112289 ⎘
Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
GENE THERAPY FOR TREATMENT OF CRX-AUTOSOMAL DOMINANT RETINOPATHIES
#302TREATMENT OF MUCOPOLYSACCHARIDOSIS IVA
#303Methods of treating solid or lymphatic tumors by combination therapy
#304Endothelium-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
#305FUSOSOME COMPOSITIONS FOR T CELL DELIVERY
#306ADENO-ASSOCIATED VIRUS VECTORS BASED GENE THERAPY FOR HEREDITARY ANGIOEDEMA
#307ENGINEERED NUCLEIC ACID REGULATORY ELEMENT AND METHODS OF USES THEREOF
#308ADENO ASSOCIATED VIRAL VECTOR DELIVERY OF ANTIBODIES FOR THE TREATMENT OF DISEASE MEDIATED BY DYSREGULATED PLASMA KALLIKREIN
#309Adeno-Associated Virus Vector Delivery of Microrna-29 to Treat Muscular Dystrophy
#310NUCLEIC ACID MOLECULES CONTAINING SPACERS AND METHODS OF USE THEREOF
#311ON-BIPOLAR CELL-SPECIFIC PROMOTERS FOR OCULAR GENE DELIVERY
#312Method for delivering RNA to neurons to treat herpes infections
#313VECTOR
#314RECOMBINANT NUCLEIC ACIDS CONTAINING ALPHAHERPESVIRUS PROMOTER SEQUENCES
#315LENTIVIRAL VECTORS IN HEMATOPOIETIC STEM CELLS TO TREAT X-LINKED CHRONIC GRANULOMATOUS DISEASE
#316Gene Therapy Approaches to Mucolipidosis IV (MLIV)
#317DNA CONSTRUCT FOR TARGETING THERAPEUTIC MOLECULES TO DISEASED TISSUE BY IMMUNE CELLS
#318PROMOTER HAVING HIGH ACTIVITY IN ACTIVATED T-CELL
#319MICROGLIA SPECIFIC PROMOTERS AND METHODS OF USE THEREFORE
#320SYSTEMS AND USES THEREOF FOR CREATING SYNTHETIC TRANSCRIPTIONAL LOGIC 'AND' GATES BASED ON PRE-mRNA TRANS-SPLICING
#321COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAUOPATHY
#322Adeno-associated virus factor VIII vectors
#323TREATMENT METHODS USING ADENOVIRUS
#324APPLICATION OF TPK AS A TARGET IN ALZHEIMER'S DISEASE
#325AAV VECTORS WITH MYELIN PROTEIN ZERO PROMOTER AND USES THEREOF FOR TREATING SCHWANN CELL-ASSOCIATED DISEASES LIKE CHARCOT-MARIE-TOOTH DISEASE
#326Replication-defective arenavirus vectors
#327Chimeric protein and related genic transfer technology
#328AAV-ZYME AND USE FOR INFUSION REPLACEMENT THERAPY
#329Adeno-associated virus vector delivery of alpha-sarcoglycan and the treatment of muscular dystrophy
#330ACOUSTIC REMOTE CONTROL OF MICROBIAL IMMUNOTHERAPY
#331HYBRID PROMOTERS AND THEIR USES IN THERAPY, NOTABLY FOR TREATING TYPE II COLLAGENOPATHIES
#332COCHLEAR OUTER HAIR CELL PROMOTERS AND USES THEREOF
#333ONCOLYTIC VIRUS AND APPLICATION THEREOF, AND DRUG FOR TREATING CANCER
#334SYNP88, A PROMOTER FOR THE EXPRESSION OF GENES
#335COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS USING OTOFERLIN DUAL VECTOR SYSTEMS
#336VECTORS AND COMPOSITIONS FOR TREATING HEMOGLOBINOPATHIES
#337Compositions and methods for enhanced gene expression in cone cells
#338CHIMERIC GENE CONSTRUCTS FOR GENERATION OF FLUORESCENT TRANSGENIC ORNAMENTAL FISH
#339SYNTHETIC GENES FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA
#340Agents and Methods for Treating Viral Infections
#341ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY
#342COMPOSITIONS AND METHODS FOR TARGETING MULTINUCLEATED CELLS
#343Engineered muscle targeting compositions
#344VIRAL VECTOR MANUFACTURING METHODS
#345Fast and Accurate Three-Plasmid Oncolytic Adenovirus Recombinant Packaging System AD5MIXPLUS and Application Thereof
#346MINIGENE THERAPY
#347RECOMBINANT ADENO-ASSOCIATED VIRUS VECTORS TO TARGET MEDULLARY THYROID CARCINOMA
#348ENGINEERED MUSCLE TARGETING COMPOSITIONS
#349Composition for treatment of Crigler-Najjar syndrome
#350SYNTHETIC PRODUCTION OF SINGLE-STRANDED ADENO ASSOCIATED VIRAL DNA VECTORS
#351NOVEL METHOD
#352INDUCING IMMUNE TOLERANCE BY rAAV VECTORS
#353Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#354METHODS AND COMPOSITIONS FOR BARCODING NUCLEIC ACID LIBRARIES AND CELL POPULATIONS
#355PROMOTER SEQUENCE AND RELATED PRODUCTS AND USES THEREOF
#356COMPOSITIONS AND METHODS FOR TREATING LAMINOPATHIES
#357REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
#358DUAL EXPRESSION VECTOR FOR GENE AUGMENTATION FOR CRUMBS COMPLEX HOMOLOGUE 1 (CRB1) MUTATIONS
#359PROMOTER FOR THE EXPRESSION OF GENES
#360CLOSED-ENDED DNA (CEDNA) AND IMMUNE MODULATING COMPOUNDS
#361REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#362Gene therapeutics for fibrodysplasia ossificans progressiva
#363CRISPR/CAS9 GENE EDITING OF ATXN2 FOR THE TREATMENT OF SPINOCEREBELLAR ATAXIA TYPE 2
#364MULTIPLEXING REGULATORY ELEMENTS TO IDENTIFY CELL-TYPE SPECIFIC REGULATORY ELEMENTS
#365AUGMENTATIONS TO LENTIVIRAL VECTORS (CCLC-MGATA/ANK-CORE LCR-BETA-AS3-FB) TO INCREASE EXPRESSION
#366HYBRID PROMOTERS FOR MUSCLE EXPRESSION
#367Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
#368COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#369COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#370Targeting Vector, Nucleic Acid Composition, and Method for Constructing Liver-injured Mouse Model
#371COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#372ONE-STEP GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY VIA GENE REPLACEMENT AND ANTI-INFLAMMATION
#373Nucleic acid constructs for VA RNA transcription
#374COMPOSITIONS AND METHODS FOR TREATING NEUROCOGNITIVE DISORDERS
#375DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#376AAV GENE THERAPY FOR TREATING NEPHROTIC SYNDROME
#377SYNP5 (PROA9), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN RETINAL GANGLION CELLS
#378GENETIC TARGETING OF CELLULAR OR NEURONAL SUB-POPULATIONS
#379COMPOSITIONS AND METHODS FOR TREATING NEUROCOGNITIVE DISORDERS
#380Compositions and Methods for the Treatment of Genetic Diseases
#381NEUROD1 COMBINATION VECTOR
#382NEUROD1 and DLX2 VECTOR
#383Liver-specific viral promoters and methods of using the same
#384Adeno-associated virus vectors for treatment of Rett syndrome
#385THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE
#386Gene therapy for juvenile Batten disease
#387METHODS OF MAKING PLATELETS COMPRISING MODIFIED RECEPTORS AND USES THEREOF
#388Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#389Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#390Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#391Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#392AAV VIRIONS WITH DECREASED IMMUNOREACTIVITY AND USES THEREFOR
#393POLYNUCLEOTIDE FOR SAFER AND MORE EFFECTIVE IMMUNOTHERAPIES
#394LIVER-SPECIFIC REGULATORY NUCLEIC ACID SEQUENCES
#395NUCLEIC ACID CONSTRUCTS AND GENE THERAPY VECTORS FOR USE IN THE TREATMENT OF WILSON DISEASE
#396DNA vector for targeted gene therapy
#397Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
#398Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
#399COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS USING OTOFERLIN DUAL VECTOR SYSTEMS
#400ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN EXCITATORY CORTICAL NEURONS
#401Frataxin expression constructs having engineered promoters and methods of use thereof
#402Muscle-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
#403SYNP17 (PROB1), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN RETINAL GANGLION CELLS
#404SYNP151 (PROC29), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN RETINAL GANGLION CELLS
#405SYNP57 (PROA14), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN PHOTORECEPTORS
#406SYNP27 (PROB12), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN PROTOPLASMIC ASTROCYTES
#407REGULATORY NUCLEIC ACID SEQUENCES
#408New Tools for Improving Gene Therapy and Use Thereof
#409Porcine Thy1 gene promoter specifically expressed in neurons
#410COMPOSITIONS AND METHODS FOR TREATING WILSON'S DISEASE
#411Optimized Liver-Specific Expression Systems for FVIII and FIX
#412Compositions and methods for treating non-age-associated hearing impairment in a human subject
#413MATERIALS AND METHODS FOR THE CORRECTION OF RETINITIS PIGMENTOSA
#414SYNP194 (PROB15), A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN RETINAL GANGLION CELLS
#415Compositions and methods for treating beta-hemoglobinopathies
#416Methods for using transcription-dependent directed evolution of AAV capsids
#417Hybrid regulatory elements
#418Adeno associated virus vectors for the treatment of hunter disease
#419TREATMENT OF RETINAL DEGENERATION USING GENE THERAPY
#420THERMAL CONTROL OF T-CELL IMMUNOTHERAPY THROUGH MOLECULAR AND PHYSICAL ACTUATION
#421RECOMBINANT AAV VECTORS USEFUL FOR REDUCING IMMUNITY AGAINST TRANSGENE PRODUCTS
#422Method for preparing fish skin mucous gland bioreactor and application thereof
#423Factor IX encoding nucleotides
#424Non-human animals models of retinoschisis
#425ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#426SYNP162, a promoter for the expression of genes
#427CODON-OPTIMIZED TRANSGENE FOR THE TREATMENT OF PROGRESSIVE FAMILIAR INTRAHEPATIC CHOLESTASIS TYPE 3 (PFIC3)
#428Treatment of mucopolysaccharidosis IVA
#429GENE THERAPY FOR TREATING FAMILIAL HYPERCHOLESTEROLEMIA
#430METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY
#431GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA
#432Optimized CLN5 genes and expression cassettes and their use
#433Methods of expressing a polynucleotide of interest in the cone photoreceptors of a subject comprising the subretinal delivery of a therapeutically effective amount of a recombinant AAV9-derived vector
#434Methods of treating phenylketonuria
#435POLYNUCLEOTIDE
#436REGULATABLE GENE EDITING COMPOSITIONS AND METHODS
#437BIO-IMAGING PROBES AND METHODS FOR NON-INVASIVE DETECTION OF HUMAN UTERINE SARCOMAS
#438Viral promoters and compositions and methods of use thereof
#439SynP159, a promoter for the expression of genes
#440FURTHER IMPROVED AAV VECTORS PRODUCED IN INSECT CELLS
#441Expression cassette with a SynP160 promoter
#442Methods and materials for GALGT2 gene therapy
#443SYNP161, a promoter for the expression of genes
#444FUSOSOME COMPOSITIONS AND USES THEREOF
#445Non-human animals expressing exogenous terminal deoxynucleotidyltransferase
#446Synthetic promoters
#447CODON OPTIMIZED NUCLEIC ACID ENCODING A RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR)
#448ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#449Embryonic cell cultures and methods of using the same
#450CRISPR therapy
#451Transcription regulatory elements and uses thereof
#452CRISPR-Based Synthetic Gene Circuits as Next Generation Gene Therapy of Inner Ear
#453PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#454HINGES 1 AND/OR 4 MODIFIED DYSTROPHINS FOR DYSTROPHINOPATHY THERAPY
#455Gene expression system
#456TREATMENT OF RETINITIS PIGMENTOSA
#457RECOMBINANT PROMOTERS AND VECTORS FOR PROTEIN EXPRESSION IN LIVER AND USE THEREOF
#458VIRAL VECTORS ENCODING RECOMBINANT FIX WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA B
#459Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
#460Therapeutical tools and methods for treating blindness by targeting photoreceptors
#461CAS12A SYSTEMS, METHODS, AND COMPOSITIONS FOR TARGETED RNA BASE EDITING
#462ANTIBODY GENE THERAPY FOR TREATMENT AND PREVENTION OF INFECTION BY RABIES LYSSAVIRUS
#463Replication-defective arenavirus vectors
#464Recombinant adenoviruses and stem cells comprising same
#465AAV/XBP1S-HA VIRUS, GENE THERAPY METHOD AND USE THEREOF IN THE OPTIMISATION AND IMPROVEMENT OF LEARNING, MEMORY AND COGNITIVE CAPACITIES
#466Primate retinal pigment epithelium cell-specific promoter
#467Bicistronic AAV vector for RNA interference in ALS
#468Retinal Promoter and Uses Thereof
#469Methods and compositions for production of aromatic and other compounds in yeast
#470Rescuing voltage-gated sodium channel function in inhibitory neurons
#471SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium
#472Optimised coding sequence and promoter
#473Expression vectors comprising engineered genes
#474MICROVESICLE AND METHOD FOR PRODUCING THE SAME
#475Diaphragm-specific nucleic acid regulatory elements and methods and use thereof
#476Muscle-specific nucleic acid regulatory elements and methods and use thereof
#477Engineered DNA binding proteins
#478METHODS AND COMPOSITIONS FOR TREATING GENETICALLY LINKED DISEASES OF THE EYE
#479GENE THERAPY TO IMPROVE VISION
#480Optimized mini-dystrophin genes and expression cassettes and their use
#481Therapeutic retroviral vectors for gene therapy
#482METHODS OF REDOSING GENE THERAPY VECTORS
#483Motor neuron-specific expression vectors
#484METHODOLOGY FOR IDENTIFYING GENE DELIVERY VECTORS WITH RETINAL CELL SPECIFICITY IN NON-HUMAN PRIMATE
#485Recombinant constructs and transgenic fluorescent ornamental fish therefrom
#486CONSTRUCTS COMPRISING NEURONAL VIABILITY FACTORS AND USES THEREOF
#487VECTORS WITH PROMOTER AND ENHANCER COMBINATIONS FOR TREATING PHENYLKETONURIA
#488KCNK3-BASED GENE THERAPY OF CARDIAC ARRHYTHMIA
#489DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#490Globin gene therapy for treating hemoglobinopathies
#491RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
#492SYNP88, a promoter for the specific expression of genes in retinal ganglion cells
#493Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
#494Wilson's disease gene therapy
#495METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY DISEASE
#496Endothelium-specific nucleic acid regulatory elements and methods and use thereof
#497Targeted gene insertion for improved immune cells therapy
#498Lentiviral vector for treating hemoglobin disorders
#499AAV vectors targeted to the central nervous system
#500HSV vectors for delivery of NT3 and treatment of CIPN
#501Adeno-associated virus (AAV) producer cell line and related methods
#502Cell therapy with lentiviral transduced CSF2RA transgene in the treatment of hereditary pulmonary alveolar proteinosis
#503ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MUSCLE SPECIFIC MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#504High-transducing HSV vectors
#505Optimized RPE65 promoter and coding sequences
#506EXPERIMENTAL RESEARCH METHOD FOR TARGETED THERAPY OF PROSTATE CANCER BY NUCLIDE 125I-LABELED DUAL-REGULATION ONCOLYTIC ADENOVIRUS
#507ENDOTHELIAL-SPECIFIC PROMOTER SEQUENCES AND USES THEREOF
#508TISSUE SELECTIVE TRANSGENE EXPRESSION
#509ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#510Safe lentiviral vectors for targeted delivery of multiple therapeutic molecules
#511Gene therapy vectors for treatment of Danon disease
#512Expression of NKG2D activating ligand proteins for sensitizing cancer cells to attack by cytotoxic immune cells
#513Transcriptionally targeted and CpG-free plasmid for theranostic gene therapy
#514Targeted gene therapies for pain and other neuro-related disorders
#515Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#516Vectors and compositions for treating hemoglobinopathies
#517Adeno-associated virus factor VIII vectors
#518Gene-therapy vectors for treating cardiomyopathy
#519Micro-dystrophins and related methods of use
#520Modified mGluR6 promoter and methods of use
#521Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#522Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#523Expressed Barcode Libraries and Uses Thereof
#524Viral vectors for treating neurogenic detrusor overactivity
#525Engineered Cellular Pathways for Programmed Autoregulation of Differentiation
#526REGENERATING FUNCTIONAL NEURONS FOR TREATMENT OF NEURAL INJURY CAUSED BY DISRUPTION OF BLOOD FLOW
#527METHODS TO MODULATE PROTEIN TRANSLATION EFFICIENCY
#528Transdermal optogenetic peripheral nerve stimulation
#529Genetic construct
#530Liver-specific nucleic acid regulatory elements and methods and use thereof
#531DOPAMINE RECEPTOR TYPE 2 SPECIFIC PROMOTER AND METHODS OF USE THEREOF
#532NOVEL MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#533TARGETED DISRUPTION OF A CSF1-DAP12 PATHWAY MEMBER GENE FOR THE TREATMENT OF NEUROPATHIC PAIN
#534Recombinant AAV variants and uses thereof
#535Widespread gene delivery to motor neurons using peripheral injection of AAV vectors
#536Treatment of retinitis pigmentosa
#537METHODS OF USE OF INHIBITORS OF PHOSPHODIESTERASES AND MODULATORS OF NITRIC OXIDE, REACTIVE OXYGEN SPECIES, AND METALLOPROTEINASES IN THE TREATMENT OF PEYRONIE'S DISEASE, ARTERIOSCLEROSIS AND OTHER FIBROTIC DISEASES
#538Methods and compositions for inhibiting or reducing hair loss, acne, rosacea, prostate cancer, and BPH
#539SYNP1, A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN INTERNEURONS
#540SYNP107, A PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN INTERNEURONS
#541AAV vectors targeted to the central nervous system
#542Gene therapy for treating citrullenemia
#543AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#544Fertility restoration gene in wheat and uses thereof
#545ERYTHROID-SPECIFIC PROMOTER AND METHOD OF USE THEREOF
#546AAV virions with decreased immunoreactivity and uses therefor
#547Method of treatment
#548Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#549TRPV1 modulatory gene product that affects TRPV1-specific pain behavioral responses identified in a functional screen of an HSV-based cDNA library
#550Transcription modulation in animals using CRISPR/Cas systems
#551CODON OPTIMIZED NUCLEIC ACID ENCODING A RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR)
#552Promoter for cell-specific gene expression and uses thereof
#553METHOD FOR AUGMENTING VISION IN PERSONS SUFFERING FROM PHOTORECEPTOR CELL DEGENERATION
#554Recombinant bagworm silk
#555Treatment of diseases by liver expression of an enzyme which has a deoxyribonuclease (DNase) activity
#556Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
#557SPATIOTEMPORAL REGULATORS
#558Adeno-associated virus vector delivery of B-sarcoglycan and microrna-29 and the treatment of muscular dystrophy
#559Methods and compositions for treating diseases and disorders of the nervous system
#560GENETIC ERASERS
#561Oncolytic expression vector comprising a nucleic acid comprising a nucleotide sequence encoding GADD34
#562Liver-specific constructs factor VIII expression cassettes and methods of use thereof
#563Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells
#564Liver-specific constructs and methods of use thereof
#565Immunomodulatory Oncolytic Adenoviral Vectors, and Methods of Production and Use Thereof for Treatment of Cancer
#566Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
#567Compositions and methods for treating beta-hemoglobinopathies
#568TRANSGENE EXPRESSION IN AVIANS
#569Lineage reporter synthetic chromosomes and methods of use
#570Non-invasive in vivo imaging and methods for treating diabetes
#571Methods of treating bladder cancer by combination therapy comprising the oncolytic adenovirus CG0070 and an immune checkpoint inhibitor
#572Replication-defective arenavirus vectors
#573Adeno-associated virus vector delivery of microRNA-29 and micro-dystrophin to treat muscular dystrophy
#574Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
#575SynP161, a promoter for the specific expression of genes in rod photoreceptors
#576Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#577Treatment methods using adenovirus
#578Liver-specific nucleic acid regulatory elements and methods and use thereof
#579Therapeutic retroviral vectors for gene therapy
#580Synthetic promoters and uses thereof
#581Gene therapy for juvenile batten disease
#582DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#583Tissue selective transgene expression
#584Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#585Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#586Adeno-associated virus vectors for treatment of glycogen storage disease
#587Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#588Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#589Human ARCAP transgenic mouse
#590Gene therapy for treating familial hypercholesterolemia
#591PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#592Methods and compositions for production of aromatic and other compounds in yeast
#593Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#594Lentiviral vector expressing membrane-anchored or secreted antibody
#595Composition for treatment of Crigler-Najjar syndrome
#596Reporter system for detecting and targeting activated cells
#597SYNP159, a promoter for the specific expression of genes in rod photoreceptors
#598Genetic construct
#599Gene therapy for the treatment of a retinal degeneration disease
#600Gene therapy for the treatment of a disease of retinal cone cells