112301 ⎘
Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
COMPOSITIONS AND METHODS FOR TREATING RETINITIS PIGMENTOSA
#2NOVEL RECEPTORS FOR TRANSCRIPTION REGULATION
#3BDNF GENE THERAPY
#4VECTORS FOR PRODUCTION OF THERAPEUTIC CONSTRUCTS
#5GENE THERAPY METHODS FOR ARRHYTHMOGENIC CARDIOMYOPATHY
#6ADENO-ASSOCIATED VIRAL VECTOR COMPOSITIONS AND METHODS OF USE
#7SYSTEMS AND METHODS FOR PROMOTING TRANS-SPLICING
#8VECTORS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA
#9VEGF-BINDING MOLECULE AND PHARMACEUTICAL USE THEREOF
#10COMPOSITIONS FOR TREATING CANCER
#11GENE THERAPY FOR FAM161A-ASSOCIATED RETINOPATHIES AND OTHER CILIOPATHIES
#12GENE THERAPY COMPOSITIONS AND METHODS FOR TREATING DISEASES OF THE RETINA
#13GENETIC REGULATORY ELEMENTS AND USES THEREOF
#14COMBINATION VECTORS AND METHODS FOR TREATING CANCER
#15LENTIVIRAL VECTORS USEFUL FOR THE TREATMENT OF DISEASE
#16AAV VECTOR ENCODING DIAMINE OXIDASE AND USES THEREOF
#17ADENOVIRAL VECTOR-BASED VACCINE FOR EMERGING VIRUSES
#18ADENO-ASSOCIATED VIRUS VIRION FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
#19Recombinant Adeno-Associated Virus (rAAV) Universal Reference Standard for Determining rAAV Genome Copy Titer by Quantitative PCR
#20COMPOSITIONS AND METHODS FOR MULTIPLEX PROJECTION TRACING AND MULTI-MODAL PROFILING OF PROJECTION NEURONS USING PROJECTION-TAGs
#21LENTIVIRAL VECTORS TARGETING ANTIGENS TO MHC-II PATHWAY AND INDUCING PROTECTIVE CD8+ AND CD4+ T-CELL IMMUNITY IN A HOST
#22RECOMBINANT ADENO-ASSOCIATED VIRUS VECTOR FOR TREATMENT OF IRON-ACCUMULATING NEURODEGENERATIVE DISEASES
#23NUCLEIC ACID CONSTRUCTS AND VECTORS FOR PODOCYTE SPECIFIC EXPRESSION
#24HOMOLOGY INDEPENDENT TARGETED INTEGRATION FOR GENE EDITING
#25GENE THERAPY OF HIPPO SIGNALING IMPROVES HEART FUNCTION IN A CLINICALLY RELEVANT MODEL
#26EXPRESSION VECTORS COMPOSITION
#27B-CELL LYMPHOMA 2-ASSOCIATED ANTHANOGENE 3 (BAG3) GENE THERAPY USING AAV VECTOR
#28RT EDITING COMPOSITIONS AND METHODS
#29DNA ORIGAMI ENCODING FOR GENE EXPRESSION AND CONTRANSFECTION
#30CAPSIDS FOR PLAKOPHILLIN-2 GENE THERAPY
#31HUMAN CONE PHOTORECEPTOR OPTOGENETIC CONSTRUCTS
#32PLAKOPHILLIN-2 GENE THERAPY TREATMENT METHODS
#33PHOSPHATE SENSING MICROBIAL GENE SWITCH
#34ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN THE CEREBELLUM AND A SECONDARY CELL TYPE
#35PRODUCTION OF VIRAL VECTORS
#36ENGINEERED INVARIANT NATURAL KILLER T (INKT) CELLS AND METHODS OF MAKING AND USING THEREOF
#37COMPOSITIONS AND METHODS FOR THE TREATMENT OF GLYCOGEN STORAGE DISORDERS
#38GENETIC TARGETING OF CELLULAR OR NEURONAL SUB-POPULATIONS
#39METHODS OF TREATING OR PREVENTING PYRUVATE KINASE DEFICIENCY
#40Cochlear and/or vestibular cell specific promoter and use thereof
#41GUIDE NUCLEIC ACID TARGETING MECP2 AND USES THEREOF
#42STABLE REGULATORY T CELLS AND METHODS OF PRODUCTION
#43PROMOTERS FOR SPECIFIC EXPRESSION OF GENES IN CONE PHOTORECEPTORS
#44PROCESS FOR PRODUCING CONE PHOTORECEPTOR CELLS
#45INTRON FRAGMENTS
#46IN VITRO AND IN VIVO PROTEIN TRANSLATION VIA IN SITU CIRCULARIZED RNAS
#47METHODS OF TREATING NEURODEGENERATIVE DISORDERS
#48METHODS FOR USE OF VIRAL VECTOR CONSTRUCTS FOR THE TREATMENT OF FABRY DISEASE
#49BASE EDITING AND CRISPR/CAS9 GENE EDITING STRATEGIES TO CORRECT CD3 SEVERE COMBINED IMMUNODEFICIENCY IN HEMATOPOIETIC STEM CELLS
#50RECOMBINANT OPTIMIZED MECP2 CASSETTES AND METHODS FOR TREATING RETT SYNDROME AND RELATED DISORDERS
#51ADENO-ASSOCIATED VIRAL VECTOR FOR GLUT1 EXPRESSION AND USES THEREOF
#52Gene Therapy Methods for Age-Related Diseases and Conditions
#53RECOMBINANT VIRUS EXPRESSING TPK AND USE THEREOF IN TREATMENT OF ALZHEIMER'S DISEASE
#54LENTIVIRAL GENE THERAPY RESCUES FUNCTION IN A MOUSE MODEL OF USHER 1B
#55SCHWANN CELL-SPECIFIC PROMOTER
#56LENTIVIRAL VECTOR FOR OVEREXPRESSING EGF, RECOMBINANT STEM CELL, AND USE THEREOF
#57GENE THERAPY FOR GENETIC HEARING LOSS
#58OPTIMIZED AAV-BASED VACCINE
#59COMPOSITIONS FOR TREATING CANCER
#60COMPOSITIONS FOR TREATING CANCER
#61RECOMBINANT LENTIVIRAL VECTOR FOR STEM CELL-BASED GENE THERAPY OF SICKLE CELL DISORDER
#62WPRE MUTANT CONSTRUCTS, COMPOSITIONS, AND METHODS THEREOF
#63AAV GENE THERAPY FOR THE KIDNEY
#64THERAPY
#65AADC/GDNF POLYNUCLEOTIDE, AND USE THEREOF IN TREATING PARKINSON'S DISEASE
#66OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#67IMPROVED GENE THERAPY CONSTRUCTS FOR THE TREATMENT OF PROPIONIC ACIDEMIA CAUSED BY MUTATIONS IN PROPIONYL-COA CARBOXYLASE ALPHA
#68GENE THERAPY FOR LAMIN A-ASSOCIATED DEFICIENCIES
#69GENE THERAPY FOR TREATMENT OF MUCOPOLYSACCHARIDOSIS IIIA
#70SELF-INACTIVATING LENTIVIRAL VECTOR ENCODING BETA- OR GAMMA-GLOBIN
#71Lentiviral Vectors
#72COMPOSITIONS AND METHODS FOR SCREENING CIS REGULATORY ELEMENTS
#73NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN
#74RETGC Gene Therapy
#75AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GALACTOSIDASE AND THEIR USE TO TREAT FABRY DISEASE
#76NOVEL METHODS OF USING ADENO-ASSOCIATED VIRUS 11 (AAV11)
#77BCMA CHIMERIC ANTIGEN RECEPTORS
#78LENTIVIRAL VECTOR FOR EXPRESSION OF HUMAN PAPILLOMAVIRUS (HPV) ANTIGENS AND ITS IMPLEMENTATION IN THE TREATMENT OF HPV INDUCED CANCERS
#79INTEGRASES, LANDING PAD ARCHITECTURES, AND ENGINEERED CELLS COMPRISING THE SAME
#80Erythroid Specific Enhancers
#81OPTIMIZED CYP4V2 GENE AND APPLICATION THEREOF
#82ADENO-ASSOCIATED VECTORS AND VIRIONS TO TREAT GALACTOSEMIA AND METHODS OF USE AND MANUFACTURE
#83GENE THERAPY BASED ADMINISTRATION OF LENTIVIRUS VECTOR FOR TREATING HEMOGLOBINOPATHIES
#84TROPONIN C (TNNC1) GENE THERAPY USING AAV VECTOR
#85JUNCTOPHILIN-2 (JPH2) GENE THERAPY USING AAV VECTOR
#86RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING BIETTI CRYSTALLINE DYSTROPHY
#87LENTIVIRAL VECTOR TECHNOLOGY FOR INNER EAR GENE THERAPY
#88CONTROL OF MULTI-GENE EXPRESSION USING SYNTHETIC PROMOTERS
#89ENHANCERS AND VECTORS
#90ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR TISSUE-TARGETED EXPRESSION OF THERAPEUTIC GENES
#91COMPOSITIONS AND METHODS FOR CELL TYPE-SPECIFIC GENE EXPRESSION IN THE INNER EAR
#92ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN NEURONS WITHIN THE THALAMUS
#93LIPID PARTICLES CONTAINING A TRUNCATED BABOON ENDOGENOUS RETROVIRUS (BaEV) ENVELOPE GLYCOPROTEIN AND RELATED METHODS AND USES
#94RECOMBINANT ADENO-ASSOCIATED VIRUS HAVING VARIANT CAPSID, AND APPLICATION THEREOF
#95EXPRESSION VECTORS, BACTERIAL SEQUENCE-FREE VECTORS, AND METHODS OF MAKING AND USING THE SAME
#96VECTORS ENCODING GENE EDITING SYSTEMS AND USES THEREOF
#97HIGH EFFICIENCY TRANS-SPLICING FOR REPLACEMENT OF TARGETED RNA SEQUENCES IN HUMAN CELLS
#98IL2 TETHERED TO ITS RECEPTOR IL2RBETA AND PORE-FORMING PROTEINS AS A PLATFORM TO ENHANCE IMMUNE CELL ACTIVITY
#99METHODS OF REDOSING GENE THERAPY VECTORS
#100COMPOSITIONS AND METHODS FOR TREATING RETINAL DEGENERATIVE DISORDERS
#101Gene Therapy for Retinal Disease
#102PROMOTER FOR SPECIFIC EXPRESSION OF GENES IN ROD PHOTORECEPTORS
#103WPRE mutant constructs, compositions, and methods thereof
#104TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
#105METHOD OF DESIGNING HIGHLY REGULATED LENTIVIRAL VECTORS POSSESING STRICT ENDOGENOUS REGULATION
#106GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
#107DNA VECTORS AND ELEMENTS FOR SUSTAINED GENE EXPRESSION IN EUKARYOTIC CELLS
#108Optimized expression cassettes for gene therapy
#109RETROVIRAL VECTORS
#110COMPOSITIONS, CONSTRUCTS AND VECTORS FOR CELL REPROGRAMMING
#111MOLONEY MURINE LEUKEMIA VIRUS-BASED SELF-INACTIVATING VECTOR AND APPLICATIONS THEREOF
#112MICROGLIAL SELECTIVE GENE EXPRESSION VECTOR
#113COMPOSITIONS AND METHODS FOR TREATMENT OF FABRY DISEASE
#114CHIMERIC ANTIGEN RECEPTOR EXPRESSION SYSTEMS
#115LENTIVIRAL VECTORS FOR DELIVERY OF PKLR TO TREAT PYRUVATE KINASE DEFICIENCY
#116TREATMENT OF NEUROMUSCULAR DISEASES VIA GENE THERAPY THAT EXPRESSES KLOTHO PROTEIN
#117TRANSGENE EXPRESSION SYSTEM
#118AAV VECTORS ENCODING PARKIN AND USES THEREOF
#119GENE THERAPIES FOR LYSOSOMAL DISORDERS
#120TGFß THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES
#121GENOMIC INSULATOR ELEMENT EXHIBITING ENHANCER BLOCKING ACTIVITIES IN LYMPHOCYTES AND USES THEREOF
#122VECTOR
#123COMPOSITIONS USEFUL IN TREATMENT OF CDKL5 DEFICIENCY DISORDER (CDD)
#124COMPOSITIONS AND METHODS FOR TREATING MACULAR DYSTROPHY
#125OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#126KIR 7.1 GENE THERAPY VECTORS AND METHODS OF USING THE SAME
#127OPTIMIZED FACTOR VIII GENES
#128ADENO-ASSOCIATED VIRUS VECTORS BASED GENE THERAPY FOR HEREDITARY ANGIOEDEMA
#129ADENO ASSOCIATED VIRAL VECTOR DELIVERY OF ANTIBODIES FOR THE TREATMENT OF DISEASE MEDIATED BY DYSREGULATED PLASMA KALLIKREIN
#130NUCLEIC ACID LOADED FLOWABLE HYDROGELS AND COMPOSITIONS, SYSTEMS AND METHODS RELATED THERETO
#131LENTIVIRAL VECTORS IN HEMATOPOIETIC STEM CELLS TO TREAT X-LINKED CHRONIC GRANULOMATOUS DISEASE
#132Engineered invariant natural killer T (iNKT) cells and methods of making and using thereof
#133VECTORS FOR THE TREATMENT OF FRIEDREICH'S ATAXIA
#134Episomal expression, genomic integrated lentiviral vector expression and mRNA expression of Potent Immunoglobulins Including Dimeric Immunoglobulin A1 and A2 via a furin cleavage site and 2A self-processing peptide to Enable Mucosal and Hematological Based Immunity or Protection via Gene Therapy for Allergens, viruses, HIV, bacteria, infections, pathology associated proteins, systemic pathologies, cancer, toxins and unnatural viruses.
#135APPLICATION OF TPK AS A TARGET IN ALZHEIMER'S DISEASE
#136Neuroreceptor compositions and methods of use
#137RECOMBINANT TRANSFER VECTORS FOR PROTEIN EXPRESSION IN INSECT AND MAMMALIAN CELLS
#138COMBINATION VECTORS AND METHODS FOR TREATING CANCER
#139AAV DELIVERY OF NUCLEOBASE EDITORS
#140Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#141Viral Vectors for Prophylaxis and Therapy of Hemoglobinopathies
#142ADENO ASSOCIATED VIRAL VECTOR DELIVERY OF ANTIBODIES FOR THE TREATMENT OF DISEASE MEDIATED BY DYSREGULATED PLASMA KALLIKREIN
#143CLOSED-ENDED DNA (CEDNA) AND IMMUNE MODULATING COMPOUNDS
#144Methods of treating or preventing pyruvate kinase deficiency
#145COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#146COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#147RECOMBINANT VECTORS COMPRISING ARYLSULFATASE A AND THEIR USES IN STEM CELL THERAPY FOR THE TREATMENT OF METACHROMATIC LEUKODYSTROPHY
#148RECOMBINANT VECTORS SUITABLE FOR THE TREATMENT OF IPEX SYNDROME
#149AAV GENE THERAPY FOR TREATING NEPHROTIC SYNDROME
#150GENETIC TARGETING OF CELLULAR OR NEURONAL SUB-POPULATIONS
#151DLX2 VECTOR
#152ASCL1 VECTOR
#153NEUROD1 COMBINATION VECTOR
#154NEUROD1 and DLX2 VECTOR
#155Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#156Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#157Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#158Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#159Intron fragments
#160Therapy
#161Adeno associated virus vectors for the treatment of hunter disease
#162FOWL ADENOVIRUS 9 (FADV-9) VECTOR SYSTEM AND ASSOCIATED METHODS
#163ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#164METHOD OF DETECTING AND/OR IDENTIFYING ADENO-ASSOCIATED VIRUS (AAV) SEQUENCES AND ISOLATING NOVEL SEQUENCES IDENTIFIED THEREBY
#165WPRE mutant constructs, compositions, and methods thereof
#166CIRCULARIZED ENGINEERED RNA AND METHODS
#167Compositions and methods for hemoglobin production
#168Vectors for the treatment of Friedreich's ataxia
#169Adenoviral vectors with two expression cassettes encoding RSV antigenic proteins or fragments thereof
#170Lentiviral vectors for delivery of to treat pyruvate kinase deficiency
#171ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#172COMPOSITIONS AND METHODS FOR THE TREATMENT OF STARGARDT DISEASE
#173AAV COMPOSITIONS, METHODS OF MAKING AND METHODS OF USE
#174Bicistronic AAV vector for RNA interference in ALS
#175Genetically modified non-human animal with human or chimeric IL15
#176Combination vectors and methods for treating cancer
#177METHODS OF REDOSING GENE THERAPY VECTORS
#178METHODS AND COMPOSITIONS FOR EXPRESSION OF POLYPEPTIDES IN A CELL
#179DNA vectors and elements for sustained gene expression in eukaryotic cells
#180Gene therapies for lysosomal disorders
#181METHODS AND COMPOSITIONS FOR THE TREATMENT OF FABRY DISEASE
#182Recombinant lentiviral vector for stem cell- based gene therapy of sickle cell disorder
#183ENDOTHELIAL-SPECIFIC PROMOTER SEQUENCES AND USES THEREOF
#184ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#185Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#186Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#187Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#188BCMA chimeric antigen receptors
#189Catecholamine enzyme fusions
#190IL-12 IMMUNOTHERAPY FOR CANCER
#191Gene therapy methods for age-related diseases and conditions
#192CHIMERIC POST-TRANSCRIPTIONAL REGULATORY ELEMENT
#193Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#194Methods of treating or preventing pyruvate kinase deficiency
#195OPTIMIZATION OF DETERMINANTS FOR SUCCESSFUL GENETIC CORRECTION OF DISEASES, MEDIATED BY HEMATOPOIETIC STEM CELLS
#196Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord
#197FOWL ADENOVIRUS 9 (FADV-9) VECTOR SYSTEM AND ASSOCIATED METHODS
#198Combination vectors and methods for treating cancer
#199Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#200Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#201Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#202Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#203Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#204Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#205Lentiviral vector expressing membrane-anchored or secreted antibody
#206Gene therapy for the treatment of a retinal degeneration disease
#207LENTIVIRAL VECTORS AND USES THEREOF
#208Method for expression of small RNA molecules within a cell
#209Bicistronic AAV vector for RNA interference in ALS
#210Cancer specific-splicing ribozyme and use thereof
#211OPTIMIZATION OF DETERMINANTS FOR SUCCESSFUL GENETIC CORRECTION OF DISEASES, MEDIATED BY HEMATOPOIETIC STEM CELLS
#212AAV delivery of nucleobase editors
#213Compositions and method for reducing seizures
#214Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord
#215Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#216Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for modeling competition of multiple cancer mutations in vivo
#217Viral vectors for prophylaxis and therapy of hemoglobinopathies
#218OPTIMIZATION OF DETERMINANTS FOR SUCCESSFUL GENETIC CORRECTION OF DISEASES, MEDIATED BY HEMATOPOIETIC STEM CELLS
#219Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#220Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#221Engineered invariant natural killer T (iNKT) cells and methods of making and using thereof
#222Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#223Globin gene therapy for treating hemoglobinopathies
#224TRANSPOSON FOR GENOME MANIPULATION
#225Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#226LENTIVIRAL VECTOR FOR STEM CELL GENE THERAPY OF SICKLE CELL DISEASE
#227Chimeric post-transcriptional regulatory element
#228Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#229Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#230Vectors and methods for the efficient generation of integration/transgene-free induced pluripotent stem cells from peripheral blood cells
#231Methods and compositions for increasing transgene activity
#232Retroviral vectors
#233Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#234Modified hepatitis post-transcriptional regulatory elements
#235IL-12 immunotherapy for cancer
#236Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#237Compositions and methods of use thereof for identifying anti-viral agents
#238Adeno-associated virus (AAV) glades, sequences, vectors containing same, and uses therefor
#239OPTIMIZATION OF DETERMINANTS FOR SUCCESSFUL GENETIC CORRECTION OF DISEASES, MEDIATED BY HEMATOPOIETIC STEM CELLS
#240LENTIVIRAL VECTOR FOR STEM CELL GENE THERAPY OF SICKLE CELL DISEASE
#241Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#242Methods and compositions relating to improved lentiviral vector production systems
#243Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#244Vector for the selective silencing of a gene in astrocytes
#245Method for expression of small RNA molecules within a cell
#246Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#247Vectors for transgene expression
#248Vectors and sequences for the treatment of diseases
#249Viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
#250Methods and compositions relating to restricted expression lentiviral vectors and their applications
#251Cells and methodology to generate non-segmented negative-strand RNA viruses
#252Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#253Methods of improving retroviral titer in transfection-based production system using eukaryotic cells
#254Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#255Neuronal specific targeting of caveolin expression to restore synaptic signaling and improve cognitive function in the neurodegenerative brain and motor function in spinal cord
#256Compositions and Methods for the Delivery of Biologically Active RNAs
#257Vectors and sequences for the treatment of diseases
#258Catecholamine enzyme fusions
#259Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#260Novel viral vector construct for neuron specific optimized continuous DOPA synthesis in vivo
#261Non-Integrating Retroviral Vector Vaccines
#262USE OF APOPTOSIS INHIBITING COMPOUNDS IN DEGENERATIVE NEUROLOGICAL DISORDERS
#263Optimization of determinants for successful genetic correction of diseases, mediated by hematopoietic stem cells
#264Treatment of metabolic-related disorders using hypothalamic gene transfer of BDNF and compositions therefor
#265Adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#266Kit for the optimisation of protein synthesis/secretion
#267Lentiviral vectors for the preparation of immunotherapeutical compositions
#268Gene of porcine alpha-s1 casein, a promoter of the same and use thereof
#269ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#270Compositions and Systems for the Regulation of Genes
#271Bovine immunodeficiency virus (BIV) based vectors
#272Method for detecting adeno-associated virus
#273LENTIVIRUS-BASED IMMUNOGENIC VECTORS
#274Method for expression of small RNA molecules within a cell
#275RECOMBINANT ALPHAVIRUS-BASED VECTORS WITH REDUCED INHIBITION OF CELLULAR MACRO-MOLECULAR SYNTHESIS
#276Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
#277IL-12 immunotherapy for cancer
#278Method for expression of small antiviral RNA molecules with reduced cytotoxicity within a cell
#279MAMMARY GLAND- SPECIFIC HUMAN ERYTHEROPOIETIN EXPRESSION VECTOR, TRANSGENIC ANIMAL AND METHOD FOR PRODUCING HUMAN ERYTHROPOIETIN USING SAME
#280Cells and methodology to generate non-segmented negative-strand RNA viruses
#281Lentiviral vectors allowing RNAi mediated inhibition of GFAP and vimentin expression
#282Methods and compositions relating to improved lentiviral vector production systems
#283Adenoviral expression vectors
#284Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same, and uses therefor
#285CANCER-SPECIFIC PROMOTERS
#286Vectors capable of immortalizing non-dividing cells and cells immortalized with said vectors
#287RAAV VECTOR-BASED COMPOSITIONS AND METHODS FOR THE PREVENTION AND TREATMENT OF MAMMALIAN DISEASES
#288Vector System
#289Adenovirus vectors, packaging cell lines, compositions, and methods for preparation and use
#290Bovine immunodeficiency virus (BIV) based vectors
#291TRANSGENIC ORGANISM
#292Amyloid peptide inactivating enzyme to treat alzheimer's disease peripherally
#293BISPECIFIC ANTIBODIES
#294GFAP-BASED GENE THERAPY FOR TREATMENT OF RETINAL DISEASES
#295Host Cells Containing Multiple Integrating Vectors
#296Compositions and methods for enhanced expression of recombinant polypeptides from a single vector using a peptide cleavage site
#297Lentiviral packaging constructs
#298Expression systems using mammalian beta-actin promoter
#299Means and methods for regulating gene expression
#300Treating immunodeficiencies by intrathymic injection of nucleotide sequences