109048 ⎘
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
Sub-classes:SYSTEMS AND METHODS FOR MODELING THROMBIN-ANTITHROMBIN
#2AGENTS FOR MODULATING SYNGAP1 SPLICING
#3RECOMBINANT AAV VECTORS USEFUL FOR REDUCING IMMUNITY AGAINST TRANSGENE PRODUCTS
#4AMINO ACID DERIVATIVES AS NOVEL DELIVERY AGENTS FOR FUNCTIONAL DELIVERY OF OLIGONUCLEOTIDES
#5METHOD FOR CIRCRNA CONSTRUCTION AND SYNTHESIS BASED ON THE COB GENE OF FUSARIUM OXYSPORUM AND RELATED APPLICATIONS
#6Modified PEG-RNAs with Improved Activity in Prime Editing Applications
#7Modulation of Apolipoprotein C-III (ApoCIII) Expression in Lipoprotein Lipase Deficient (LPLD) Populations
#8Compounds, Compositions and Methods for Reducing MAPT (TAU) Expression
#9OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#10TRANSTHYRETIN (TTR) iRNA COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING OR PREVENTING TTR-ASSOCIATED OCULAR DISEASES
#11METHODS AND COMPOSITIONS INCLUDING CONJUGATED DNAZYMES
#12APTAZYME-BASED REGULATABLE GENE EXPRESSION SYSTEMS
#13USE OF SRSF1 IN PREPARATION OF MEDICAMENT FOR PROPHYLAXIS AND/OR TREATMENT OF HEART FAILURE WITH PRESERVED EJECTION FRACTION
#14METHOD TO GENERATE IMMUNO-FREE AND STRESS-RESISTANT DONOR CELLS
#15Three Cluster Galactose Type Compound, Conjugate, Making Method and Use Thereof
#16Extracellular MiRNA Motifs for Inflammation, Methods of Discovery and Uses Thereof
#17OLIGONUCLEOTIDE-BASED THERAPEUTICS TARGETING CYCLIN D2 FOR THE TREATMENT OF HEART FAILURE
#18ATTENUATION OF CYTOKINE RELEASE SYNDROME IN IMMUNOTHERAPY
#19MICRORNA-134 BIOMARKER
#20Orthogonal Cas9 Proteins for RNA-Guided Gene Regulation and Editing
#21COMPOSITIONS AND METHODS OF USE OF CRISPR-CAS SYSTEMS IN NUCLEOTIDE REPEAT DISORDERS
#22OLIGONUCLEOTIDE-MEDIATED KNOCKDOWN OF PLN
#23OLIGONUCLEOTIDE-MEDIATED KNOCKDOWN OF DUX4
#24COMPOSITIONS, METHODS, AND KITS RELATING TO THERAPEUTICS FOR THE MODULATION OF SEL1L-HRD1 ERAD ACTIVITY AND FUNCTION
#25OLIGONUCLEOTIDES
#26RNA-EDITING OLIGONUCLEOTIDES AND USES THEREOF
#27ENGINEERED CONSTRUCTS FOR INCREASED TRANSCRIPTION OF RNA PAYLOADS
#28CRISPR/CAS EFFECTOR PROTEIN AND SYSTEM
#29ENGINEERING OF SYSTEMS, METHODS AND OPTIMIZED GUIDE COMPOSITIONS WITH NEW ARCHITECTURES FOR SEQUENCE MANIPULATION
#30METHODS OF USING ZSCAN4 FOR REJUVENATING HUMAN CELLS
#31METHODS FOR TREATING MUSCULAR DYSTROPHY
#32DNA COMPOSITIONS AND RELATED METHODS
#33ASSEMBLING SYNTHETIC DNA CONSTRUCTS FROM NATURAL DNA
#34Compositions for Modulating C9ORF72 Expression
#35APOLIPOPROTEIN C3 (APOC3) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#36METHODS OF FORMING CIRCULARIZED RNA
#37CHIMERIC GENOME ORGANISMS AND METHODS SYNTHESIZING THEREOF
#38RECEPTOR INHIBITION BY PHOSPHATASE RECRUITMENT
#39COMPOSITIONS AND METHODS FOR THE TREATMENT OF LEUKODYSTROPHY AND WHOLE ANIMAL AND CELLULAR MODELS FOR IDENTIFYING EFFICACIOUS AGENTS FOR TREATMENT OF THE SAME
#40METHODS FOR TREATING HEPATITIS B INFECTION
#41SERS BIOSENSOR BASED ON CRISPR/CAS13A SYSTEM AND APPLICATION THEREOF
#42SMALL RNA-BASED PROGNOSTIC SIGNATURES AND THERAPEUTIC COMPOSITIONS FOR IDIOPATHIC PULMONARY FIBROSIS
#43COMPOSITIONS AND SYSTMES FOR HERPES SIMPLEX VIRUS PACKAGING AND USES THEREOF
#44COMPOSITIONS AND METHODS FOR ENHANCED INTESTINAL ABSORPTION OF CONJUGATED OLIGOMERIC COMPOUNDS
#45COMPOSITIONS AND METHODS COMPRISING PROGRAMMABLE SNRNAS FOR RNA EDITING
#46GENETIC EDITING OF TARGET GENES TO ENHANCE NATURAL KILLER CELL FUNCTION
#47NOVEL MICROBIAL GENOTOXINS
#48STEM CELL BASED DELIVERY OF TUMOR-SPECIFIC RETROVIRAL VECTORS
#49GENES CONFERRING RESISTANCE TO ANTHRACNOSE FRUIT ROT OF STRAWBERRIES
#50RNAi Agents for Inhibiting Expression of Mucin 5AC (MUC5AC), Compositions Thereof, and Methods of Use
#51METHODS AND COMPOSITIONS FOR THE TREATMENT OF CANCER BY TARGETING ONCOGENIC TRANSFER RNAS
#52GENE ACTIVATION AND INTERFERENCE TARGETS FOR EXHAUSTION/DYSFUNCTIONRESISTANT T CELL PRODUCTS AND USES THEREOF
#53PRE-MEDICATION AND IMPROVED TREATMENT REGIMEN
#54VIRAL EXPRESSION CONSTRUCT COMPRISING A FIBROBLAST GROWTH FACTOR (FGF21) CODING SEQUENCE
#55POLYNUCLEOTIDES FOR SILENCING TRANSCRIPT VARIANT 1 OF ASSEMBLY FACTOR FOR SPINDLE MICROTUBULES AND APPLICATIONS THEREOF
#56COMPOSITIONS OF EXTRACELLULAR VESICLES AND METHOD USING THE SAME
#57COMPOSITIONS AND METHODS FOR TRANSGENE EXPRESSION FROM AN ALBUMIN LOCUS
#58Modulation of Apolipoprotein C-III (ApoCIII) Expression in Lipoprotein Lipase Deficient (LPLD) Populations
#59MODIFIED OLIGONUCLEOTIDES AND DOUBLE-STRANDED RNAS
#60ANTISENSE OLIGONUCLEOTIDES FOR TARGETING PROGRANULIN
#61Compounds and Methods for Modulating UBE3A-ATS
#62RNAi AGENTS FOR MODULATING SNCA
#63STABILIZED RNA AGENTS
#64GENE EDITING SYSTEMS COMPRISING REVERSE TRANSCRIPTASES
#65GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#66METHODS FOR ENHANCING TCRab+ CELL DEPLETION EFFICIENCY
#67REELIN COMPOSITIONS FOR TREATMENT OF NEUROLOGICAL DISORDERS
#68METHODS OF TREATING AN ISCHEMIC DISEASE
#69NON-CODING SMALL RNA (SRNA) FROM CORYNEBACTERIUM GLUTAMICUM AND ITS USE
#70COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY
#71COMPOSITIONS AND METHODS FOR IMMUNE CHECKPOINT INHIBITION
#72FINE-TUNING INFLAMMATION BY ALTERING 3'UTR LENGTH
#73DNA IMMORTALIZATION CONSTRUCT AND PERFORMING PRIMARY CELL IMMORTALIZATION
#74Compositions and Methods for Treating Alpha-1 Antitrypsin Deficiency
#75RNA MOLECULES
#76DOWNREGULATING TROLLS USING NOVEL ANTISENSE OLIGONUCLEOTIDES TO OVERCOME RESISTANCE TO CHEMOTHERAPY
#77USE OF MIR-15A-5P IN TREATMENT OF FUNDUS OCULI DISEASE
#78COMBINATION THERAPY FOR SPINAL MUSCULAR ATROPHY
#79OLIGONUCLEOTIDES CONJUGATED TO OLEIC ACID AND USES THEREOF
#80USE OF TNKS INHIBITORS FOR REGENERATION OF CARTILAGE
#81COMPOSITIONS FOR AQUACULTURING
#82RUNX1 MODULATORS
#83POMPE DISEASE MOUSE MODEL GENERATION, CHARACTERIZATION AND METHODS OF USE
#84INHIBITORY RNA FOR THE CONTROL OF PHYTOPATHOGENS
#85COMPOSITIONS AND METHODS FOR TREATING MYOPATHY
#86INHIBITORY RNA NANOPARTICLES
#87SLC2A1 LNCRNA AS A BIOLOGIC AND RELATED TREATMENTS AND METHODS
#88COMPOSITIONS AND METHODS FOR TREATING PRIMARY CILIARY DYSKINESIA
#89COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#90LONG NON-CODING RNAS AS TARGET FOR TREATING FIBROSIS AND CANCER
#91COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#92MODULATORY POLYNUCLEOTIDES
#93REPROGRAMMABLE ISCB NUCLEASES AND USES THEREOF
#94SURF4 GENE KNOCKOUT ERYTHROID PROGENITOR CELLS AND METHODS OF ERYTHROID DIFFERENTIATION THEREOF
#95MEANS TO TREAT AUTOIMMUNITY BY REFINING REGULATORY T CELLS
#96EPO RECEPTOR AGONISTS AND ANTAGONISTS
#97METHODS AND COMPOSITIONS FOR TREATING CORNEAL WOUNDS
#98DRUG DELIVERY SYSTEM FOR BLOOD-BRAIN BARRIER PENETRATION
#99RIBOZYME-ACTIVATED RNA CONSTRUCTS AND USES THEREOF
#100ANTIBACTERIAL PHAGE, THERAPEUTIC COMPOSITION, BACTERICIDAL AGENT, FOOD, BACTERIA IDENTIFICATION KIT, THERAPEUTIC COMPOSITION MANUFACTURING METHOD, BACTERIA ELIMINATION METHOD, BACTERIA IDENTIFICATION METHOD, AND ANIMAL THERAPEUTIC METHOD
#101Compounds and Methods for Reducing ATXN3 Expression
#102POLYMER DERIVATIVES AND THEIR USE AS LIPID NANOPARTICLE MODIFIERS
#103LIPID NANOPARTICLES
#104METHODS FOR GENE TRAINING
#105SARNA DELIVERY SYSTEM, PREPARATION METHOD THEREFOR AND USE THEREOF
#106HIPPO AND DYSTROPHIN COMPLEX SIGNALING IN CARDIOMYOCYTE RENEWAL
#107OLIGONUCLEOTIDES FOR MAPT MODULATION
#108CELLULAR COMPOSITIONS AND METHODS OF TREATMENT I
#109RNA-GUIDED DNA INTEGRATION USING TN7-LIKE TRANSPOSONS
#110NUCLEIC ACID REGULATORY ELEMENTS FOR GENE EXPRESSION IN THE LIVER AND METHODS OF USE
#111NUCLEIC ACID REGULATORY ELEMENTS FOR CONSTITUTIVE GENE EXPRESSION AND METHODS OF USE
#112CRISPR KNOCKDOWN OT ACIDITHIOBACILLUS FERROOXIDANS GENES
#113SGRNA FOR UTROPHIN ACTIVATION TO TREAT DUCHENNE MUSCULAR DYSTROPHY AND USE THEREOF
#114METHODS AND COMPOSITIONS FOR SELECTIVE REGULATION OF PROTEIN EXPRESSION
#115ANTI-FIBROTIC MICRORNA COMPOSITION
#116METHODS AND COMPOSITIONS FOR SINGLE-STRANDED RNA-INITIATED RISC ASSEMBLY
#117COMPOSITION AND METHOD FOR INHIBITING ANGIOTENSINOGEN (AGT) PROTEIN EXPRESSION
#118Antisense oligonucleotides for treatment of USHER 2A. Exon 68
#119siRNA COMPOSITIONS AND METHODS TARGETING ALPHA-SYNUCLEIN NUCLEIC ACIDS
#120EPITOPE OF EPB41L5, AND MONOCLONAL ANTIBODY
#121METHODS OF ATTENUATING AN IMMUNE RESPONSE BY INHIBITION OF BFL1
#122COMPOSITIONS AND METHODS FOR MODULATING MECP2 EXPRESSION
#123METHODS AND COMPOSITIONS FOR DESIGNING AND SELECTING TINYRNAS TO MAXIMIZE TARGET CLEAVAGE
#124COMPOSITIONS AND METHODS FOR TREATMENT OF SPINOCEREBELLAR ATAXIAS
#125COMPOSITIONS AND METHODS FOR TREATMENT OF NEUROLOGICAL DISORDERS
#126MODIFIED DOUBLE-STRANDED RNA AGENTS
#127COMPOUNDS AND METHODS FOR SKIPPING EXON 44 IN DUCHENNE MUSCULAR DYSTROPHY
#128CRISPR/CPF1 Systems and Methods
#129EXOSOMES ISOLATED FROM MESENCHYMAL STEM CELLS AND USES THEREOF
#130ANTI-C5 ANTIBODY/C5 IRNA DOSING REGIMENS FOR TREATING C5-ASSOCIATED DISEASES
#131RESPIRATORY SYNCYTIAL VIRUS (RSV) POLYANHYDRIDE NANOPARTICLE VACCINE
#132RNA AND DNA BASE EDITING VIA ENGINEERED ADAR RECRUITMENT
#133METHODS TO MODULATE PROTEIN TRANSLATION EFFICIENCY
#134METHODS FOR IMPROVING THE HEALTH OF PORCINE SPECIES BY TARGETED INACTIVATION OF CD163
#135Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications
#136PRODUCTS AND COMPOSITIONS
#137METHODS AND CONTROL COMPOSITIONS FOR SEQUENCING
#138Method to Rejuvenate Human Cells Through Transcriptional Reprogramming
#139CELL-PENETRATING PEPTIDES
#140MICRORNA AND USES THEREOF IN PREVENTION AND/OR TREATMENT OF FIBROPLASIA MEDICAL SIGN AND/OR SYNDROME
#141METHOD OF DETECTING A POLYNUCLEOTIDE ANALYTE
#142SIRNA FOR TARGETED INHIBITION OF AGT GENE EXPRESSION AND USE THEREOF IN TREATING HYPERTENSION
#143USE OF HOMEOBOX CONTAINING 1 (HMBOX1) INHIBITOR IN PREPARATION OF DRUG FOR PREVENTION AND/OR TREATMENT OF MUSCLE ATROPHY
#144POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF CANCER
#145CONJUGATED OLIGONUCLEOTIDE COMPOUNDS, METHODS OF MAKING AND USES THEREOF
#146COMPOUNDS AND METHODS FOR MODULATING SMN2
#147COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#148COMPOSITIONS AND METHODS FOR REDUCING RNA LEVELS
#149ANTISENSE OLIGONUCLEOTIDE TARGETING TDP-43 MRNA OR PRE-MRNA
#150COMPOSITIONS AND METHODS FOR CONTROLLING PLANT PESTS
#151POLYNUCLEOTIDES TARGETING NR4A3 AND USES THEREOF
#152METHODS TO TREAT NEURODEGENERATIVE DISORDERS BY REDUCING NUCLEAR AGGREGATES AND MODULATING ADENOSINE-TO-INOSINE RNA EDITING
#153ZIKV-BASED GENE DELIVERY SYSTEM
#154PROBIOTICS DESIGNED TO EXPRESS AND SECRETE AKKERMANSIA MUCINIPHILA TARS, AND A VECTOR FOR PRODUCING THE SAME
#155NOVEL SELF-CLEAVING DNAZYMES AND SELECTION PROCESS
#156Cyclic Antisense Therapeutics
#157NUCLEIC ACIDS FOR INHIBITING EXPRESSION OF LPA IN A CELL
#158CALCIUM CHANNEL 3.2 INHIBITORY PEPTIDES AND USES THEREOF
#159SELF-CIRCULARIZATION RNA STRUCTURE
#160Method of Inhibiting Durotaxis and/or Treating Fibrosis
#161METHODS AND COMPOSITIONS FOR TREATING PANCREATIC CANCER
#162DEVICES AND SYSTEMS FOR DNA CAPTURE
#163Engineered Cell and Application Thereof
#164NUCLEIC ACID CARRIER AND METHOD FOR ADMINISTERING NUCLEIC ACID
#165METHODS FOR TREATING CANCER WITH HYPERACTIVE ADAR ENZYMES
#166SPLIT RIBOZYME BIOSENSOR SYSTEMS
#167A NON-INVASIVE DEVICE AND METHOD FOR DETECTING RNA ASSOCIATED DISEASE
#168COMPOSITIONS AND METHODS FOR INHIBITING LPA EXPRESSION
#169Oral Delivery of Antisense Conjugates Targeting PCSK9
#170ANTISENSE OLIGOMER COMPOUNDS
#171OLIGONUCLEOTIDES FOR MODULATING REGULATORY T CELL MEDIATED IMMUNOSUPPRESSION
#172SYSTEMS FOR ENHANCING TARGET MRNA EXPRESSION AND USES THEREOF
#173ANTAGONISTS OF HUMAN MICRO RNA MIR-100, MIR-20, MIR-222, MIR-181 AND MIR-92 AND USES OF SAME
#174ENGINEERED CASCADE COMPONENTS AND CASCADE COMPLEXES
#175HUMAN iPSC-DERIVED BRAIN ORGANOIDS AND USES THEREOF
#176ORGANIC COMPOSITIONS TO TREAT KRAS-RELATED DISEASES
#177RNA INTERFERENCE (RNAI) FOR CONTROL OF JAPANESE BEETLE
#178FIELD-DEPLOYABLE FLUIDIC REACTOR
#179CELL MEMBRANE PENETRATING CONJUGATES FOR GENE EDITING
#180COMPOSITIONS AND METHODS FOR TREATING STARGARDT DISEASE
#181Delivery of RNA Therapeutics Using Circular Prodrug Nucleic Acids
#182TEMPORAL CONTROL OF CRISPR-Cas9 ACTIVITY USING BIOORTHOGONAL CHEMISTRY
#183COMPOSITIONS AND METHODS FOR TREATING MENINGIOMA
#184ENGINEERED RETRONS AND METHODS OF USE
#185METHODS AND COMPOSITIONS FOR MODULATING SPLICING AT ALTERNATIVE SPLICE SITES
#186HIGH DRUG-LOADING RATIO APTAMER-DRUG CONJUGATES AND ITS APPLICATION
#187OLIGONUCLEOTIDE COMPOSITIONS AND METHODS OF USE THEREOF
#188METHODS AND COMPOSITIONS OF TREATING RETINAL DEGENERATIVE DISEASES
#189NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#190LIPOSOMAL PARTICLES, METHODS OF MAKING SAME AND USES THEREOF
#191miR-25802 CLUSTER AS BIOMARKER FOR INFLAMMATION-ASSOCIATED DISEASE, AND USE OF miR-25802 CLUSTER
#192CLICK CHEMISTRY LIGAND
#193COMPOUNDS AND METHODS FOR TREATING HUMAN SUBJECTS
#194CELL-SPECIFIC CIS-REGULATORY ELEMENTS, USES THEREOF, AND METHODS OF GENERATING THE SAME
#195COMPOSITIONS AND METHODS FOR TREATING NONALCOHOLIC FATTY LIVER DISEASE
#196METHODS FOR TREATMENT OF POLYCYSTIC KIDNEY DISEASE
#197TREATMENT
#198EXTRACELLULAR VESICLE COMPRISING A BIOLOGICALLY ACTIVE MOLECULE AND A CELL PENETRATING PEPTIDE CLEAVABLE LINKER
#199TREATMENT OF ALZHEIMER'S DISEASE WITH VASCULAR PROGENITOR-DERIVED EXOSOMES ENRICHED WITH MICRORNAS OR THIOREDOXIN
#200OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#201MODIFIED OLIGONUCLEOTIDES
#202EFFECTOR PROTEINS, COMPOSITIONS, SYSTEMS AND METHODS OF USE THEREOF
#203CRISPR/CAS9-BASED REPRESSORS FOR SILENCING GENE TARGETS IN VIVO AND METHODS OF USE
#204ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS WITH IMPROVED RETINAL TRANSDUCTION AND USES THEREOF
#205CATIONIC PEPTIDE/PROTEIN-MODIFIED EXOSOMES FOR APPLICATIONS IN DRUG DELIVERY
#206NONCODING RNA AGENTS (BCYRN1 AND DERIVATIVES THEREOF) TO TREAT DISEASES OF IMMUNITY
#207COMPOSITIONS AND METHODS FOR TREATING TRANSTHYRETIN (TTR) MEDIATED AMYLOIDOSIS
#208CELL-BASED ASSAY FOR QUANTIFYING THE POTENCY AND EFFICACY OF CANNABINOIDS AND/OR TERPENOIDS, AND METHODS OF USE THEREOF
#209Therapeutic Strategies To Reduce Thrombotic Risk In Factor V Leiden (FVL) Carriers
#210MULTIPLEXED RNA QUANTIFICATION USING CRISPR-CAS13
#211REGULATION OF NUCLEOTIDE EXCISION REPAIR (NER) BY MICRORNA FOR TREATMENT OF CANCER
#212COMPLEMENT COMPONENT C3 IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#213Harnessing MicroRNAs (miRNAs) for Targeted Therapeutics in Cardiac Arrest
#214METHOD FOR REDUCING THE RISK OF A CARDIOVASCULAR EVENT WITH CONJUGATED ANTISENSE COMPOUNDS TARGETING APO(A)
#215REGULATION OF ACTIVITY OF RNAI MOLECULES
#216CRISPR/CAS SCREENING PLATFORM TO IDENTIFY GENETIC MODIFIERS OF TAU SEEDING OR AGGREGATION
#217COMPOSITIONS AND METHODS OF USING PRKAG2-TARGETING ANTIBODY-OLIGONUCLEOTIDE CONJUGATES
#218METHODS FOR DECREASING OR INHIBITING HUMAN L1 RETROTRANSPOSON RNA AND COMPOSITIONS FOR USE THEREIN
#219Inhibition of TCF4/ITF2 in the Treatment of Cancer
#220METHODS AND SYSTEMS FOR GENERATING NUCLEIC ACID DIVERSITY IN CRISPR-ASSOCIATED GENES
#221AMBIENT TEMPERATURE NUCLEIC ACID AMPLIFICATION AND DETECTION
#222METHODS, SYSTEMS AND COMPOSITIONS FOR ANALYTE DETECTION
#223NATURE-DERIVED LIPID PARTICLES FOR USE IN AGRICULTURE
#224TREATMENT OF SPINAL MUSCULAR ATROPHY
#225LINKER, COMPOUND INCLUDING LINKER, METHOD OF PREPARING COMPOUND, USE OF LINKER, AND METHOD OF DELIVERING OLIGONUCLEOTIDES THROUGH LINKER
#226Compositions and Methods for Hydroxyacid Oxidase 1 (HAO1) Gene Editing for Treating Primary Hyperoxaluria Type 1 (PH1)
#227RNAi Agents And Compositions for Inhibiting Expression of Apolipoprotein C-III (APOC3)
#228MIRNAS TARGETING ATNX2 FOR THE TREATMENT OF ALS AND SCA2
#229GENE THERAPY FOR LEVODOPA-INDUCED DYSKINESIA
#230SIRNA TARGETING KEY GENE OF MAGNAPORTHE ORYZAE FOR CONTROLLING RICE BLAST, AND USE THEREOF
#231GENETICALLY MODIFIED CELLS, TISSUES, AND ORGANS FOR TREATING DISEASE
#232NOVEL TRANSMEMBRANE SENSORS AND METHOD OF CHARACTERIZATION FOR LYSIS-FREE DETECTION OF INTRACELLULAR TARGETS
#233Compositions and Methods for Correcting Limb Girdle Muscular Dystrophy Type 2C Using Exon Skipping
#234OLIGONUCLEOTIDES FOR PRNP MODULATION
#235COMPRISING EXOSOMAL MIR-664A-5P AS AN ACTIVE INGREDIENT, A DIAGNOSTIC BIOMARKER COMPOSITION FOR PARP INHIBITOR RESPONSE AND A PHARMACEUTICAL COMPOSITION FOR CANCER TREATMENT
#236Compositions and Methods for Lactate Dehydrogenase (LDHA) Gene Editing
#237COMBINATION TREATMENT FOR CANCER
#238COMPOSITIONS AND METHODS FOR MODULATING APOLIPOPROTEIN C-III EXPRESSION
#239ANTISENSE OLIGOMERS FOR TREATMENT OF NON-SENSE MEDIATED RNA DECAY BASED CONDITIONS AND DISEASES
#240SYSTEMS FOR CELL PROGRAMMING TOWARDS HEMATOPOIETIC LINEAGE AND METHODS THEREOF
#2413D CELL CULTURE METHODS
#242MICROFLUIDIC TRANSWELL SYSTEMS FOR IMAGING PAIRED CELL INTERACTIONS VIA EXOSOMES
#243INHIBITORS OF SARM1 IN COMBINATION WITH NAD+ OR A NAD+ PRECURSOR
#244METHODS AND COMPOSITIONS FOR SPATIALLY-RESOLVED SINGLE CELL SEQUENCING
#245ARTIFICIAL NUCLEIC ACID MOLECULES FOR IMPROVED PROTEIN OR PEPTIDE EXPRESSION
#246Differential Knockout of An Allele of A Heterozygous Elane Gene - II
#247PRODUCT PREPARATION BASED ON APPLICATION OF SGRNA FOR THE TREATMENT OF HUNTINGTON'S DISEASE
#248AAV-MEDIATED DELIVERY OF OSTEOBLAST/OSTEOCLAST-REGULATING MIRNAS FOR OSTEOPOROSIS THERAPY
#249RNAi AGENTS OF PRION EXPRESSION
#250DIFFERENTIATION OF STEM CELLS IN SUSPENSION CULTURE
#251SYSTEMS AND METHODS FOR PROMOTING TRANS-SPLICING
#252POLYPEPTIDES SELF-ASSEMBLING INTO NANOPARTICLES
#253GUIDE RNAS, VECTORS, AND VIRIONS FOR TARGETING MUTATIONS IN THE PLN GENE
#254ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#255USE OF AKR1C3 INHIBITOR IN PREPARATION OF DRUG FOR TREATING OR PREVENTING METABOLIC ASSOCIATED FATTY LIVER DISEASE
#256GENOME EDITING OF THE KOZAK SEQUENCE FOR TREATING DISEASES
#257B CELL-SPECIFIC MAB-SIRNA CONJUGATES IMPROVE MYASTHENIA
#258EXTRACELLULAR VESICLE-ASO CONSTRUCTS TARGETING CEBP/BETA
#259COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
#260APTAMER DISCOVERY AND SELECTION TECHNIQUES
#261AAV-BASED TREATMENT FOR ALAGILLE SYNDROME
#262CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION
#263SYSTEMS FOR CELL PROGRAMMING AND METHODS THEREOF
#264Single Construct Platform for Simultaneous Delivery of Gene Editing Machinery and Nucleic Acid Cargo
#265SUBPOPULATION-DIRECTED REMEDIATION OF DISEASE-ASSOCIATED GENE PRODUCTS
#266Compositions and Methods for Modulating Complement Factor B Expression
#267PHARMACEUTICAL COMPOSITION FOR PREVENTING OR TREATING CANCER COMPRISING SIRNA AS ACTIVE INGREDIENT
#268POLYNUCLEOTIDE COMPOSITIONS AND METHODS FOR TREATMENT OF NEURODEGENERATIVE DISEASES
#269TAU-SEED INTERACTOR INHIBTORS FOR THE TREATMENT OF NEURODEGENERATIVE DISORDERS
#270METHODS AND COMPOSITIONS FOR SELECTIVELY MODULATING GENE EXPRESSION IN MEGAKARYOCYTES AND PLATELETS
#271Use Of Serine/Arginine-Rich Splicing Factor 1 (SRSF1) As Therapeutic Target For Aortic Dissection
#272BROAD SPECTRUM NANOZYMES
#273PHASE TRANSITION ADJUSTING ELEMENT AND USE THEREOF
#274METHODS FOR TREATING GLIOBLASTOMA OR RECURRENT GLIOBLASTOMA UTILIZING A WIRELESS SIGNAL ALONE OR IN COMBINATION WITH ONE OR MORE CANCER DRUGS, AND ASSOCIATED SYSTEMS, APPARATUSES, AND DEVICES
#275METHODS AND COMPOSITIONS FOR TREATING TUMOR CELLS
#276RNAI AGENTS FOR INHIBITING EXPRESSION OF HUNTINGTIN (HTT), COMPOSITIONS THEREOF, AND METHODS OF USE
#2771'-ALKYL MODIFIED RIBOSE DERIVATIVES AND METHODS OF USE
#278METHODS AND COMPOSITIONS OF MATTER FOR INERT BIOENGINEERING OF A BIOLOGICAL ENTITY
#279SPERMATOGENESIS ALTERATION
#280BRANCHED LIPID CONJUGATES OF siRNA FOR SPECIFIC TISSUE DELIVERY
#281FACTOR XII (HAGEMAN FACTOR) (F12), KALLIKREIN B, PLASMA (FLETCHER FACTOR) 1 (KLKB1), AND KININOGEN 1 (KNG1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#282NOVEL STAPLE NUCLEIC ACID
#283COMPOSITIONS AND METHODS OF NUCLEIC ACID-TARGETING NUCLEIC ACIDS
#284TREATMENT OF AUTOIMMUNE DISEASES WITH BCMA-TARGETING ENGINEERED IMMUNE CELLS
#285TXNIP INHIBITION TO ENHANCE ADOPTIVE IMMUNOTHERAPY
#286ENGINEERED TUBEROUS SCLEROSIS COMPLEX 2 POLYPEPTIDES
#287POLYMER NANOPARTICLE AND DNA NANOSTRUCTURE COMPOSITIONS AND METHODS FOR NON-VIRAL DELIVERY
#288RNA-BASED CONTROL OF PRODUCTION OF DEOXYNIVALENOL BY FUSARIUM
#289High throughput prime editing screens identify functional DNA variants in the human genome
#290REGULATORY NUCLEIC ACID MOLECULES FOR ENHANCING CONSTITUTIVE GENE EXPRESSION IN PLANTS
#291ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TREATING DYSTROPHIES
#292ANTI-C9ORF72 OLIGONUCLEOTIDES AND RELATED METHODS
#293MODIFYING THE SPECIFICITY OF PLANT NON-CODING RNA MOLECULES FOR SILENCING GENE EXPRESSION
#294COMPOUND FOR INHIBITING AGT GENE EXPRESSION, AND PHARMACEUTICAL COMPOSITION AND USE THEREOF
#295COMPOUND FOR INHIBITING EXPRESSION OF LPA GENE, PHARMACEUTICAL COMPOSITION AND USE THEREOF
#296REGULATORY SEQUENCES COMPRISING MicroRNA TARGET SITES
#297METHODS OF MAKING AND USING IMMUNE-TOLERIZED FETAL STEM CELL-DERIVED EXTRACELLULAR VESICLES FOR HAIR LOSS TREATMENT
#298DESIGN AND APPLICATION OF NOVEL PHOTORECEPTOR-SPECIFIC PROMOTERS FOR RETINAL GENE THERAPY APPLICATIONS
#299COMPOSITIONS AND METHODS OF TREATING MUSCLE ATROPHY AND MYOTONIC DYSTROPHY
#300Neutral Liposomes Containing Biologically Active Agents