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
RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
#2102Oligomeric compounds and compositions for use in modulation of small non-coding RNAs
#2103IN SITU AND IN VIVO ANALYSIS OF CHROMATIN INTERACTIONS BY BIOTINYLATED DCAS9 PROTEIN
#2104Adeno-associated virus vector delivery of microRNA-29 and micro-dystrophin to treat muscular dystrophy
#2105Microbubble complexes and methods of use
#2106POLYNUCLEOTIDE-POLY(DIOL) CONJUGATES, PROCESS OF PREPARATION AND USES THEREOF
#2107CRISPR mediated recording of cellular events
#2108Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
#2109Micro-RNAS and compositions comprising same for the treatment and diagnosis of serotonin-, adrenalin-, noradrenalin-, glutamate-, and corticotropin-releasing hormone-associated medical conditions
#2110Polynucleotide nanoparticles for the modulation of gene expression and uses thereof
#2111COMPOSITIONS AND METHODS FOR MODULATING HBV EXPRESSION
#2112PRNA three-way junctions
#2113LNA-G Process
#2114MIRNA PROFILING COMPOSITIONS AND METHODS OF USE
#2115COMPOSITIONS AND METHODS FOR DELIVERING MICRORNA
#2116Anti-angiogenic miRNA therapeutics for inhibiting corneal neovascularization
#2117Reduced size self-delivering nucleic acid compounds targeting long non-coding RNA
#2118Compositions and Methods for Recombinant CXADR Expression
#2119Pharmaceutical composition for treating and/or preventing cancer
#2120Liposomes for modulating Wiskott-Aldrich syndrome protein
#2121Thermostable protease and methods of making and using the same
#2122Decreasing lactate level and increasing polypeptide production by down regulating the expression of lactate dehydrogenase and pyruvate dehydrogenase kinase
#2123TRACKING AND MANIPULATING CELLULAR RNA VIA NUCLEAR DELIVERY OF CRISPR/CAS9
#2124METHODS AND COMPOSITIONS FOR IMPROVING PLANT TRAITS
#2125Methods for performing antisense oligonucleotide-mediated exon skipping in the retina of a subject in need thereof
#2126CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
#2127NUCLEIC ACID-CONTAINING LIPID PARTICLES AND RELATED METHODS
#2128Process for mixing nucleic acids with transfection reagents for delivery
#2129RNA nanoparticle for treatment of gastric cancer
#2130Crispr enzyme mutations reducing off-target effects
#2131Preparation method for a DNA origami based carrier system
#2132In vitro toxicity screening assay
#2133Interfering RNA molecules
#2134RNA ligand-displaying exosomes for specific delivery of therapeutics to cell by RNA nanotechnology
#2135Methods and compositions for inhibiting detoxification response
#2136Gene modification assays
#2137Oligonucleotides
#2138METHOD FOR ANALYSIS OF AN RNA MOLECULE
#2139Methods for genomic integration
#2140Transposon that promotes functional DNA expression in episomal DNAs and method to enhance DNA transcription during functional analysis of metagenomic libraries
#2141Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#2142Oligonucleotide compositions with enhanced efficiency
#2143Dual molecular delivery of oligonucleotides and peptide containing conjugates
#2144Liposomes comprising a calcium phosphate-containing precipitate
#2145METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2146RNA INTERFERENCE MEDIATED INHIBITION OF GENE EXPRESSION USING CHEMICALLY MODIFIED SHORT INTERFERING NUCLEIC ACID (siNA)
#2147Materials and methods for treatment of alpha-1 antitrypsin deficiency
#2148Crispr enzyme mutations reducing off-target effects
#2149METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2150METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2151METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2152CRISPR RNA targeting enzymes and systems and uses thereof
#2153Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#2154RNA-interference-inducing nucleic acid molecule able to penetrate into cells, and use therefor
#2155DOUBLE-STRANDED OLIGONUCLEOTIDES
#2156CRISPR RNA targeting enzymes and systems and uses thereof
#2157METHOD FOR GENE EDITING
#2158CRISPR enabled multiplexed genome engineering
#2159Nucleic acid-guided nucleases
#2160Compositions and methods for modulation of IKBKAP splicing
#2161Formulation comprising extracellular vesicles, method for producing the same, and uses thereof
#2162Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#2163Chimeric antigen receptor targeting human NKG2DL and methods of preparing said receptor and pharmaceutical composition
#2164Targeted augmentation of nuclear gene output
#2165RNA interference mediated inhibition of gene expression using short interfering nucleic acids (siNA)
#2166Use of a combination of Dbait molecule and PARP inhibitors to treat cancer
#2167Modified guide RNAs, CRISPR-ribonucleotprotein complexes and methods of use
#2168RNA-guided DNA nucleases and uses thereof
#2169POLYPEPTIDES WITH TYPE V CRISPR ACTIVITY AND USES THEREOF
#2170Enzymatic replacement therapy and antisense therapy for pompe disease
#2171Compositions and methods for modulating stem cells and uses thereof
#2172Methods and compositions for delivery of polynucleotides
#2173Modulation of pre-MRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
#2174Peptide, Complex of Peptide and siRNA, and Methods of Use Thereof
#2175INHIBITING GENE EXPRESSION WITH dsRNA
#2176LIPIDS FOR DELIVERY OF THERAPEUTICS
#2177Nucleic acid-containing lipid nanoparticles
#2178DNA aptamer binding to cancer cell
#2179DNA methylation editing kit and DNA methylation editing method
#2180Chemically modified multifunctional short interfering nucleic acid molecules that mediate RNA interference
#2181System and method for reversible photo-controlled gene silencing
#2182POLYNUCLEOTIDES FOR MULTIVALENT RNA INTERFERENCE, COMPOSITIONS AND METHODS OF USE THEREOF
#2183Delivery method of target material into extracellular vesicles using extracorporeal shockwave
#2184Supplementation of milk formulas with microvesicles isolated from milk
#2185CRISPR-CAS SGRNA LIBRARY
#2186Production of viruses in avian eggs
#2187Tissue selective transgene expression
#2188lincRNA for the detection of esophageal cancer
#2189CRISPR enzymes and systems
#2190RNA interference in skin indications
#2191iRNA agents with biocleavable tethers
#2192Antisense Polynucleotides to Induce Exon Skipping and Method of Treating Dystrophies
#2193Targeted self-assembly of functionalized carbon nanotubes on tumors
#2194Nucleic acid nanostructures with core motifs
#2195High-throughput CRISPR-based library screening
#2196Argonaute protein-double stranded RNA complexes and uses related thereto
#2197Toxic RNAi active seed sequences for killing cancer cells
#2198Methods and Compositions for Selecting siRNA of Improved Functionality
#2199Chimeric double-stranded nucleic acid
#2200Processes and host cells for genome, pathway, and biomolecular engineering
#2201Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2202Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2203Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2204STAT3 inhibitors and uses thereof
#2205RIG-I ligands and methods for producing them
#2206METHODS AND COMPOSITIONS FOR INCREASING SMN EXPRESSION
#2207Compounds and compositions including phosphorothioated oligodeoxynucleotide, and methods of use thereof
#2208Promoter, promoter control elements, and combinations, and uses thereof
#2209De novo synthesized nucleic acid libraries
#2210Nucleobase editors comprising nucleic acid programmable DNA binding proteins
#2211Nanoparticle formulations for delivery of nucleic acid complexes
#2212Inducible modification of a cell genome
#2213Chemically ligated RNAs for CRISPR/Cas9-1gRNA complexes as antiviral therapeutic agents
#2214Linkage modified oligomeric compounds
#2215NUCLEIC ACID-LIPOPOLYMER COMPOSITIONS
#2216METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2217POLYNUCLEOTIDES HAVING BIOREVERSIBLE GROUPS
#2218METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2219METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2220Modified AAV constructs and uses thereof
#2221SMALL INTERFERING RNA MODIFICATION METHOD BY COMBINING WITH ISONUCLEOSIDE MODIFICATION, TERMINAL PEPTIDE CONJUGATION AND CATIONIC LIPOSOMES, AND PREPARATION
#2222Cas9 variants and methods of use thereof
#2223TARGETED THERAPEUTIC NUCLEOSIDES AND THEIR USE
#2224CRISPR/Cas-related methods and compositions for improving transplantation
#2225Interfering RNA molecules
#2226COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING
#2227METHODS OF PRESERVING THE BIOLOGICAL ACTIVITY OF RIBONUCLEIC ACIDS
#2228Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2229CRISPR-associated (Cas) protein
#2230METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2231Induced exon inclusion in spinal muscle atrophy
#2232Compositions and methods for modulating apolipoprotein (a) expression
#2233Compositions and methods for modulating apolipoprotein C-III expression
#2234Methods and compositions for the production of siRNAs
#2235Methods and compositions for generating CRISPR/Cas guide RNAs
#2236LIN28- MEDIATED CONTROL OF LET-7 BIOGENESIS
#2237Biodegradable lipids for delivery of nucleic acids
#2238Biodegradable lipids for delivery of nucleic acids
#2239Biodegradable lipids for delivery of nucleic acids
#2240Biodegradable lipids for delivery of nucleic acids
#2241Methods for treating progeroid laminopathies using oligonucleotide analogues targeting human LMNA
#2242Viral vectors for the treatment of diabetes
#2243AGENTS USEFUL IN TREATING FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
#2244Short interfering nucleic acid (siNA) molecules containing a 2′ internucleoside linkage (3dT)
#2245RAAV-BASED COMPOSITIONS AND METHODS
#2246Genome engineering methods using a cytosine-specific Cas9
#2247Modified iRNA agents
#2248Conjugated antisense compounds and their use
#2249Hydrogels for delivery of therapeutic compounds
#2250Delivery, use and therapeutic applications of the CRISPR-cas systems and compositions for modeling mutations in leukocytes
#2251Method for identification of anti-HIV human miRNA mimics and miRNA inhibitors and anti-HIV pharmaceutical compounds
#2252METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2253METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2254METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2255METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2256Antisense oligonucleotide compositions
#2257Combination comprising immunostimulatory oligonucleotides
#2258Oligonucleotide conjugates
#2259Use of trinucleotide repeat RNAs to treat cancer
#2260Oligomeric compounds comprising bicyclic nucleotides and uses thereof
#2261COMPOSITIONS AND METHODS FOR PROMOTING HOMOLOGY DIRECTED REPAIR
#2262Methods and compositions for determining pH
#2263METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2264METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2265Compositions and methods of engineered CRISPR-Cas9 systems using split-nexus Cas9-associated polynucleotides
#2266Compounds and methods for trans-membrane delivery of molecules
#2267Stabilization method of functional nucleic acid
#2268METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2269UNA oligomers for therapeutics with prolonged stability
#2270ANTISENSE DESIGN
#2271ANTISENSE DESIGN
#2272Hybrid tRNA/pre-miRNA molecules and methods of use
#2273METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2274METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2275METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2276METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2277Methods and compositions for RNA-guided treatment of HIV infection
#2278METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2279METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2280METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2281METHODS AND COMPOSITIONS FOR RNA-DIRECTED TARGET DNA MODIFICATION AND FOR RNA-DIRECTED MODULATION OF TRANSCRIPTION
#2282CRISPR enabled multiplexed genome engineering
#2283CRISPR enabled multiplexed genome engineering
#2284APTAMER-GUIDED GENE TARGETING
#2285Chemically Ligated RNAs for CRISPR/Cas9-lgRNA Complexes as Antiviral Therapeutic Agents
#2286METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2287METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2288METHODS AND COMPOSITIONS FOR RNRA-GUIDED TREATMENT OF HIV INFECTION
#2289Systems and methods for nucleic acid preparation
#2290Interfering RNA molecules
#2291Multimeric oligonucleotides having decreased kidney clearance
#2292Compositions and methods for treating leukemia
#2293SITE-SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS
#2294METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2295Nuclease with enhanced efficiency of genome editing
#2296Sustained polypeptide expression from synthetic, modified RNAs and uses thereof
#2297METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2298Methods and compositions for selecting siRNA of improved functionality
#2299SCREENING METHOD FOR SELECTED AMINO-LIPID-CONTAINING COMPOSITIONS
#2300Methods and compositions for RNA-directed target DNA modification and for RNA-directed modulation of transcription
#2301Treatment of reprogramming factor related diseases by inhibition of natural antisense transcript to a reprogramming factor
#2302Multi-targeted single entity conjugates
#2303Method for introducing site-directed RNA mutation, target editing guide RNA used in the method and target RNA-target editing guide RNA complex
#2304Method of synthesis of an ionizable cationic lipid
#2305METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2306HUMAN CANCER MICRO-RNA EXPRESSION PROFILES PREDICTIVE OF CHEMO-RESPONSE
#2307Compositions, systems and methods for cell therapy
#2308Methods and compositions for selecting siRNA of improved functionality
#2309Albumin production and cell proliferation
#2310Micromirs
#2311Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#2312METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2313Methods for treating neurological disorders using a synergistic small molecule and nucleic acids therapeutic approach
#2314Transgenic microalgae and use thereof as a feed for delivery of interfering RNA molecules
#2315DISCONTINUOUS OLIGONUCLEOTIDE LIGANDS
#2316Diester and triester based low molecular weight, biodegradeable cationic lipids for oligonucleotide delivery
#2317Methods and compositions for enhancing the efficacy and specificity of single and double blunt-ended siRNA
#2318Synthetic hammerhead ribozymes with ligand-responsive tertiary interactions
#2319Nucleic acid functionalized nanoparticles for therapeutic applications
#2320NOVEL CONSTRUCTS AND SCREENING METHODS
#2321CRISPR/CPF1 systems and methods
#2322Genetically modified human cell with a corrected mutant sickle cell mutation
#2323ANTISENSE NUCLEIC ACIDS
#2324Method and Medicament For Inhibiting The Expression of A Given Gene
#2325CRISPR-BASED COMPOSITIONS AND METHODS OF USE
#2326Modified CRISPR RNA and modified single CRISPR RNA and uses thereof
#2327Composition
#2328IDENTIFICATION OF EPIGENETIC BIOMARKERS IN THE SALIVA OF CHILDREN WITH AUTISM SPECTRUM DISORDER
#2329METHODS FOR INCREASING CAS9-MEDIATED ENGINEERING EFFICIENCY
#2330Antisense molecules and methods for treating pathologies
#2331Ionizable cationic lipid for RNA delivery
#2332Self-assembled ribonucleoprotein nanoparticles
#2333METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2334Methods and compositions for RNA-guided treatment of HIV infection
#2335METHODS AND COMPOSITIONS FOR RNA-GUIDED TREATMENT OF HIV INFECTION
#2336Compositions related to rna in circularized form
#2337Combined therapy for duchenne muscular dystrophy
#2338Lipid-derived neutral nanoparticles
#2339Lipid-derived neutral nanoparticles
#2340In vivo production of small interfering RNAs that mediate gene silencing
#2341Engineering of a minimal SaCas9 CRISPR/Cas system for gene editing and transcriptional regulation optimized by enhanced guide RNA
#2342NON-INTEGRATING VIRAL DELIVERY SYSTEM AND METHODS OF USE THEROF
#2343Methods and compositions for nucleic acid and protein payload delivery
#2344COMPOSITION AND METHODS FOR REGULATING INHIBITORY INTERACTIONS IN GENETICALLY ENGINEERED CELLS
#2345P-ethoxy nucleic acids for liposomal formulation
#2346SCREENING METHODS FOR TARGETS FOR CANCER THERAPY
#2347Methods and compositions for translational profiling and molecular phenotyping
#2348Engineered nucleic acid-targeting nucleic acids
#2349RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
#2350CRISPR enzymes and systems
#2351Methods for RNA promoter identification
#2352UNA oligomers and compositions for treating amyloidosis
#2353Chemical modification of short small hairpin RNAs for inhibition of gene expression
#2354Delivering CRISPR therapeutics with lipid nanoparticles
#2355Compounds and methods for increasing antisense activity
#2356GENOME EDITING VECTORS
#2357Cells containing CRISPR hybrid DNA/RNA polynucleotides
#2358Antisense nucleic acids
#2359Method of regulating gene expression
#2360Methods and compositions for the specific inhibition of gene expression by double-stranded RNA
#2361SYNTHETIC SINGLE GUIDE RNA FOR CAS9-MEDIATED GENE EDITING
#2362Targeted nanocarriers for targeted drug delivery of gene therapeutics
#2363Methods for creating a transgenic cell with microbubbles
#2364Delivery of therapeutic agent
#2365Modified oligonucleotides and methods of use
#2366Therapeutic oligomers for treating amyloidosis
#2367COMPOSITIONS AND METHODS TO CONTROL INSECT PESTS
#2368Nucleic acid complexes for screening barcoded compounds
#2369CRISPR-related methods and compositions with governing gRNAS
#2370BIVALENT ANTISENSE OLIGONUCLEOTIDES
#2371SIRNA STRUCTURE FOR MINIMIZING OFF-TARGET EFFECTS AND RELAXING SATURATION OF RNAI MACHINERY AND THE USE THEREOF
#2372Lipids and compositions for the delivery of therapeutics
#2373Cell engineering using MICRORNAs
#2374RNA amidates and thioamidates for RNAi
#2375Methods and compositions for selecting siRNA of improved functionality
#2376Chemically modified guide RNAs for CRISPR/CAS-mediated gene regulation
#2377Segmented micro RNA mimetics
#2378Smooth muscle specific inhibition for anti-restenotic therapy
#2379DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
#2380A METHOD OF REGULATING GENE EXPRESSION
#2381Short interfering nucleic acid (siNA) compositions
#2382Cationic lipid
#2383Multiple sclerosis treatment
#2384Modulation of pre-mRNA using splice modulating oligonucleotides as therapeutic agents in the treatment of disease
#2385Exosomes and micro-ribonucleic acids for tissue regeneration
#2386Compositions and methods for the treatment of nucleotide repeat expansion disorders
#2387Methods to detect motor neuron disease comprising micro-RNAs
#2388Three-component CRISPR/Cas complex system and uses thereof
#2389Lipids and compositions for the delivery of therapeutics
#2390Oligonucleotide analogues targeting human LMNA
#2391NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#2392COMPETITIVE MODULATION OF MICRORNAS
#2393BACTERIAL MEDIATED THF ALPHA GENE SILENCING
#2394Oligonucleotide compositions with enhanced efficiency
#2395Polyvalent RNA-nanoparticle compositions
#2396POLYNUCLEOTIDE-POLY(DIOL) CONJUGATES, PROCESS OF PREPARATION AND USES THEREOF
#2397NOVEL LIPID FORMULATIONS FOR NUCLEIC ACID DELIVERY
#2398MULTI-CONJUGATE OF SIRNA AND PREPARING METHOD THEREOF
#2399Single-stranded RNAi agents containing an internal, non-nucleic acid spacer
#2400Catalytic strands of minimal hammerhead ribozymes and methods of using the same