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
Method for producing exosomes and exosomes obtained thereby
#5102Medical devices for delivery of siRNA
#5103Method for treating or preventing heart failure
#5104Direct reprogramming of cells to cardiac myocyte fate
#5105Serum amyloid P component (APCS) iRNA compositions and methods of use thereof
#5106Use of specific siRNA against protein S for the treatment of hemophilia
#5107Liposomes for modulating Wiskott-Aldrich syndrome protein
#5108Genetically modified non-human animals and methods for producing heavy chain-only antibodies
#5109Use of leucine-zipper protein for diagnosis or treatment of fatty liver
#5110COMPOSITIONS AND METHODS FOR ENHANCING CELL TRANSPLANTATION EFFICACY
#5111IN VITRO SELECTION FOR NUCLEIC ACID APTAMERS
#5112Enzymatic methods to generate high yields of sequence specific rna with extreme precision
#5113METHODS AND COMPOSITIONS FOR INHIBITION OF INNATE IMMUNE RESPONSE ASSOCIATED WITH AAV TRANSDUCTION
#5114CRISPR-CAS GENOME ENGINEERING VIA A MODULAR AAV DELIVERY SYSTEM
#5115CENH3 DELETION MUTANTS
#5116RNAi induced huntingtin gene suppression
#5117Amyloid precursor protein (APP) RNAi agent compositions and methods of use thereof
#5118RNAi CONJUGATES, PARTICLES AND FORMULATIONS THEREOF
#5119NUCLEIC ACID NANOSTRUCTURES WITH CORE MOTIFS
#5120RAAV-based compositions and methods
#5121ASSAYS AND METHODS FOR DETERMINING EXPRESSION OF THE LECT2 GENE
#5122Treatment of HER-2 dependent cancer using an agent that modulates the activity of a miRNA
#5123EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#5124Multivalent oligonucleotide assemblies
#5125Methods of polynucleotide preparation using multivalent cation salt compositions
#5126Generation of induced pluripotent cells by CRISPR activation
#5127Systems and methods for treating hyper-igm syndrome
#5128METHODS AND MATERIALS FOR TREATING CANCER
#5129Reducing beta-catenin and IDO expression to potentiate immunotherapy
#5130Methods for treating hepatitis B infection
#5131Methods for treating hepatitis B infection
#5132Compositions and methods for treating allergic inflammatory conditions
#5133Altering microbial populations and modifying microbiota
#5134Large animal model for developing therapeutic agents to treat impaired ophthalmic function in usher syndrome
#5135MULTI-TARGET MODULATION FOR TREATING FIBROSIS AND INFLAMMATORY CONDITIONS
#5136ANTISENSE OLIGONUCLEOTIDE DIRECTED REMOVAL OF PROTEOLYTIC CLEAVAGE SITES FROM PROTEINS
#5137Use of MIR-92A or MIR-145 in the treatment of Angelman syndrome
#5138ARTIFICIAL NUCLEIC ACID MOLECULES FOR IMPROVED PROTEIN OR PEPTIDE EXPRESSION
#5139Dual Targeting siRNA Agents
#5140Use of p38 inhibitors to reduce expression of DUX4
#5141miRNA as biomarker for Parkinson's disease and diagnostic kit using same
#5142GAPMER OLIGONUCLEOTIDES COMPRISING A PHOSPHORODITHIOATE INTERNUCLEOSIDE LINKAGE
#5143Variant CAS12 proteins with improved dna cleavage selectivity and methods of use
#5144Enzymes with RuvC domains
#5145Enzymes with RuvC domains
#5146SYSTEMS, METHODS, AND COMPOSITIONS FOR TARGETED NUCLEIC ACID EDITING
#5147METHOD TO BIOENGINEER DESIGNER RED BLOOD CELLS USING GENE EDITING AND STEM CELL METHODOLOGIES
#5148CATIONIC LIPIDS
#5149Liposomal system for drug delivery
#5150TUMOR INFILTRATING LYMPHOCYTES AND METHODS OF THERAPY
#5151METHODS FOR THE TREATMENT OF CANCER USING COENZYME Q10 AND FATTY ACID METABOLISM INHIBITORS
#5152Ophthalmic delivery device and ophthalmic drug compositions
#5153Pig genome-wide specific sgRNA library, preparation method therefor and application thereof
#5154CD133 related to anticancer agent resistance in colon cancer and use thereof
#5155CANCER BIOMARKERS AND METHODS OF USE
#5156METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#5157Antisense Oligonucleotides (ODN) Against SMAD7 and Uses Thereof in Medical Field
#5158Methods and compositions for modulating gene expression using oligonucleotide based drugs administered in vivo or in vitro
#5159RNA-GUIDED DNA INTEGRATION USING TN7-LIKE TRANSPOSONS
#5160Compositions and methods for modulating Ataxin 3 expression
#5161Mammalian cell line for protein production and library generation
#5162VECTORS CONTAINING AIMP2-DX2 AND TARGET NUCLEIC ACIDS FOR miR 142 AND USES THEREOF
#5163COLON DISEASE TARGETS AND USES THEREOF
#5164PEPTIDE NUCLEIC ACID MOLECULES FOR TREATMENT OF GRAM POSITIVE BACTERIAL INFECTION
#5165MicroRNA-294 and Lin28A as a driver of cardiac tissue proliferation in response to pathological injury
#5166METHODS FOR THE PRODUCTION OF STERILE FISH AND OTHER EGG-PRODUCING AQUATIC ANIMALS AND COMPOUNDS FOR USE IN THE METHODS
#5167RNA targeting methods and compositions
#5168Method for determining decrease in functions of hippocampus by using correlation between micro RNA and NMDA receptor, method for inhibiting decrease in functions, and method for screening for inhibitors of decrease in functions
#5169CRISPR enzymes and systems
#5170NOVEL CRISPR ENZYMES AND SYSTEMS
#5171Regulatory nucleic acid molecules for enhancing constitutive gene expression in plants
#5172CRISPR-CAS COMPONENT SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION
#5173Unnatural base pair compositions and methods of use
#5174Compositions and their uses directed to huntingtin
#5175Gene therapy for neurodegenerative disorders
#5176C/EBP ALPHA SHORT ACTIVATING RNA COMPOSITIONS AND METHODS OF USE
#5177POLYNUCLEOTIDE CONJUGATES AND USES THEREOF
#5178CANCER TREATMENT
#5179RNAI AGENTS, COMPOSITIONS AND METHODS OF USE THEREOF FOR TREATING TRANSTHYRETIN (TTR) ASSOCIATED DISEASES
#5180A COMPOSITION AND METHOD OF USING MIR-302 PRECURSORS AS ANTI-CANCER DRUGS FOR TREATING HUMAN LUNG CANCER
#5181LNA-G process
#5182CONTROLLING PHENOTYPE OF ORGANISMS WITH CRISPR/CAS GENE TARGETING
#5183Engineered nucleic-acid targeting nucleic acids
#5184METHODS FOR ENHANCING TCRab+ CELL DEPLETION
#5185Cell-penetrating peptides for antisense delivery
#5186Compositions and methods for inhibition of HAO1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
#5187Compositions and methods for inhibiting PTPN22
#5188METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#5189METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#5190METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#5191IN VITRO METHOD OF MRNA DELIVERY USING LIPID NANOPARTICLES
#5192HIGH MOBILITY GROUP BOX-1 (HMGB1) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
#5193Composition for delivery of genetic material
#5194Thiomorpholino oligonucleotides for the treatment of muscular dystrophy
#5195TARGETING GLIOBLASTOMA STEM CELLS THROUGH THE TLX-TET3 AXIS
#5196Oligonucleotide compounds for targeting huntingtin mRNA
#5197Composition for genome editing using CRISPR/CPF1 system and use thereof
#5198KCNK3-BASED GENE THERAPY OF CARDIAC ARRHYTHMIA
#5199CRISPR/Cas system and method for genome editing and modulating transcription
#5200Combinations for the modulation of SMN expression
#5201Liposomal spherical nucleic acid (SNA) constructs presenting antisense oligonucleotides (ASO) for specific knockdown of interleukin 17 receptor mRNA
#5202Modification of D- and T-arms of tRNA enhancing D-amino acid and β-amino acid uptake
#5203Adenine DNA base editor variants with reduced off-target RNA editing
#5204Cas9 variants and methods of use
#5205Trialkyl cationic lipids and methods of use thereof
#5206Intracellular genomic transplant and methods of therapy
#5207INTRACELLULAR GENOMIC TRANSPLANT AND METHODS OF THERAPY
#5208ApoCIII and the Treatment and Diagnosis of Diabetes
#5209Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#5210Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#5211RNA-Guided Transcriptional Regulation
#5212PRIMARY CELL GENE EDITING
#5213Gene silencing kills emerald ash borer, an exotic, invasive tree-killing insect
#5214Components and methods for producing toxic RNAs in eukaryotic cells
#5215RNA INTERFERENCE MEDIATING SMALL RNA MOLECULES
#5216Oligonucleotide compositions and methods thereof
#5217RNAi agents for inhibiting expression of alpha-ENaC and methods of use
#5218RNAi agents and compositions for inhibiting expression of apolipoprotein C-III (APOC3)
#5219MODIFIED CPF1 GUIDE RNA
#5220POLYNUCLEOTIDE COMPLEXES HAVING IMPROVED DELIVERY INTO CELLS
#5221CRISPR/Cas screening platform to identify genetic modifiers of tau seeding or aggregation
#5222CRISPR/Cas dropout screening platform to reveal genetic vulnerabilities associated with tau aggregation
#5223CASZ compositions and methods of use
#5224CRISPR-associated systems and components
#5225ENGINEERED NUCLEASES THAT TARGET HUMAN AND CANINE FACTOR VIII GENES AS A TREATMENT FOR HEMOPHILIA A
#5226Method and carrier complexes for delivering molecules to cells
#5227Therapy specific, pre-programmed auto injection device
#5228Fungal immunogens and related materials and methods
#5229METHODS, SYSTEMS, AND COMPOSITIONS RELATING TO TREATMENT OF NEUROLOGICAL CONDITIONS, DISEASES, AND INJURIES AND COMPLICATIONS FROM DIABETES
#5230miR-133a as a marker for colorectal cancer
#5231Engineered Herpes Simplex Virus-1 (HSV-1) vectors and uses thereof
#5232TREATMENT OF GLIAL CELL DERIVED NEUROTROPHIC FACTOR (GDNF) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPT TO GDNF
#5233PREVENTIVE OR THERAPEUTIC AGENT FOR FIBROSIS
#5234ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHOD OF TREATING DYSTROPHIES
#5235Methods for the treatment of Leber congenital amaurosis
#5236Methods of rescuing stop codons via genetic reassignment with ACE-tRNA
#5237MicroRNA Compounds and Methods for Modulating MIR-21 Activity
#5238Oligonucleotide comprising an inosine for treating DMD
#5239Gapmers and methods of using the same for the treatment of muscular dystrophy
#5240METHODS AND COMPOSITIONS FOR ENHANCING THE EFFICACY AND SPECIFICITY OF RNA SILENCING
#5241CRISPR-associated transposon systems and components
#5242Methods and compositions for diagnosing and treating diseases
#5243Method for polyalkoxylation of nucleic acids that enables recovery and reuse of excess polyalkoxylation reagent
#5244Compositions and methods for expressing factor IX
#5245TUMOR INFILTRATING LYMPHOCYTES AND METHODS OF THERAPY
#5246Tumor infiltrating lymphocytes and methods of therapy
#5247Methods for treating chondrosarcoma using microrna(miR)
#5248Delivery method of target material into extracellular vesicles using extracorporeal shockwave
#5249METHODS FOR GENDER DETERMINATION OF AVIAN EMBRYOS IN UNHATCHED EGGS AND MEANS THEREOF
#5250Mutant cell-free DNA isolation kit and mutant cell-free DNA isolation method using the same
#5251GENE THERAPIES FOR NEURODEGENERATIVE DISEASES
#5252Nucleotide sequence and application thereof in enhancing plant pest resistance
#5253Iterative genome editing in microbes
#5254Antisense oligonucleotides for inducing exon skipping and methods of use thereof
#5255ALBUMIN PRODUCTION AND CELL PROLIFERATION
#5256METHODS AND COMPOSITIONS TO TREAT DRUG-INDUCED DISEASES AND CONDITIONS
#5257RNA-guided DNA integration using Tn7-like transposons
#5258ANTISENSE ANTIBACTERIAL COMPOUNDS AND METHODS
#5259as a diagnostic and therapeutic target for NAFLD, NASH, metabolic syndrome, and hepatic fibrosis
#5260Compositions and Methods for Inhibiting Expression of Transthyretin
#5261Methods for Treatment of Alport Syndrome
#5262Oligonucleotide inhibiting the expression of Chop
#5263Composition for preventing or treating liver fibrosis, containing exosome or exosome-derived ribonucleic acid
#5264Personalized cells, tissues, and organs for transplantation from a humanized, bespoke, designated-pathogen free, (non-human) donor and methods and products relating to same
#5265NUCLEIC ACID-POLYPEPTIDE COMPOSITIONS AND METHODS OF INDUCING EXON SKIPPING
#5266BIOTIN COMPLEXES FOR TREATMENT AND DIAGNOSIS OF ALZHEIMER'S DISEASE
#5267NOVEL CATIONIC LIPIDS AND METHODS OF USE THEREOF
#5268H5 avian influenza vaccine strain which differentiates infected from vaccinated animals, preparation method therefor, and application
#5269SEQUENCE SPECIFIC ANTIMICROBIALS
#5270Sequence specific antimicrobials
#5271Combination therapy for C3 inhibition
#5272Methods of using anti-MIR126 compounds
#5273Methods and compositions for gene editing in hematopoietic stem cells
#5274MicroRNA inhibitor therapy in systemic lupus erythematosus
#5275METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#5276Gene manipulation for treatment of retinal dysfunction disorder
#5277RNAi-MEDIATED INHIBITION OF HIF1A FOR TREATMENT OF OCULAR ANGIOGENESIS
#5278RNA targeting of mutations via suppressor tRNAs and deaminases
#5279Synthetic bacteriophages and bacteriophage compositions
#5280Modulators of MALAT1 expression
#5281Use of miR-18b for prevention, treatment, or diagnosis of muscle disease and neuromuscular disease
#5282Methods and compositions to inhibit metastasis and to treat fibrosis and to enhance wound healing
#5283SYNTHETIC HAMMERHEAD RIBOZYMES WITH LIGAND-RESPONSIVE TERTIARY INTERACTIONS
#5284Multi-effector CRISPR based diagnostic systems
#5285Insulin-like growth factor binding protein, acid labile subunit (IGFALS) and insulin-like growth factor 1 (IGF-1) iRNA compositions and methods of use thereof
#5286Peptide oligonucleotide conjugates
#5287Engineered cascade components and cascade complexes
#5288Modified Cas9 protein and use thereof
#5289Recombinant EGFL7, EGFL7 antibodies, and uses thereof
#5290Methods and compositions for modulating splicing
#5291METHODS AND COMPOSITIONS FOR TREATING AND DIAGNOSING DISEASES
#5292Treatments of oxidative stress conditions
#5293Isolated recombinant oncolytic adenoviruses, pharmaceutical compositions, and uses thereof for drugs for treatment of tumors and/or cancers
#5294Combination immunotherapies for treatment of cancer
#5295METHODS AND COMPOSITIONS FOR MODULATING APOLIPOPROTEIN (A) EXPRESSION
#5296ANTISENSE COMPOUNDS
#5297GENERATION AND CORRECTION OF A HUMANIZED MOUSE MODEL WITH A DELETION OF DYSTROPHIN EXON 44
#5298Methods for analyzing nucleic acid sequences
#5299MODULATORY POLYNUCLEOTIDES
#5300USE OF MEDEA ELEMENTS FOR BIOCONTROL OF D. SUZUKII POPULATIONS
#5301MULTIPLEX PRODUCTION AND BARCODING OF GENETICALLY ENGINEERED CELLS
#5302Method for modifying target site in double-stranded DNA in cell
#5303COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTITRYPSIN DEFICIENCY
#5304COMPOSITIONS AND METHODS FOR TRANSGENE EXPRESSION FROM AN ALBUMIN LOCUS
#5305Cancer-specific trans-splicing ribozymes and use thereof
#5306Single-Stranded RNAI Oligonucleotides Targeting APOC-III
#5307Modulation of huntingtin expression
#5308Methods and compositions for selecting siRNA of improved functionality
#5309Beta catenin nucleic acid inhibitor molecule
#5310Dynamic pharmacokinetic-modifying anchors
#5311MODIFYING THE SPECIFICITY OF NON-CODING RNA MOLECULES FOR SILENCING GENE EXPRESSION IN EUKARYOTIC CELLS
#5312USE OF ANTI-CRISPR POLYPEPTIDES FOR SPECIFIC ACTIVATION OF CAS NUCLEASES
#5313RNA SEQUENCE-SPECIFIC MEDIATORS OF RNA INTERFERENCE
#5314Pharmaceutical use of FAM19A5 involved in regulating gliogenesis
#5315RNA-modulating agents
#5316GLP-1 RECEPTOR LIGAND MOIETY CONJUGATED OLIGONUCLEOTIDES AND USES THEREOF
#5317Glaucoma treatment
#5318Compositions and methods for inducing a treg phenotype and methods for use for the same
#5319Altering microbial populations and modifying microbiota
#5320Cell-based assay for quantifying the potency and efficacy of cannabinoids and/or terpenoids, and methods of use thereof
#5321ACTIVITY-DEPENDENT GENE PAIRS AS THERAPEUTIC TARGETS AND METHODS AND DEVICES TO IDENTIFY THE SAME
#5322TARGETED INTEGRATION SYSTEMS AND METHODS FOR THE TREATMENT OF HEMOGLOBINOPATHIES
#5323Genomic insulator elements and uses thereof
#5324Methods and compositions for in situ germline genome engineering
#5325NOVEL CRISPR ENZYMES AND SYSTEMS
#5326Methods for genetic engineering host cells
#5327RNA targeting of mutations via suppressor tRNAs and deaminases
#5328COMPOSITIONS AND METHODS FOR INHIBITING GENE EXPRESSION OF LPA
#5329Compositions and methods for treating hepatic fibrosis
#5330Riboswitch-mediated regulatory control of gene expression in thermophilic bacteria
#5331Compositions and Methods for Inhibition of Expression of Apolipoprotein C-III (APOC3) Genes
#5332CHANNEL MODULATORS
#5333RNA molecules
#5334Compositions and methods for treating transposon associated diseases
#5335Cpf1 complexes with reduced indel activity
#5336METHODS AND COMPOSITIONS FOR POLYMERASE II (POL-II) BASED GUIDE RNA EXPRESSION
#5337CRISPR/Cas fusion proteins and systems
#5338Regulated multiplex reactions in a single tube
#5339Regulation of nucleic acid molecule expression
#5340EXON DELETION CORRECTION OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS IN THE DYSTROPHIN ACTIN BINDING DOMAIN 1 USING CRISPR GENOME EDITING
#5341Switchable CAS9 nucleases and uses thereof
#5342Strong insulator and uses thereof in gene delivery
#5343CasY compositions and methods of use
#5344CRISPR/CAS-RELATED METHODS AND COMPOSITIONS FOR TREATING BETA HEMOGLOBINOPATHIES
#5345Oligonucleotide sequences targeting transcription factor TSC22D4 for the treatment of insulin resistance
#5346Therapeutic compositions
#5347Antisense oligonucleotides for the treatment of leber congenital amaurosis
#5348miRNA biogenesis in exosomes for diagnosis and therapy
#5349NOVEL CRISPR-ASSOCIATED TRANSPOSON SYSTEMS AND COMPONENTS
#5350NOVEL CRISPR-ASSOCIATED TRANSPOSON SYSTEMS AND COMPONENTS
#5351METHODS AND COMPOSITIONS FOR ALTERING FUNCTION AND STRUCTURE OF CHROMATIN LOOPS AND/OR DOMAINS
#5352Stabilized HNF4A saRNA compositions and methods of use
#5353HNF4A saRNA compositions and methods of use
#5354BIOLOGICAL MATERIALS AND THERAPEUTIC USES THEREOF
#5355PROSS OPTIMIZED ENZYMES
#5356Facile assembly of soft nanoarchitectures and co-loading of hydrophilic and hydrophobic molecules via flash nanoprecipitation
#5357Method for treating neurological conditions and improving human cognition
#5358Virus for treatment of tumor
#5359Methods of treating an ischemic disease
#5360Therapeutic pharmaceutical composition for cancer including miRNA
#5361Compositions for inhibiting 3′ repair exonuclease 2 and methods of screening for such compositions
#5362TREATMENT OF A DISEASE OF THE GASTROINTESTINAL TRACT WITH AN INTEGRIN INHIBITOR
#5363RNA targeting methods and compositions
#5364Modifying the specificity of plant non-coding RNA molecules for silencing gene expression
#5365POLYNUCLEOTIDE AGENTS TARGETING PATATIN-LIKE PHOSPHOLIPASE DOMAIN CONTAINING 3 (PNPLA3) AND METHODS OF USE THEREOF
#5366Antisense oligomers targeting PCSK9
#5367Compositions and Methods for TTR Gene Editing and Treating ATTR Amyloidosis
#5368MiRNA Detargeting System for Tissue Specific Interference
#5369COMBINATION THERAPIES FOR TREATING MUSCULAR DYSTROPHY
#5370SMALL GUIDE ANTISENSE NUCLEIC ACID AND USE THEREOF
#5371Agents that modulate TMEM230 as angiogenesis regulators and that detect TMEM230 as markers of metastasis
#5372Methods and compositions for modulating splicing
#5373COMPOSITIONS AND METHODS FOR TREATING INFLAMMATORY BOWEL DISEASES
#5374TARGETING GDF6 AND BMP SIGNALING FOR ANTI-MELANOMA THERAPY
#5375METHODS OF TREATING MOTOR NEURON DISEASES
#5376Methods and Compositions for RNAi Mediated Inhibition of Gene Expression in Mammals
#5377Antisense oligonucleotide and composition for prevention or treatment of glycogen storage disease type Ia
#5378Drug delivery systems containing oxidized cholesterols
#5379RNA targeting methods and compositions
#5380METHODS FOR DIAGNOSING HUNTINGTON'S DISEASE
#5381Bioinformatics methods of in silico validation and selection of circRNAs
#5382Sensor for cyanuric acid detection
#5383Treatment of amyotrophic lateral sclerosis (ALS)
#5384MULTI-CONJUGATE OF SIRNA AND PREPARING METHOD THEREOF
#5385Control of plant pests using RNA molecules
#5386Compositions and methods for inhibition of expression of protein C (PROC) genes
#5387Channel modulators
#5388Compositions and methods for modulation of protein aggregation
#5389Lipid formulated compositions and methods for inhibiting expression of serum amyloid A gene
#5390Methods and means for efficient skipping of exon 45 in Duchenne muscular dystrophy pre-mRNA
#5391Antisense oligonucleotides for treatment of cancer stem cells
#5392METHODS OF TREATING DIABETES AND/OR PROMOTING SURVIVAL OF PANCREATIC ISLETS AFTER TRANSPLANTATION
#5393Compositions for modulating expression of C9ORF72 antisense transcript
#5394COMPOSITIONS FOR MODULATING C9ORF72 EXPRESSION
#5395Morpholino modified oligomeric compounds
#5396Compositions and methods for regulating proteins and nucleic acids activities
#5397Tracking and manipulating cellular RNA via nuclear delivery of CRISPR/CAS9
#5398Delivery of hydrophobic active agent particles
#5399MOLECULES TARGETING SURVIVAL MOTOR NEURON 2
#5400Nanoparticle conjugates and uses thereof