CONTENT
#759COMPOSITIONS AND METHODS FOR SILENCING UGT1A1 GENE EXPRESSION
#760Compositions and Methods for Simultaneously Modulating Expression of Genes
#761Composition for preventing or treating fibrosis including an inhibitor of CDK17 expression or activity
#762RNA interference mediated inhibition of prolyl hydroxylase domain 2 (PHD2) gene expression using short interfering nucleic acid (siNA)
#763Xanthine dehydrogenase (XDH) iRNA compositions and methods of use thereof
#764Nucleus-encoded male sterility through mutation in cytochrome P450 oxidase
#765TREATMENT OF RENAL CYSTIC DISEASE
#766REACTIVATING P53 MUTANTS FOR CANCER TREATMENT BY TARGETING PROLIDASE (PEPD)
#767PPM1A INHIBITORS AND METHODS OF USING SAME
#768COMPOSITIONS AND METHODS FOR TREATING NEURODEGENERATIVE DISORDERS
#769ANTISENSE COMPOUNDS AND USES THEREOF
#770FN3 domain-siRNA conjugates and uses thereof
#771Inhibitor of DJ-1 for Use in Treating Immunoaging
#772BIODEGRADABLE LIPIDS FOR THE DELIVERY OF ACTIVE AGENTS
#773OLIGONUCLEOTIDE COMPOUNDS FOR TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS
#774Methods of treating liver diseases with phosphodiesterase 3B (PDE3B) inhibitors
#775Compositions and methods for modulating PNPLA3 expression
#776Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
#777Base editing enzymes
#778Superoxide dismutase 1 (SOD1) iRNA compositions and methods of use thereof for treating or preventing superoxide dismutase 1-(SOD1-) associated neurodegenerative diseases
#779Cathepsin L gene, dsRNA thereof, and use thereof
#780PHARMACEUTICAL COMPOSITION CONTAINING HERES EXPRESSION INHIBITOR FOR PREVENTING OR TREATING SQUAMOUS CELL CARCINOMA
#781PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#782SUGAR CHAIN-RELATED GENE AND USE THEREOF
#783COMPOSITIONS AND METHODS FOR INHIBITING KETOHEXOKINASE (KHK)
#784METHODS FOR DEPLETION OF DELETERIOUS MITOCHONDRIAL GENOMES
#785TREATMENT OF HR DEFICIENT CANCER
#786RECOMBINANT MICROORGANISMS FOR IN VIVO PRODUCTION OF SULFATED GLYCOSAMINOGLYCANS
#787SiRNA, medical compositions, and methods for treating type ii diabetes using the same
#788INHIBITION OF MTHFD1L FOR USE IN HYPERTROPHIC HEART DISEASE AND HEART FAILURE
#789Nucleic acids for inhibiting expression of TMPRSS6 and iron chelators
#790Method for Identification of Sensitivity of a Patient to Telomerase Inhibition Therapy
#791SMALL INTERFERING RNA (siRNA) FOR INHIBITING THE EXPRESSION OF MINI-CHROMOSOME MAINTENANCE 7 (MCM7) GENE, AND COMPOSITION AND USE THEREOF
#792TREATMENT FOR SOD1 ASSOCIATED DISEASE
#793NUCLEIC ACID, PHARMACEUTICAL COMPOSITION AND CONJUGATE, PREPARATION METHOD THEREFOR AND USE THEREOF
#794Extended dicer substrate agents and methods for the specific inhibition of gene expression
#795Methods and Compositions for Selecting siRNA of Improved Functionality
#796Using fatty acid synthase inhibitors to treat fibrosis
#797INHIBITING APE2 EXPRESSION IN A SUBJECT TREATED WITH A DRUG AGENT THAT CAUSES INCREASED APE2 EXPRESSION
#798Treatment Of Liver Disease With Ring Finger Protein 213 (RNF213) Inhibitors
#799Treatment of liver disease with mitochondrial glycerol-3-phosphate acyltransferase (GPAM) inhibitors
#800INHIBITION OF KMT2D FOR THE TREATMENT OF CANCER
#801USES OF SYNTHETIC LETHAL PARTNERS FOR TREATMENT OF CANCER
#802METHODS OF PREPARING PROTEIN-OLIGONUCLEOTIDE COMPLEXES
#803Oligonucleotide compositions and methods of making the same
#804SIRNA SEQUENCES TARGETING THE EXPRESSION OF HUMAN GENES JAK1 OR JAK3 FOR A THERAPEUTIC USE
#805APPLICATION OF COMBINED INHIBITION OF LNCRNA SAMMSON AND BRAF KINASE IN DELAYING ADAPTIVE DRUG-RESISTANCE OF MELANOMA
#806METHODS OF TREATING CANCERS CONTAINING FUSION GENES
#807COMPOSITIONS AND METHODS FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS
#808COMPOSITIONS AND METHODS FOR INHIBITING PCSK9
#809A Method Of Promoting Survival And/Or Function Of A Motor Neuron And Related Agents, Uses And Methods
#810Antisense oligonucleotides rescue aberrant splicing of ABCA4
#811Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
#812METHODS AND COMPOSITIONS USING AUXOTROPHIC REGULATABLE CELLS
#813Muscle targeting complexes and uses thereof for treating dystrophinopathies
#814METHOD OF MODULATING ADIPOSITY
#815Treatment of liver diseases with cell death inducing DFFA like effector B (CIDEB) inhibitors
#816METHODS OF TREATING P53 MUTANT CANCERS USING OGDH INHIBITORS
#817METHOD AND COMPOSITION FOR TARGETED DELIVERY OF THERAPEUTIC AGENTS
#818Methods And Compositions For Enhancing AAV-Mediated Homologous Recombination Using Ribonucleotide Reductase Inhibitors
#819METHODS AND COMPOSITIONS FOR TREATING MALIGNANT TUMORS ASSOCIATED WITH KRAS MUTATION
#820CONJUGATE AND USES THEREOF
#821USE OF RAB7 GTPASE (RAB7) INHIBITORS IN ENHANCING PERMEABILITY OF THE BLOOD BRAIN BARRIER (BBB)
#822Striatin interacting protein inhibitor and use thereof in preparation of anti-tumor drug
#823ANTISENSE THERAPY FOR PTP1B RELATED CONDITIONS
#824MODULATORS OF HSD17B13 EXPRESSION
#825SiRNA structures for high activity and reduced off target
#826NUCLEIC ACID DRUG TARGETING MURF1
#827DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF RPE65
#828METHODS FOR TREATING RAN PROTEIN-ASSOCIATED NEUROLOGICAL DISEASES
#829CANCER TREATMENT USING TARGETED siRNA PHARMACEUTICAL FORMULATIONS TO DOWNREGULATE EXPRESSION OF PRDM14 PROTEIN
#830HSD17B13 variants and uses thereof
#831HEXOKINASE 1 ISOFORM B FOR USE AS A PROGNOSIS MARKER AND SPECIFIC TARGET AGAINST CANCER
#832INCREASING ATOH1 LIFE TO DRIVE SENSORINEURAL HAIR CELL DIFFERENTIANTION
#833UBE3A ANTISENSE THERAPEUTICS
#834COMPOUNDS AND METHODS FOR MODULATING TMPRSS6 EXPRESSION
#835COMPOSITIONS AND METHODS FOR INHIBITING EXPRESSION OF TMPRSS6 GENE
#836TREATMENT AND PREVENTION OF DISEASE MEDIATED BY WWP2
#837Methods and compositions related to RNA-targeted Rho small GTPase RND3/RhoE therapy
#838COMBINATION VECTORS AND METHODS FOR TREATING CANCER
#839Treatment and detection of inherited neuropathies and associated disorders
#840Modulators of Complement Factor B
#841ANTICANCER COMBINATIONS
#842NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PREPARATION METHOD, AND USE
#843OLIGONUCLEOTIDE THERAPY FOR WOLMAN DISEASE AND CHOLESTERYL ESTER STORAGE DISEASE
#844Carboxypeptidase B2 (CPB2) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#845COMPLEMENT INHIBITION FOR IMPROVED NERVE REGENERATION
#846METHODS AND COMPOSITIONS FOR MODULATION OF AN INTERSPECIES GUT BACTERIAL PATHWAY FOR LEVODOPA METABOLISM
#847METHODS AND COMPOSITIONS OF SHORT SMALL HAIRPIN RNAS AND MICRORNAS FOR WOUND HEALING
#848METHOD FOR INHIBITING INFECTION AND ACTIVATION OF VIRUS
#849REPROGRAMMING OF LIPID METABOLISM TO INHIBIT T CELL SENESCENCE AND ENHANCE TUMOR IMMUNOTHERAPY
#850LIPID OLIGONUCLEOTIDE ANTISENSE AGAINST ANTIBIOTIC RESISTANCE
#851METHODS AND COMPOSITIONS FOR TREATMENT OF NLRP3 INFLAMMASOME MEDIATED IL-1BETA DEPENDENT DISORDERS
#852MODULATORS OF TELOMERE DISEASE
#853Treatment of liver diseases with cell death inducing DFFA like effector B (CIDEB) inhibitors
#854EXTRACELLULAR MATRIX-PRODUCING COMPOSITION USING MAST4 GENE AND PREPARATION METHOD THEREFOR
#855COMPLEMENT COMPONENT iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#856Nucleic acids and methods for the treatment of Pompe disease
#857DUAL TARGETING siRNA AGENTS
#858Bruton's Tyrosine Kinase as Anti-Cancer Drug Target
#859OLIGOMERIC NUCLEIC ACID MOLECULE, AND APPLICATION THEREOF IN AN ACUTE INTERMITTENT PORPHYRIA TREATMENT
#860COMPOSITIONS AND METHODS FOR RAPID AND DYNAMIC FLUX CONTROL USING SYNTHETIC METABOLIC VALVES
#861COMPOSITIONS AND METHODS FOR TREATING RAS-MUTANT CANCERS
#862METHODS AND COMPOSITIONS FOR INHIBITING EXPRESSION OF CYP27A1
#863INHIBITION OF HISTONE METHYL TRANSFERASES TO TREAT CANCER
#864Treatment and diagnosis of inflammatory disorders
#865HIPK INHIBITORS AND METHODS OF USE THEREOF
#866Pharmaceutical Composition for Treating Muscle Disease
#867COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION IN THE CENTRAL NERVOUS SYSTEM
#868ANTISENSE-INDUCED EXON2 INCLUSION IN ACID ALPHA-GLUCOSIDASE
#869Modulation of prekallikrein (PKK) expression
#870TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD
#871Extended dicer substrate agents and methods for the specific inhibition of gene expression
#872Method for stimulating axonal regeneration
#873ANTISENSE NUCLEIC ACID TARGETING PCSK9
#874Methods to identify and treat subjects having corticosteroid-resistant inflammatory diseases
#875COMPOSITIONS AND METHODS FOR THE TREATMENT OF KRAS ASSOCIATED DISEASES OR DISORDERS
#876RSK INHIBITORS IN THE TREATMENT OF VIRUS DISEASES
#877Oligonucleotides for inducing paternal UBE3A expression
#878Muscle targeting complexes and uses thereof for treating dystrophinopathies
#879TARGETED SENSITIZATION OF NON-DEL(5q) MALIGNANT CELLS
#880Compositions for treating heart disease by inhibiting the action of mAKAP-β
#881COMPOSITIONS FOR SUPPRESSING TRIM28 AND USES THEREOF
#882Modulatory polynucleotides
#883KETOHEXOKINASE (KHK) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#884TREATMENT OF CNS INJURY WITH RNAi THERAPEUTICS
#885COMPOSITIONS AND METHODS FOR TREATING GLYCOGEN STORAGE DISEASE TYPE 1A
#886CGAS/DNCV-LIKE NUCLEOTIDYLTRANSFERASES AND USES THEREOF
#887TREATMENT AND DETECTION OF INHERITED NEUROPATHIES AND ASSOCIATED DISORDERS
#888EXTRAHEPATIC DELIVERY
#889IMMUNOSTIMULATORY BACTERIA-BASED VACCINES, THERAPEUTICS, AND RNA DELIVERY PLATFORMS
#890Polycomb-associated Non-Coding RNAs
#891NOVEL TARGET TO TREAT A METABOLIC DISEASE IN AN INDIVIDUAL
#892PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#893Methods of Detecting and Treating a Tumor Expressing PT346 PDK1
#894Modulation of GYS1 expression
#895Engineered immunostimulatory bacterial strains and uses thereof
#896Modifications of mammalian cells using artificial micro-RNA to alter their properties and the compositions of their products
#897IN VIVO GENE SILENCING BY CHEMICALLY MODIFIED AND STABLE siRNA
#898COMPOSITIONS AND METHODS FOR THE TREATMENT AND PREVENTION OF NEUROLOGICAL DISORDERS
#899COMPOSITION, METHOD AND USE THEREOF
#900SPLICE ACCEPTOR SITE DISRUPTION OF A DISEASE-ASSOCIATED GENE USING ADENOSINE DEAMINASE BASE EDITORS, INCLUDING FOR THE TREATMENT OF GENETIC DISEASE