110539 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses Use of virus as therapeutic agent, other than vaccine, e.g. as cytolytic agent
METHOD FOR THE PRODUCTION OF RECOMBINANT AAV PARTICLE PREPARATIONS
#2GENE SEQUENCE CONSTRUCT USED FOR TREATMENT OF CENTRAL NERVOUS SYSTEM DISEASES
#3NUCLEIC ACID REGULATORY ELEMENTS FOR GENE EXPRESSION IN THE LIVER AND METHODS OF USE
#4METHOD FOR PRODUCING NONHUMAN PRIMATE ANIMAL MODEL OF CEREBRAL INFARCTION AND PHARMACEUTICAL COMPOSITION FOR TREATMENT OF CEREBRAL INFARCTION
#5CHIMERIC AAV AND USES THEREOF
#6DUAL AAV-MYO7A VECTORS WITH IMPROVED SAFETY FOR THE TREATMENT OF USH1B
#7Adeno-Associated Virus Factor VIII Vectors
#8GENE THERAPY
#9Intrathecal Delivery of Recombinant Adeno-Associated Virus Encoding Methyl-CPG Binding Protein 2
#10Genetic Construct
#11MATERIALS AND METHODS FOR TREATMENT OF USHER SYNDROME TYPE 2A AND/OR NON-SYNDROMIC AUTOSOMAL RECESSIVE RETINITIS PIGMENTOSA (ARRP)
#12Gene Therapy Methods for Age-Related Diseases and Conditions
#13METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#14METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#15DELIVERY AND USE OF THE CRISPR-CAS SYSTEMS, VECTORS AND COMPOSITIONS FOR HEPATIC TARGETING AND THERAPY
#16FACTOR VIII (FVIII) GENE THERAPY METHODS
#17Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof
#18Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof
#19VIRAL VECTOR CONSTRUCTS FOR DELIVERY OF NUCLEIC ACIDS ENCODING CYTOKINES AND USES THEREOF FOR TREATING CANCER
#20TREATMENT OF MUCOPOLYSACCHARIDOSIS II WITH RECOMBINANT HUMAN IDURONATE-2-SULFATASE (IDS) PRODUCED BY HUMAN NEURAL OR GLIAL CELLS
#21ADENOVIRUSES AND METHODS FOR USING ADENOVIRUSES
#22RECOMBINANT AAV FORMULATIONS
#23AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY IN GLAUCOMA
#24HIGH ACTIVITY REGULATORY ELEMENTS
#25RECOMBINANT VIRUS PRODUCTS AND METHODS FOR INHIBITION OF EXPRESSION OF DUX4
#26AAV9-MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
#27GENE THERAPIES FOR LYSOSOMAL DISORDERS
#28RAAV-BASED COMPOSITIONS AND METHODS
#29Recombinant Adeno-Associated Vectors For Targeted Treatment
#30Compositions and Methods for Producing Recombinant AAV
#31ENGINEERED LIGHT-SENSITIVE PROTEINS
#32AAV COMPOSITIONS HAVING HIGH EXPRESSION LEVELS IN BRAIN
#33Adenoviruses and methods for using adenoviruses
#34GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS IIIB
#35Adenoviruses and methods for using adenoviruses
#36ADENO-ASSOCIATED VIRUS VECTORS
#37Intrathecal Delivery of Recombinant Adeno-Associated Virus Encoding Methyl-CPG Binding Protein 2
#38AAV DELIVERY SYSTEM FOR LUNG CANCER TREATMENT
#39Immunomodulating Gene Therapy
#40TREATMENT OF GLYCOGEN STORAGE DISEASE III
#41DUAL AAV-MYO7A VECTORS WITH IMPROVED SAFETY FOR THE TREATMENT OF USH1B
#42Recombinant Adeno-Associated Virus Delivery of Exon 2-Targeted U7SNRNA Polynucleotide Constructs
#43CRISPR/Cas-related methods and compositions for treating Leber's congenital amaurosis 10 (LCA10)
#44HUMAN GENE THERAPY METHODS FOR HEMOPHILIA A
#45Adeno-associated virus factor VIII vectors
#46IMPROVED PRODUCTION OF RECOMBINANT AAV USING EMBRYONATED AVIAN EGGS
#47GENE THERAPY FOR TREATING HEMOPHILIA B
#48RAAV-based compositions and methods
#49REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#50Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
#51DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#52AAV-MEDIATED GENE THERAPY RESTORING THE OTOFERLIN GENE
#53METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#54Materials and methods for treatment of usher syndrome type 2A and/or non-syndromic autosomal recessive retinitis pigmentosa (ARRP)
#55Codon optimized otoferlin AAV dual vector gene therapy
#56COMPOSITIONS AND METHODS USEFUL FOR TARGETING THE BLOOD-BRAIN BARRIER
#57ADENOVIRUSES AND METHODS FOR USING ADENOVIRUSES
#58METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#59Adeno associated virus vectors for the treatment of hunter disease
#60CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND METHODS OF USE IN GENE THERAPY OF HUMAN LIVER CANCER
#61METHODS FOR DETERMINING THE PRESENCE OR RISK OF DEVELOPING FACIOSCAPULOHUMERAL DYSTROPHY (FSHD)
#62RECOMBINANT AAVS HAVING USEFUL TRANSCYTOSIS PROPERTIES
#63CAPSID-MODIFIED RAAV VECTORS AND METHODS OF USE
#64Recombinant adeno-associated vectors for targeted treatment
#65Efficient systemic treatment of dystrophic muscle pathologies
#66Delivery and use of the CRISPR-CAS systems, vectors and compositions for hepatic targeting and therapy
#67RAAV-based compositions and methods
#68Methods and materials for NT-3 gene therapy
#69Modulators of chromosome 9 open reading frame 72 gene expression and uses thereof
#70Large animal model for developing therapeutic agents to treat impaired ophthalmic function in usher syndrome
#71Gene therapies for lysosomal disorders
#72VECTORS CONTAINING AIMP2-DX2 AND TARGET NUCLEIC ACIDS FOR miR 142 AND USES THEREOF
#73Materials and methods for treatment of Usher syndrome type 2A and/or non-syndromic autosomal recessive retinitis pigmentosa (ARRP)
#74KCNK3-BASED GENE THERAPY OF CARDIAC ARRHYTHMIA
#75DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#76Gene therapy for mucopolysaccharidosis IIIB
#77Treatment of glycogen storage disease III
#78Compositions and methods for producing recombinant AAV
#79ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II
#80NOVEL RECOMBINANT ADENO-ASSOCIATED VIRAL VECTORS RESTRICTING OFF-TARGET TRANSDUCTION IN LIVER AND USES THEREOF
#81Life-cycle-defective adenovirus helper viruses, their production and use for producing rAAV
#82FACTOR VIII (FVIII) GENE THERAPY METHODS
#83High activity regulatory elements
#84Intrathecal delivery of recombinant adeno-associated virus encoding Methyl-CpG binding protein 2
#85Method for inducing production of vascular endothelial growth factor
#86APHERESIS METHODS AND USES
#87Genetic construct
#88METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#89Adenoviruses and methods for using adenoviruses
#90Compositions and methods of treating Huntington's disease
#91Method for Introducing Polynucleotide to Male Germ Cell or Sertoli Cell
#92Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS) produced by human neural or glial cells
#93AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
#94Adeno-associated virus factor VIII vectors
#95Engineered light-sensitive proteins
#96REGENERATING FUNCTIONAL NEURONS FOR TREATMENT OF NEURAL INJURY CAUSED BY DISRUPTION OF BLOOD FLOW
#97Genetic construct
#98Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
#99Liver targeting adeno-associated viral vectors
#100CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)
#101Acid-alpha glucosidase variants and uses thereof
#102Gene therapy methods for age-related diseases and conditions
#103Recombinant virus products and methods for inhibition of expression of DUX4
#104Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#105REMOTE CONTROL OF LIGHT-TRIGGERED VIROTHERAPY
#106AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY IN GLAUCOMA
#107Drug composition for angiogenesis therapy
#108RAAV-based compositions and methods
#109Gene therapy
#110Efficient systemic treatment of dystrophic muscle pathologies
#111Recombinant AAVS having useful transcytosis properties
#112Gene therapy for treating hemophilia B
#113METHODS AND COMPOSITIONS FOR ATTENUATING GENE EXPRESSION MODULATING ANTI-VIRAL TRANSFER VECTOR IMMUNE RESPONSES
#114DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#115Immunomodulating gene therapy
#116COMPOSITIONS AND METHODS FOR REGULATABLE ANTIBODY EXPRESSION
#117Promoters and uses thereof
#118Recombinant promoters and vectors for protein expression in liver and use thereof
#119Reagents and methods for modulating cone photoreceptor activity
#120METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#121MUTANT ADENO-ASSOCIATED VIRUS VIRIONS AND METHODS OF USE THEREOF
#122Chimeric adeno-associated virus/ bocavirus parvovirus vector
#123RAAV-BASED COMPOSITIONS AND METHODS
#124Selective gene therapy expression system
#125Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer
#126CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)
#127Recombinant adeno-associated vectors for targeted treatment
#128ADENO-ASSOCIATED VIRUS FACTOR VIII VECTORS
#129DELIVERY OF POLYNUCLEOTIDES USING RECOMBINANT AAV9
#130Capsid modified rAAV vectors and methods of use
#131RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES
#132Recombinant promoters and vectors for protein expression in liver and use thereof
#133Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma
#134METHODS AND COMPOSITIONS FOR TREATING CONDITIONS ASSOCIATED WITH SPINAL CORD INJURY
#135Recombinant adeno-associated vectors for targeted treatment
#136Recombinant adeno-associated vectors for targeted treatment
#137Recombinant adeno-associated vectors for targeted treatment
#138RAAV-BASED COMPOSITIONS AND METHODS FOR TREATING ALPHA-1 ANTI-TRYPSIN DEFICIENCIES
#139Efficient systemic treatment of dystrophic muscle pathologies
#140Compositions and methods for purifying recombinant adeno-associated virus
#141METHOD FOR INDUCING PRODUCTION OF VASCULAR ENDOTHELIAL GROWTH FACTOR
#142Adeno-associated virus factor VIII vectors
#143Recombinant adeno-associated vectors for targeted treatment
#144Recombinant virus products and methods for inhibition of expression of DUX4
#145rAAV-based compositions and methods
#146Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
#147S100 based treatment of cardiac power failure
#148Recombinant AAV-crumbs homologue composition and methods for treating LCA-8 and progressive RP
#149RAAV-based compositions and methods for treating alpha-1 anti-trypsin deficiencies
#150Cell and gene based methods to improve cardiac function
#151Delivery and use of the CRISPR-Cas systems, vectors and compositions for hepatic targeting and therapy
#152DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR TARGETING AND MODELING DISEASES AND DISORDERS OF POST MITOTIC CELLS
#153Capsid-modified rAAV vectors and methods of use
#154Immunomodulating gene therapy
#155Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#156METHODS AND COMPOSITIONS FOR ATTENUATING GENE EDITING ANTI-VIRAL TRANSFER VECTOR IMMUNE RESPONSES
#157METHODS AND COMPOSITIONS FOR ATTENUATING GENE THERAPY ANTI-VIRAL TRANSFER VECTOR IMMUNE RESPONSES
#158Methods and compositions for attenuating exon skipping anti-viral transfer vector immune responses
#159Chimeric adeno-associated virus/ bocavirus parvovirus vector
#160Methods and compositions for attenuating anti-viral transfer vector immune responses
#161Selective gene therapy expression system
#162Delivery of polynucleotides using recombinant AAV9
#163Vectors comprising stuffer/filler polynucleotide sequences and methods of use
#164METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#165REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#166Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same
#167METHOD AND COMPOSITION FOR TREATING SPASTICITY
#168HYPOXIA REGULATED CONDITIONALLY SILENCED AAV EXPRESSING ANGIOGENIC INDUCERS
#169Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to Friedreich ataxia
#170CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
#171Mutant adeno-associated virus virions and methods of use thereof
#172Method of treating or retarding the development of blindness
#173Viral vectors with modified transduction profiles and methods of making and using the same
#174Methods for alleviating facioscapulohumeral dystrophy (FSHD) by N siRNA molecule inhibiting the expression of DUX4-FL
#175Delivery of MECP2 polynucleotide using recombinant AAV9
#176FROG/TOAD conditionally silenced vectors for hypoxia gene therapy
#177METHOD OF TREATING OR RETARDING THE DEVELOPMENT OF BLINDNESS
#178Parvovirus methods and compositions for killing neoplastic cells
#179REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#180DRUG COMPOSITION FOR ANGIOGENESIS THERAPY
#181Immunomodulating gene therapy
#182AAV VECTOR COMPOSITIONS AND METHODS FOR ENHANCED EXPRESSION OF IMMUNOGLOBULINS USING THE SAME
#183CYP46A1 gene for the treatment of alzheimer's disease
#184Method for Restoring an Ejaculatory Failure
#185Method of treating or retarding the development of blindness
#186Tyrosine-modified recombinant rAAV vector compositions and methods for use
#187Mutant adeno-associated virus virions and methods of use thereof
#188Parvovirus methods and compositions for killing neoplastic cells
#189Method of Treating or Retarding the Development of Blindness
#190Recombinant adeno-associated virus expressing human antisense gene CyP2J2 and its preparation methods
#191Method of treating or retarding the development of blindness
#192AAV vector compositions and methods for enhanced expression of immunoglobulins using the same
#193Method for introducing a biological molecule using a viral envelope and heparin and system therefore