109047 ⎘
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 General methods applicable to biologically active non-coding nucleic acids
Compositions and methods for delivery of biomacromolecule agents
#2402Method for blocking miRNA
#2403Chimeric double-stranded nucleic acid
#2404Recombinant protein-based method for the delivery of silencer RNA to target the brain
#2405SINGLE-CHAIN CIRCULAR RNA AND METHOD OF PRODUCING THE SAME
#2406TAT-INDUCED CRISPR/ENDONUCLEASE-BASED GENE EDITING
#2407COMPOSITIONS AND METHODS FOR PREPARING SHORT RNA MOLECULES AND OTHER NUCLEIC ACIDS
#2408Dendronized polymers for nucleic acid delivery
#2409NOVEL LIPID FORMULATIONS FOR DELIVERY OF THERAPEUTIC AGENTS TO SOLID TUMORS
#2410MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
#2411NON-VIRAL NANOPARTICLE-BASED DELIVERY SYSTEM
#2412NOVEL LIPIDS AND COMPOSITIONS FOR THE DELIVERY OF THERAPEUTICS
#2413Conjugation of lipophilic albumin-binding moiety to RNA for improved carrier-free in vivo pharmacokinetics and gene silencing
#2414Particle-nucleic acid conjugates and therapeutic uses related thereto
#2415siRNA sequence-independent modification formats for reducing off-target phenotypic effects in RNAi, and stabilized forms thereof
#2416Compositions and methods for inhibition of nucleic acids function
#2417Compositions and methods for nucleic acid transfer
#2418Functional screening with optimized functional CRISPR-Cas systems
#2419COMPOSITIONS AND METHODS FOR ENHANCING THE THERAPEUTIC POTENTIAL OF STEM CELLS
#2420Therapeutic nanoparticles and methods of use thereof
#2421Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#2422Cationic lipid
#2423Guide RNA with chemical modifications
#2424Preeclampsia
#2425Delivery of RNA to different cell types
#2426Oligonucleotide lipid nanoparticle compositions, methods of making and methods of using the same
#2427Nucleic acid-containing lipid particles and related methods
#2428Double-stranded ribonucleic acid for adjuvants
#2429Compositions and methods for modulating TTR expression
#2430Zn-DPA complex compounds and siRNA delivery systems containing the same
#2431Gene vector
#2432Use and production of CHD8+/− transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
#2433Lipids and compositions for the delivery of therapeutics
#2434COMPOSITIONS AND METHODS FOR EFFICACIOUS AND SAFE DELIVERY OF SIRNA USING SPECIFIC CHITOSAN-BASED NANOCOMPLEXES
#2435ANTISENSE POLYNUCLEOTIDES TO INDUCE EXON SKIPPING AND METHODS OF TREATING DYSTROPHIES
#2436Therapeutic miRNAs for treating heart and skeletal muscle diseases
#2437RNAi molecule delivery platform based on single-siRNA and shRNA nanocapsules
#2438Identification of a 5-Gene Expression Signature Predicting Clinical Outcome of Patients with Brain Tumors
#2439Use of aptamers in proteomics
#2440Compositions and methods using capsids resistant to hydrolases
#2441Multimeric oligonucleotide compounds having non-nucleotide based cleavable linkers
#2442siRNA compounds comprising terminal substitutions
#2443Acyl-amino-LNA and/or hydrocarbyl-amino-LNA oligonucleotides
#2444Hybrid oligonucleotides and uses thereof
#24452′-methoxy substituted oligomeric compounds and compositions for use in gene modulations
#2446Delivering functional nucleic acids to mammalian cells via bacterially-derived, intact minicells
#2447Peptide/particle delivery systems
#2448Disulfide-containing alkyne linking agents
#2449Morpholino oligonucleotide manufacturing method
#2450Methods and agents to increase therapeutic dystrophin expression in muscle
#2451EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#2452Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for modeling competition of multiple cancer mutations in vivo
#2453Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#2454Dually derivatized chitosan nanoparticles and methods of making and using the same for gene transfer in vivo
#2455Use of homology direct repair to record timing of a molecular event
#2456Compositions and methods thereof for down-regulation of genes following oral delivery of an RNAi molecule bioencapsulated within plant cells
#2457COMPOUNDS AND METHODS FOR IMPROVED CELLULAR UPTAKE OF ANTISENSE COMPOUNDS
#2458Reducing intron retention
#2459COMPOSITIONS AND METHODS
#2460EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#2461Heterodimeric Cas9 and methods of use thereof
#2462Method for identification of anti-HIV human miRNA mimics and miRNA inhibitors and anti-HIV pharmaceutical compounds
#2463FULLY STABILIZED ASYMMETRIC SIRNA
#2464Compositions comprising alternating 2′-modified nucleosides for use in gene modulation
#2465Reduction of off-target RNA interference toxicity
#2466EXON SKIPPING COMPOSITIONS FOR TREATING MUSCULAR DYSTROPHY
#2467RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES
#2468REVERSIR TM COMPOUNDS
#2469Pyrrolobenzodiazepines and conjugates thereof
#2470HPV particles and uses thereof
#2471MUSCLE-SPECIFIC CRISPR/CAS9 EDITING OF GENES
#2472METHODS AND COMPOSITIONS FOR SELECTIVELY ELIMINATING CELLS OF INTEREST
#2473Formulations for targeted release of agents under low pH conditions and methods of use thereof
#2474Nucleic acid, medical nanoparticle, and pharmaceutical composition thereof
#2475High specificity genome editing using chemically modified guide RNAs
#2476Oligonucleotide having non-natural nucleotide at 5′-terminal thereof
#2477Method of conveniently producing genetically modified non-human mammal with high efficiency
#2478DELIVERY, USE AND THERAPEUTIC APPLICATIONS OF CRISPR SYSTEMS AND COMPOSITIONS FOR GENOME EDITING AS TO HEMATOPOIETIC STEM CELLS (HSCs)
#2479Methods and Compositions for Selecting siRNA of Improved Functionality
#2480NUCLEASE-RESISTANT DNA ANALOGUES
#2481Escorted and functionalized guides for CRISPR-Cas systems
#2482USE OF MICRORNA PRECURSORS AS DRUGS FOR INDUCING CD34-POSITIVE ADULT STEM CELL EXPANSION
#2483RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#2484Antisense design
#2485Antisense design
#2486Structurally designed shRNAs
#2487Compositions and methods of engineered CRISPR-CAS9 systems using split-nexus CAS9-associated polynucleotides
#2488Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
#2489Nucleic acid molecules inducing RNA interference, and uses thereof
#2490Chiral library screen
#2491Targeting epigenetic regulators using a bacterial delivery system
#2492Polymers and polymeric nanogels with hydrophilics encapsulation and release capabilities and methods thereof
#2493Nanoparticle
#2494Selective Reactivation of Genes on the Inactive X Chromosome
#2495RNA interference mediating small RNA molecules
#2496OLIGONUCLEOTIDES FOR GENOMIC DNA EDITING
#2497Engineered CRISPR-Cas9 nucleases with altered PAM specificity
#2498Sucrose ester based cationic gene vector and preparation method thereof
#2499Efficient delivery of therapeutic molecules in vitro and in vivo
#2500Biotin complexes for treatment and diagnosis of Alzheimer'S disease
#2501Oligonucleotide compositions with enhanced efficiency
#2502Albumin production and cell proliferation
#2503Compositions and methods for inhibiting expression of transthyretin
#2504CRISPR enzymes and systems
#2505Recombinant RNA Viruses and Uses Thereof
#2506Antisense nucleic acids
#2507Single-molecule mechanoanalytical DNA device for ultrasensitive sensing of analytes
#2508Modulatory polynucleotides
#2509AGENT FOR TREATING FIBROSIS OF THE INTESTINE
#2510Method for RGEN RNP delivery using 5′-phosphate removed RNA
#2511Branched oligonucleotides
#2512Methods for characterizing alternatively or aberrantly spliced mRNA isoforms
#2513RNA-targeting system
#2514Microrna inhibitor system and methods of use thereof
#2515Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma
#2516DEFIBROTIDE FOR USE IN PROPHYLAXIS AND/OR TREATMENT OF GRAFT VERSUS HOST DISEASE (GVHD)
#2517MiRNA and its diagnostic and therapeutic uses in diseases or conditions associated with melanoma, or in diseases or conditions associated with activated BRAF pathway
#2518Gene carrier using cell-derived nanovesicles and method for preparing the same
#2519Nucleic acids related to MiR-29B-1-5P and uses thereof
#2520Methods and compositions for use of non-coding RNA in cell culturing and selection
#2521Genetic correction of mutated genes
#2522MULTIPLE EXON SKIPPING COMPOSITIONS FOR DMD
#2523Protected guide RNAS (PGRNAS)
#2524Compositions for enhancing targeted gene editing and methods of use thereof
#2525RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#2526Therapeutic compositions
#2527Programmable polypeptide and nucleic acid nanoparticles
#2528Cas9 proteins and guiding features for DNA targeting and genome editing
#2529Antisense design
#2530Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
#2531Modified double-stranded RNA agents
#2532Chiral control
#2533Triggering RNA interference with RNA-DNA and DNA-RNA nanoparticles
#2534Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#2535Targeted elimination of bacterial genes
#2536Delivery of dsRNA to arthropods
#2537Tricyclo-DNA antisense oligonucleotides, compositions, and methods for the treatment of disease
#2538Compositions and methods for controlling insect pests
#2539Bacterially derived intact minicells that encompass plasmid free DNA and methods of using the same
#2540Use of micropeptides in order to stimulate mycorrhizal symbiosis
#2541Antisense oligonucleotides useful in treatment of Pompe disease
#2542Interfering RNA molecules
#2543Antibacterial antisense oligonucleotide and method
#2544Phosphorothioate DNAzyme complexes and use thereof
#2545Methods for treating cells containing fusion genes
#2546CRISPR enabled multiplexed genome engineering
#2547Micellic assemblies
#2548Method for affinity purification
#2549Antisense oligonucleotide directed removal of proteolytic cleavage sites, the HCHWA-D mutation, and trinucleotide repeat expansions
#2550MicroRNA compounds and methods for modulating miR-122
#2551miR-96-5p inhibitor and a screening method for the inhibitor
#2552Morpholino oligonucleotide manufacturing method
#2553Crispr enzymes and systems
#2554RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#2555Antisense oligonucleotide compositions
#2556T7 RNA polymerase variants with expanded substrate range and enhanced transcriptional yield
#2557Modifications for antisense compounds
#2558Aminoalcohol lipidoids and uses thereof
#2559ENGINEERING MAMMALIAN GENOME USING DNA-GUIDED ARGONAUTE INTERFERENCE SYSTEMS (DAIS)
#2560Programmable RNA shredding by the type III-A CRISPR-Cas system of
#2561Decreasing lactate level and increasing polypeptide production by downregulating the expression of lactate dehydrogenase and pyruvate dehydrogenase kinase
#2562Oligonucleotides for inducing paternal UBE3A expression
#2563Composition for delivery of genetic material
#2564MULTIPLE OLIGONUCLEOTIDE MOIETIES ON PEPTIDE CARRIER
#2565Antisense compounds
#2566CRISPR-CAS SYSTEMS AND METHODS FOR ALTERING EXPRESSION OF GENE PRODUCTS
#2567CRISPR-CAS-RELATED METHODS, COMPOSITIONS AND COMPONENTS FOR CANCER IMMUNOTHERAPY
#2568Composition and method of using miR-302 precursors as anti-cancer drugs for treating human lung cancer
#2569Antisense oligonucleotide directed removal of proteolytic cleavage sites from proteins
#2570Method for treating fibrosis using siRNA and a retinoid-lipid drug carrier
#2571Isolation of novel AAV's and uses thereof
#2572Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2573Isolation of novel AAV's and uses thereof
#2574TRANSPOSON FOR GENOME MANIPULATION
#2575Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#2576Reducing nonsense-mediated mRNA decay
#2577TAL-EFFECTOR NUCLEASE FOR TARGETED KNOCKOUT OF THE HIV CO-RECEPTOR CCR5
#2578Cell lines for virus production and methods of use
#2579Nucleic acid functionalized nanoparticles for therapeutic applications
#2580Amine cationic lipids and uses thereof
#2581Small molecules that enhance the activity of oligonucleotides
#2582Multifunctional RNA nanoparticles and methods of use
#2583Compositions and methods for rapid and dynamic flux control using synthetic metabolic valves
#2584Nucleic acid enzyme substrates
#2585RAAV-based compositions and methods for treating amyotrophic lateral sclerosis
#2586Ionizable cationic lipid for RNA delivery
#2587All sterile males of culicine mosquitoes: a method of creation
#2588Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#2589RNA MOLECULES AND USES THEREOF
#2590p-Ethoxy nucleic acids for liposomal formulation
#2591Compounds and methods for enhanced cellular uptake
#2592Methods for enhancing or decreasing the levels of MIR124 and MIR29 in subjects with muscular dystrophy
#2593Retinoid-lipid drug carrier
#2594Cell penetrating peptides for intracellular delivery of molecules
#2595Defibrotide for use in prophylaxis and/or treatment of graft versus host disease (GVHD)
#2596Individualized cancer therapy
#2597Nucleic acid fragment binding to target protein
#2598Binding-induced DNA nanomachines
#2599Methods for selecting competent oocytes and competent embryos with high potential for pregnancy outcome
#2600Antisense nucleic acids
#2601RNA-interference by single-stranded RNA molecules
#2602Antisense nucleic acids
#2603Methods for genomic integration
#2604Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2605METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
#2606RNA interference for the treatment of gain-of-function disorders
#2607Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
#2608Compositions for the inactivation of virus replication and methods of making and using the same
#2609SiRNA in tandem expression and uses thereof in treating chronic lymphocytic leukemia
#2610Oligomeric compounds comprising alpha-beta-constrained nucleic acid
#2611CRISPR-based compositions and methods of use
#2612CRISPR-based compositions and methods of use
#2613Linkage modified oligomeric compounds
#2614Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
#2615Aptamer-mRNA conjugates for targeted protein or peptide expression and methods for their use
#2616Bioactive conjugates for oligonucleotide delivery
#2617Compositions and methods for modulating stem cells and uses thereof
#2618Modulation of pre-MRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
#2619Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#2620Oligonucleotide analogues incorporating 5-aza-cytosine therein
#2621Method and compositions for controlling pest insects on plants by silencing genes of the chitin synthase and of the vitellogenin family, as well as alternatively by expressing the gene of a cry toxin
#2622Multivalent oligonucleotide assemblies
#2623RNAi agents modified at the 4′-C position
#2624Agents useful in treating facioscapulohumeral muscular dystrophy
#2625Methods and compositions for RNA-directed repression of transcription using CRISPR-associated genes
#2626Targeting molecule and a use thereof
#2627METHOD OF REGULATING GENE EXPRESSION
#2628Compositions and methods for nanoparticle lyophile forms
#2629MicroRNA expression in human peripheral blood microvesicles and uses thereof
#2630RNA interference in skin indications
#2631Short interfering RNA (siRNA) analogues
#2632Compositions and methods for inhibition of nucleic acids function
#2633Microbubble complexes and methods of use
#2634Multi-targeting short interfering RNAs
#2635iRNA agents with biocleavable tethers
#2636Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#2637Micro-RNA inhibitor
#2638Oligonucleotide-ligand conjugates and process for their preparation
#2639Peptide/particle delivery systems
#2640Double-stranded and single-stranded RNA molecules with 5′ triphosphates and their use for inducing interferon
#2641Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
#2642Polycation-functionalized nanoporous silicon carrier for systemic delivery of gene silencing agents
#2643Compositions comprising synthetic polynucleotides encoding CRISPR related proteins and synthetic sgRNAs and methods of use
#2644Compositions for in vivo expression of therapeutic sequences in the microbiome
#2645Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#2646In vivo production of small interfering RNAs that mediate gene silencing
#2647CRISPR hybrid DNA/RNA polynucleotides and methods of use
#2648Potent LNA oligonucleotides for the inhibition of HIF-1A expression
#2649Antisense oligonucleotides for treatment of spinal muscular atrophy
#2650Double stranded oligonucleotide compounds comprising positional modifications
#2651Targeted therapeutic nucleosides and their use
#2652RNA aptamers against BAFF-R as cell-type specific delivery agents and methods for their use
#2653Antisense design
#2654Alternative export pathways for vector expressed RNA interference
#2655Micro-RNAs and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone- associated medical conditions
#2656Nanoscale carriers for the delivery or co-delivery of chemotherapeutics, nucleic acids and photosensitizers
#2657Antiviral therapy
#2658Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#2659DNA vector production system
#2660STAT3 inhibitors and uses thereof
#2661rAAV-based compositions and methods
#2662Cationic lipid
#2663MicroRNAs and uses thereof
#2664Multilayered nanoparticle and methods of manufacturing and using the same
#2665Sequence-specific inhibition of small RNA function
#2666Therapeutic miRNAs for treating heart and skeletal muscle diseases
#2667Targeted nanocarriers for the administration of immunosuppressive agents
#2668Nucleotide derivative or salt thereof, nucleotide-derived 5′-phosphate ester or salt thereof, nucleotide-derived 3′-phosphoramidite compound or salt thereof, and polynucleotide
#2669Liposomal particles, methods of making same and uses thereof
#2670Nucleic acid inducing RNA interference modified for preventing off-target, and use thereof
#2671Compositions of asymmetric interfering RNA and uses thereof
#26723′end caps for RNAi agents for use in RNA interference
#2673Coexpression of CAS9 and TREX2 for targeted mutagenesis
#2674Complement component iRNA compositions and methods of use thereof
#2675Targeted augmentation of nuclear gene output
#2676PERMANENT GENE CORRECTION BY MEANS OF NUCLEOTIDE-MODIFIED MESSENGER RNA
#2677Compositions and methods related to a type-II CRISPR-Cas system in
#2678Guide RNA with chemical modifications
#2679Optimized small guide RNAs and methods of use
#2680Disulfide-containing alkyne linking agents
#2681Methods and compositions for multiplex RNA guided genome editing and other RNA technologies
#2682Use of microRNA precursors as drugs for inducing CD34-positive adult stem cell expansion
#2683RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#2684Methods for increasing CAS9-mediated engineering efficiency
#2685Method to trigger RNA interference
#2686Interfering RNA delivery system and uses thereof
#2687Cationic lipid
#2688MicroRNA compounds and methods for modulating miR-122
#2689Poly(amine-co-ester) nanoparticles and methods of use thereof
#2690Micellic assemblies
#2691Methods and compositions for RNA-guided treatment of HIV infection
#2692Method of modulating the activity of a nucleic acid molecule
#2693Reduced size self-delivering RNAI compounds
#2694Reducing intron retention
#2695Lipid particles with asymmetric cationic lipids for RNA delivery
#2696Nucleic acid-lipopolymer compositions
#2697Modular RNA regulators and methods
#2698Compositions and methods for selective delivery of oligonucleotide molecules to specific neuron types
#2699siRNA sequence-independent modification formats for reducing off-target phenotypic effects in RNAI, and stabilized forms thereof
#2700POTENT LNA OLIGONUCLEOTIDES FOR THE INHIBITION OF HIF-1A EXPRESSION