112289 ⎘
Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
SynP160, a promoter for the specific expression of genes in rod photoreceptors
#602Viral vector targeting cancer stem cells
#603Fetal hemoglobin for genetic correction of sickle cell disease
#604SYNP162, a promoter for the specific expression of genes in rod photoreceptors
#605Recombinant promoters and vectors for protein expression in liver and use thereof
#606Reagents and methods for modulating cone photoreceptor activity
#607Methods of treating muscular dystrophy
#608Viral vectors encoding recombinant fix with increased expression for gene therapy of hemophilia B
#609Tissue selective transgene expression
#610Compositions and methods for enhanced gene expression in cone cells
#611OLIG1 mini-promoters: PIe305
#612METHODS 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
#613Expression systems
#614Nucleic acid molecules containing spacers and methods of use thereof
#615Promoter for the specific expression of genes in retinal endothelial cells
#616AAV VIRIONS WITH DECREASED IMMUNOREACTIVITY AND USES THEREFOR
#617Motor neuron-specific expression vectors
#618Porcine Thy1 gene promoter specifically expressed in neurons
#619Therapeutical tools and methods for treating blindness by targeting photoreceptors
#620Selective gene therapy expression system
#621Non-human animal models of retinoschisis
#622Gene expression system using alternative splicing in insects
#623Methods and materials for GALGT2 gene therapy
#624Tumor suppressor SALL1 as a therapeutic agent for treating cancer
#625Method for expression of small RNA molecules within a cell
#626Bicistronic AAV vector for RNA interference in ALS
#627Methods and compositions for treating genetically linked diseases of the eye
#628Restoration of Visual Responses by In Vivo Delivery of Rhodopsin Nucleic Acids
#629Adenoviral vector encoding human atonal homolog-1 (HATH1)
#630NR2E1 MINI-PROMOTERS
#631Gene therapy methods
#632Recombinant adeno-associated virus vectors to target medullary thyroid carcinoma
#633Variant AAV and Compositions, Methods and Uses for Gene Transfer to Cells, Organs and Tissues
#634Cancer specific-splicing ribozyme and use thereof
#635Immunomodulatory oncolytic adenoviral vectors, and methods of production and use thereof for treatment of cancer
#636Self-limiting viral vectors encoding nucleases
#637In vivo production of small interfering RNAs that mediate gene silencing
#638ADENO-ASSOCIATED VIRUS FACTOR VIII VECTORS
#639PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#640Micro-dystrophins and related methods of use
#641Nucleic acid construct with a p16 promoter that causes a prodrug converting enzyme to be expressed specifically in senescent cells
#642RECOMBINANT AAV VECTORS FOR GENE THERAPY OF HUMAN HEMATOPOIETIC DISORDERS
#643Oncolytic HSV1 vector and methods of use
#644Therapeutical tools and methods for treating blindness
#645Transgenic mouse model for dementia
#646Methods and compositions for restoration of cone function in BCM
#647Smooth muscle specific inhibition for anti-restenotic therapy
#648DUAL AAV VECTOR SYSTEM FOR CRISPR/Cas9 MEDIATED CORRECTION OF HUMAN DISEASE
#649Materials and method for modifying a biochemical component in a plant
#650ADENOVIRAL TUMOR DIAGNOSTICS
#651RAAV-GUANYLATE CYCLASE COMPOSITIONS AND METHODS FOR TREATING LEBER'S CONGENITAL AMAUROSIS-1 (LCA1)
#652Compositions and method for reducing seizures
#653Optimized liver-specific expression systems for FVIII and FIX
#654Recombinant constructs and transgenic fluorescent ornamental fish therefrom
#655Chimeric gene constructs for generation of fluorescent transgenic ornamental fish
#656rAAV vector compositions, methods for targeting vascular endothelial cells and use in treatment of type I diabetes
#657Compositions and methods for treating diseases
#658Use and production of CHD8+/− transgenic animals with behavioral phenotypes characteristic of autism spectrum disorder
#659RP2 and RPGR vectors for treating X-linked retinitis pigmentosa
#660GENE AUGMENTATION THERAPIES FOR INHERITED RETINAL DEGENERATION CAUSED BY MUTATIONS IN THE PRPF31 GENE
#661DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#662AAV-mediated gene therapy for RPGR X-linked retinal degeneration
#663Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#664GENE THERAPY TO IMPROVE VISION
#665Recombinant AAV variants and uses thereof
#666METHOD AND COMPOSITION TO INCREASE RADIATION-INDUCED TUMOR THERAPEUTIC EFFECTS
#667Synthetic promoters for high throughput screening and gene modulation
#668Optimised coding sequence and promoter
#669GENETICALLY MODIFIED MESENCHYMAL STEM CELLS EXPRESSING ALPHA-1 ANTITRYPSIN (AAT)
#670Compositions for treating dystroglycanopathy disorders
#671OPTIMIZED MINI-DYSTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE
#672AAV/XBP1s-HA virus, gene therapy method and use thereof in the optimisation and improvement of learning, memory and cognitive capacities
#673AAV vectors targeted to the central nervous system
#674Insect muscle actin promoter
#675Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson's disease and other conditions
#676Methods and compositions for transducing lymphocytes and regulating the activity thereof
#677Further improved AAV vectors produced in insect cells
#678TARGETED DISRUPTION OF A CSF1-DAP12 PATHWAY MEMBER GENE FOR THE TREATMENT OF NEUROPATHIC PAIN
#679Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease
#680AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#681Non-human animals expressing exogenous terminal deoxynucleotidyltransferase
#682Dopamine receptor type 2 specific promoter and methods of use thereof
#683Recombinant promoters and vectors for protein expression in liver and use thereof
#684Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#685Composition and method for imaging stem cells
#686Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#687Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
#688Adeno-associated viral vectors for the gene therapy of metabolic diseases
#689TRPV1 modulatory gene product that affects TRPV1-specific pain behavioral responses identified in a functional screen of an HSV-based cDNA library
#690Muscle-specific nucleic acid regulatory elements and methods and use thereof
#691REGULATION OF ENDOGENOUS GENE EXPRESSION IN CELLS USING ZINC FINGER PROTEINS
#692Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#693Adeno-associated virus vectors for treatment of glycogen storage disease
#694Liver-specific nucleic acid regulatory elements and methods and use thereof
#695PAX6 minipromoters
#696GENE EXPRESSION SYSTEM
#697Globin gene therapy for treating hemoglobinopathies
#698Treatment of lysosomal storage diseases
#699Recombinant AAV vectors useful for reducing immunity against transgene products
#700CODON OPTIMIZED NUCLEIC ACID ENCODING A RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR)
#701Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#702Fetal hemoglobin for genetic correction of sickle cell disease
#703Transgenic mouse expressing kidney-specific human TGF-beta1, and biomarker for tissue fibrosis mouse model
#704Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
#705GENETICALLY MODIFIED NON-HUMAN MAMMALS HAVING MODIFIED LIVER CELLS AND/OR TISSUE AND METHODS OF MAKING SAME
#706Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#707LDLR variants and their use in compositions for reducing cholesterol levels
#708Adeno-associated virus factor VIII vectors
#709Transgenic mice expressing Y114F mutant prohibitin
#710Adenoassociated virus vectors for the treatment of lysosomal storage disorders
#711Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#712Methods of delivering a polypeptide molecule to Otx2 target cells using an Otx2 targeting peptide
#713METHODS AND COMPOSITIONS RELATING TO RESTRICTED EXPRESSION LENTIVIRAL VECTORS AND THEIR APPLICATIONS
#714Rodent model of prostate cancer
#715NOVEL METHOD FOR LABELLING CANCERIZATION-CAUSING CELL IN STEM CELLS, AND THERAPY METHOD
#716Enhanced AAV-mediated gene transfer for retinal therapies
#717Engineered Cellular Pathways for Programmed Autoregulation of Differentiation
#718ADENOVIRAL TARGETING, COMPOSITIONS AND METHODS THEREFOR
#719Pharmaceutical composition comprising Nanog shRNA, and method of using Nanog shRNA to treat cancer
#720Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#721Treatment of hyperbilirubinemia
#722Transgenic mouse having a transgene that converts a prodrug into a cytotoxic compound in senescent cells
#723Muller cell-specific promoter
#724Modified MGLUR6 promoter and methods of use
#725Method of Treating or Retarding the Development of Blindness
#726METHODS AND COMPOSITIONS FOR GENE DELIVERY TO ON BIPOLAR CELLS
#727METHOD AND COMPOSITION TO INCREASE RADIATION-INDUCED TUMOR THERAPEUTIC EFFECTS
#728Recombinant constructs and transgenic fluorescent ornamental fish therefrom
#729Synthetic Promoters
#730Non-invasive in vivo imaging and methods for treating type I diabetes
#731Compositions and methods for treating diseases
#732In vivo production of small interfering RNAs that mediate gene silencing
#733Capsid-mutated rAAV vectors and methods of use
#734Treatment of retinal degeneration using gene therapy
#735GENE THERAPY COMPOSITIONS FOR USE IN THE PREVENTION AND/OR TREATMENT OF NON-ALCOHOLIC FATTY LIVER DISEASE
#736Method of using a transgenic chicken to produce exogenous proteins in its eggs
#737Method of making polyethylene glycol-modified mammalian erythropoietin in a transgenic chicken
#738Methods and compositions for inhibiting or reducing hair loss, acne, rosacea, prostate cancer, and BPH
#739Muscle-specific nucleic acid regulatory elements and methods and use thereof
#740NOV mini-promoters
#741Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
#742Compositions and methods for treatment of muscular dystrophy
#743Artificial DNA-binding proteins and uses thereof
#744Compositions and methods for cardiac regeneration
#745Synthetic regulation of gene expression
#746Therapeutical tools and methods for treating blindness
#747Method to kill pathogenic microbes in a patient
#748Assay for drug discovery based on in vitro differentiated cells
#749Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#750Replication-defective arenavirus vectors
#751System and method for electroporating a sample
#752Cells prepared by transient transfection and methods of use thereof
#753Materials and methods for the treatment of pathological neovascularization in the eye
#754Sickled Erythrocytes and Progenitors Target Cytotoxics to Tumors
#755Retinal on bipolar cells-specific artificial promoter
#756mda-9/syntenin promoter to image and treat metastatic cancer cells
#757Gene expression system using alternative splicing in insects
#758Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
#759Tilapia () myosin light chain 3 promoter
#760Gene-therapy vectors for treating cardiomyopathy
#761-Prom mediated surface expression of avidin/streptavidin
#762Tripartite cancer theranostic nucleic acid constructs
#763INSECT MUSCLE ACTIN PROMOTER
#764Adenoviral vector encoding human atonal homolog-1 (HATH1)
#765Use of a truncated CCN1 promoter for cancer diagnostics, therapeutics and theranostics
#766Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#767Tissue preferential codon modified expression cassettes, vectors containing same, and uses thereof
#768Materials and method for increasing a plant's resistance to stress
#769Expression systems
#770Selective gene therapy expression system
#771Placental like alkaline phosphatase (PLAP) promoter mediated cell targeting
#772Delivery of polynucleotides using recombinant AAV9
#773Pharmaceutical composition comprising Nanog shRNA, and method of using Nanog shRNA to treat cancer
#774S100B mini-promoters
#775Method for augmenting vision in persons suffering from photoreceptor cell degeneration
#776Therapeutic retroviral vectors for gene therapy
#777Promoter compositions
#778TGIF2-induced reprogramming of hepatic cells to pancreatic progenitor cells and medical uses thereof
#779Constructs containing multiple expression cassettes for cancer therapy
#780REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#781Methods and compositions for treating genetically linked diseases of the eye
#782Materials and method for modifying a biochemical component in a plant
#783Enhanced AAV-mediated gene transfer for retinal therapies
#784Amyloid beta expression constructs and uses therefor
#785Retinal OFF circuit-specific promoter
#786ANIMAL MODEL WITH A FILAGGRIN GENE PROMOTER
#787Recombinant adenovirus with increased safety and anticancer activities, and use thereof
#788Adeno-associated virus (AAV) glades, sequences, vectors containing same, and uses therefor
#789NON-VIRAL VECTOR
#790TRPV1 modulatory gene product that affects TRPV1-specific pain behavioral responses identified in a functional screen of an HSV-based cDNA library
#791Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
#792Optimised coding sequence and promoter
#793Compositions and methods for enhanced gene expression in cone cells
#794FLORAL TRANSITION GENES IN MAIZE AND USES THEREOF
#795PCP2 Mini-Promoters
#796Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#797Synthetic promoter for modulating gene expression
#798Gene therapy methods
#799AAV-mediated gene therapy for RPGR X-linked retinal degeneration
#800SMOOTH MUSCLE SPECIFIC INHIBITION FOR ANTI-RESTENOTIC THERAPY
#801Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#802GPR88 MINI-PROMOTERS
#803Method for expression of small RNA molecules within a cell
#804System and method for electroporating a sample
#805Recombinant herpes virus and pharmaceutical composition containing the same
#806Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#807MATERIALS AND METHODS FOR MAKING A RECESSIVE GENE DOMINANT
#808Endothelial cell-specific polynucleotides and use thereof
#809Methods for delivery to the central nervous system of nucleic acid nanoparticles to treat central nervous system disorders
#810Vectors and sequences for the treatment of diseases
#811Adeno-associated virus factor VIII vectors
#812Viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
#813Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
#814Non-invasive in vivo imaging and methods for treating type I diabetes
#815Transgenic animal model of mood disorders
#816Brain-specific enhancers for cell-based therapy
#817Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
#818Mito-Ob: a transgenic mouse model for obesity
#819CLDN5 mini-promoters
#820Variant AAV and compositions, methods and uses for gene transfer to cells, organs and tissues
#821Methods of inducing regulated pancreatic hormone production in non-pancreatic islet tissues
#822Adenoviral tumor diagnostics
#823Method of treating or retarding the development of blindness
#824Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
#825Methods and compositions relating to restricted expression lentiviral vectors and their applications
#826AAV1-caspase gene therapy induced pyroptosis for the treatment of tumors
#827Chimeric promoter for cone photoreceptor targeted gene therapy
#828APC-mediated tolerance induction for therapy of multiple sclerosis
#829Engineered cellular pathways for programmed autoregulation of differentiation
#830Recombinant constructs and transgenic fluorescent ornamental fish therefrom
#831Infectivity-enhanced conditionally-replicative adenovirus and uses thereof
#832In vivo production of small interfering RNAs that mediate gene silencing
#833Use of integrase for targeted gene expression
#834Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
#835Lentiviral vectors containing an MHC class I promoter
#836Reagents and methods for modulating cone photoreceptor activity
#837System and method for electroporating a sample
#838Micro-RNAs of the miR-15 family modulate cardiomyocyte survival and cardiac repair
#839Replication-defective arenavirus vectors
#840Method of treatment
#841Targeting pseudotyped retroviral vectors
#842Methods for use of a specific anti-angiogenic adenoviral agent
#843Chimeric gene constructs for generation of fluorescent transgenic ornamental fish
#844Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#845Gene delivery system having enhanced tumor-specific expression, and recombinant gene expression regulating sequence
#846Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
#847MIR-21 promoter driven targeted cancer therapy
#848Growth-regulated viral vector containing the aurora kinase promoter
#849Hexon Tat-PTD modified adenovirus and uses thereof
#850Therapeutic strategies to treat CNS pathology in mucopolysaccharidoses
#851Promoters exhibiting endothelial cell specificity and methods of using same
#852Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#853Nucleic acids encoding FOXD3 promoter and methods to isolate FOXD3 expressing cells
#854Polynucleotide constructs, pharmaceutical compositions and methods for targeted downregulations of angiogenesis and anticancer therapy
#855Synthetic regulation of gene expression
#856Delivery of MECP2 polynucleotide using recombinant AAV9
#857rAAV-guanylate cyclase compositions and methods for treating lebers congenital amaurosis-1 (LCA1)
#858Preparation of recombinant human plasma phospholipid transfer protein (PLTP) from the milk of transgenic rabbits
#859Transgenic ungulates expressing CTLA4-IG and uses thereof
#860Vectors and sequences for the treatment of diseases
#861Transgenic fish and uses thereof
#862Methods and compositions for gene therapy and GHRH therapy
#863Tumor specific promoter and oncolytic virus vector comprising the same
#864Novel Therapeutical Tools and Methods for Treating Blindness
#865Methods of Producing Adenovirus Vectors and Viral Preparations Generated Thereby
#866Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#867Optimised coding sequence and promoter
#868Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
#869Use of Light Sensitive Genes
#870Gene therapy vectors for adrenoleukodystrophy and adrenomyeloneuropathy
#871Methods and compositions for expressing negative-sense viral RNA in canine cells
#872Anti-cancer therapeutic strategy to overcome cancer resistance and to enable tailoring treatment to patients
#873Compositions and Methods for Treating Diseases
#874Modified INSM1-promoter for neuroendocrine tumor therapy and diagnostics
#875Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
#876Regulation of endogenous gene expression in cells using zinc finger proteins
#877DNA regulatory element for the expression of transgenes in neurons of a subject and uses thereof
#878Constructs for enhancement of gene expression in muscle
#879Fish cancer model
#880Transgenic avians and protein production
#881Isolation, Cloning, Sequencing and Functional analysis of ss-casein promoter along with the regions of exon1, intron1 and exon2 using mammary gland derived cell line of Buffalo (Bubulus bubalis)
#882Novel Therapeutical Tools and Methods for Treating Blindness
#883Retroviral vector particles and methods for their generation and use
#884Floral transition genes in maize and uses thereof
#885Method and Composition to Increase Radiation-Induced Tumor Therapeutic Effects
#886Cardiac-specific nucleic acid regulatory elements and methods and use thereof
#887TUMOR-SPECIFIC BACTERIAL PROMOTER ELEMENTS
#888METHODS OF IMPROVING CARDIAC FUNCTION AND ATTENUATING AND/OR PREVENTING CARDIAC REMODELING WITH HSP20
#889Methods for inhibiting starvation of a cell
#890REGULATED EXPRESSION SYSTEMS
#891METHOD OF TREATING OR RETARDING THE DEVELOPMENT OF BLINDNESS
#892Materials and method for modifying a biochemical component in a plant
#893ENHANCER FOR PROMOTER, AND USE THEREOF
#894CRE-LOX BASED GENE KNOCKDOWN CONSTRUCTS AND METHODS OF USE THEREOF
#895INFECTIVITY-ENHANCED CONDITIONALLY-REPLICATIVE ADENOVIRUS AND USES THEREOF
#896DUAL VECTOR FOR INHIBITION OF HUMAN IMMUNODEFICIENCY VIRUS
#897Promoters exhibiting endothelial cell specificity and methods of using same
#898Targeted cell death
#899MICROBUBBLE ASSISTED VIRAL DELIVERY
#900DNA capable of inducing osteoblast-specific expression