110543 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses; Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
CALCIUM CHANNEL 3.2 INHIBITORY PEPTIDES AND USES THEREOF
#2ENGINEERED ADENO-ASSOCIATED VIRUS CAPSIDS
#3MINIGENE THERAPY
#4Adeno-Associated Virus Factor VIII Vectors
#5COMPOSITIONS AND METHODS FOR TREATMENT OF DOMINANT RETINITIS PIGMENTOSA
#6METHODS AND COMPOSITIONS FOR TARGETED GENE TRANSFER
#7METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#8METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#9METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#10COMPOSITIONS AND METHODS TO RESTORE PATERNAL UBE3A GENE EXPRESSION IN HUMAN ANGELMAN SYNDROME
#11ENGINEERED MUSCLE TARGETING COMPOSITIONS
#12COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
#13KCNV2 Variants and Their Use
#14NUCLEIC ACID CONSTRUCTS, VIRAL VECTORS AND VIRAL PARTICLES
#15AAV CAPSID COMPOSITIONS AND METHODS FOR DELIVERY
#16HIGH ACTIVITY REGULATORY ELEMENTS
#17AAV Vectors Targeted to Oligodendrocytes
#18Methods of Treating Rheumatoid Arthritis Using RNA-Guided Genome Editing of HLA Gene
#19METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#20CAPSID VARIANTS AND METHODS OF USING THE SAME
#21COMPOSITION AND METHOD FOR TREATING HEMOPHILIA B
#22USE OF MULLERIAN INHIBITING SUBSTANCE (MIS) PROTEINS FOR CONTRACEPTION AND OVARIAN RESERVE PRESERVATION
#23GENE THERAPY OF HEMOPHILIA A USING VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION
#24METHODS OF REDOSING GENE THERAPY VECTORS
#25VECTOR-MEDIATED IMMUNE TOLERANCE IN THE EYE
#26ADENO-ASSOCIATED VIRUS VECTOR
#27Method of Increasing the Function of an AAV Vector
#28Compositions and methods of treating ocular diseases
#29METHODS FOR ANALYSIS OF VIRAL CAPSID PROTEIN COMPOSITION
#30ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY DELIVERY
#31METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#32ADENO-ASSOCIATED VIRUS VECTORS
#33METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#34Compositions and Methods of Treatment
#35METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#36Methods for using transcription-dependent directed evolution of AAV capsids
#37Methods for using transcription-dependent directed evolution of AAV capsids
#38Methods of predicting ancestral virus sequences and uses thereof
#39METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#40Adeno-associated variants, formulations and methods for pulmonary delivery
#41METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV2.5 VECTOR
#42Methods and compositions for antibody-evading virus vectors
#43METHODS FOR USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
#44NOVEL AAV3B VARIANTS THAT TARGET HUMAN HEPATOCYTES IN THE LIVER OF HUMANIZED MICE
#45CRISPR/Cas-related methods and compositions for treating Leber's congenital amaurosis 10 (LCA10)
#46METHOD OF IN VIVO ADMINISTRATION OF THE CODING SEQUENCE OF THE SIRT6 GENE VIA ADENO-ASSOCIATED VIRUS
#47Calcium Channel 3.2 Inhibitory Peptides and Uses Thereof
#48AAV CAPSID-PROMOTER INTERACTIONS AND CELL SELECTIVE GENE EXPRESSION
#49EFFECTIVE DOSAGES OF AN ADENOVIRAL-BASED BIOLOGICAL DELIVERY AND EXPRESSION SYSTEM FOR USE IN THE TREATMENT OF OSTEOARTHRITIS IN HUMANS, AND COMPOSITIONS COMPRISING THE SAME
#50NOVEL AAV VARIANT
#51VIRAL CAPSID POLYPEPTIDES
#52METHODS FOR TREATING PATIENTS HAVING CFH MUTATIONS WITH CFH-ENCODING VECTORS
#53FACTOR VIII POLYPEPTIDE
#54COMPOSITIONS AND METHODS OF INDUCING DIFFERENTIATION OF A HAIR CELL
#55Compositions and methods of treatment
#56ON-BIPOLAR CELL-SPECIFIC PROMOTERS FOR OCULAR GENE DELIVERY
#57KCNV2 variants and their use
#58VACCINE AND METHOD FOR TREATING CANCER
#59REGENERATION OF RETINAL GANGLION CELLS
#60COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAUOPATHY
#61Adeno-associated virus factor VIII vectors
#62ENGINEERED ADENO-ASSOCIATED VIRUS CAPSIDS
#63RECOMBINANT HERPESVIRALES VECTOR
#64COMPOSITIONS AND METHODS FOR MULTIPLEXED QUANTITATIVE ANALYSIS OF CELL LINEAGES
#65PROGRAMMABLE DNA BASE EDITING BY NME2CAS9-DEAMINASE FUSION PROTEINS
#66MODIFIED ADENO-ASSOCIATED VIRUS (AAV) PARTICLES FOR GENE THERAPY
#67CHEMICALLY-MODIFIED ADENO-ASSOCIATED VIRUS
#68SATIATION PEPTIDES FOR WEIGHT LOSS AND ALTERED TASTE SENSITIVITY
#69New Adeno-Associated Virus (AAV) Variants and Uses Thereof for Gene Therapy
#70Methods for analysis of viral capsid protein composition
#71ENHANCED TRANSDUCTION OF AAV VECTORS ENCODING MICRORNAS
#72ENGINEERED ADENO-ASSOCIATED VIRUS CAPSIDS
#73METHODS FOR THE TREATMENT OF EPILEPSY
#74NOVEL AAV LIBRARY
#75ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN SELECTED NEURONAL CELL POPULATIONS
#76Adeno-Associated Virus Virions with Variant Capsid and Methods of Use Thereof
#77METHOD FOR PRODUCING NUCLEIC ACID-ENCAPSULATED AAV HOLLOW PARTICLE
#78MINIGENE THERAPY
#79AAV MUTANT THAT EFFICIENTLY INFECTS SUPPORTING CELLS
#80ENGINEERED MUSCLE TARGETING COMPOSITIONS
#81CELLS FOR ENHANCED PRODUCTION OF ADENO-ASSOCIATED VIRUS
#82NOVEL AAV CAPSIDS AND COMPOSITIONS CONTAINING SAME
#83NOVEL AAV CAPSIDS AND COMPOSITIONS CONTAINING SAME
#84RAAV WITH CHEMICALLY MODIFIED CAPSID
#85Methods and compositions for dual glycan binding AAV2.5 vector
#86TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#87ADENO-ASSOCIATED VIRUS VECTOR
#88OLIGONUCLEOTIDES FOR HTT-1A MODULATION
#89Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#90Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#91METHODS OF PREDICTING ANCESTRAL VIRUS SEQUENCES AND USES THEREOF
#92Adeno-associated variants, formulations and methods for pulmonary delivery
#93MODIFIED FACTOR IX POLYPEPTIDES
#94IN VIVO HOMOLOGY DIRECTED REPAIR IN HEART, SKELETAL MUSCLE, AND MUSCLE STEM CELLS
#95Compositions and methods to restore paternal UBE3A gene expression in human Angelman syndrome
#96Factor IX encoding nucleotides
#97ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#98Vector-mediated immune tolerance in the eye
#99rAAV with chemically modified capsid
#100AAV VARIANTS WITH ENHANCED TROPISM
#101MULTIPLEXED SHRNAS AND USES THEREOF
#102CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND METHODS OF USE IN GENE THERAPY OF HUMAN LIVER CANCER
#103Adeno-associated virus vector
#104Compositions and methods for treatment of dominant retinitis pigmentosa
#105RAAV with chemically modified capsid
#106ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#107FORMULATION OPTIMIZATION FOR VIRAL PARTICLES
#108Methods and compositions for dual glycan binding AAV vectors
#109METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#110CAPSID-MODIFIED RAAV VECTORS AND METHODS OF USE
#111NOVEL DELIVERY OF LARGE PAYLOADS
#112Use of Mullerian inhibiting substance (MIS) proteins for contraception and ovarian reserve preservation
#113METHODS OF REDOSING GENE THERAPY VECTORS
#114Methods and materials for NT-3 gene therapy
#115Method of increasing the function of an AAV vector
#116Methods of predicting ancestral virus sequences and uses thereof
#117METHODS AND COMPOSITIONS FOR TREATING EQUINE CONDITIONS USING RECOMBINANT SELF-COMPLEMENTARY ADENO-ASSOCIATED VIRUS
#118Methods for analysis of viral capsid protein composition
#119Column-based fully scalable rAAV manufacturing process
#120ADENO-ASSOCIATED VIRAL VECTORS FOR TREATING MUCOLIPIDOSIS TYPE II
#121Compositions and methods for transient delivery of nucleases
#122High activity regulatory elements
#123METHOD OF INCREASING THE FUNCTION OF AN AAV VECTOR
#124Methods of treating rheumatoid arthritis using RNA-guided genome editing of HLA gene
#125ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#126Methods for analysis of viral capsid protein composition
#127Methods of predicting ancestral virus sequences and uses thereof
#128Adeno-associated virus factor VIII vectors
#129CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)
#130RAAV with chemically modified capsid
#131Methods and compositions for treating hemophilia
#132AAV vectors targeted to oligodendrocytes
#133AAV delivery of shRNA for treatment of pancreatic cancer
#134Adeno-associated virus virions with variant capsid
#135Method of increasing the function of an AAV vector
#136VIRAL NANOPARTICLES AND METHODS OF USE THEREOF
#137Therapeutic for treatment of diseases including the central nervous system
#138Methods of predicting ancestral virus sequences and uses thereof
#139Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#140Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#141Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#142Methods and compositions for dual glycan binding AAV vectors
#143Methods and compositions for genome engineering
#144TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#145REGULATABLE EXPRESSION USING ADENO-ASSOCIATED VIRUS (AAV)
#146METHODS OF TREATING PARKINSON'S DISEASE USING VIRAL VECTORS
#147Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer
#148CRISPR/CAS-related methods and compositions for treating Leber's Congenital Amaurosis 10 (LCA10)
#149Compositions and methods for transient delivery of nucleases
#150ADENO-ASSOCIATED VIRUS VECTOR
#151Treatment of ocular neovascularization using anti-VEGF proteins
#152Method of increasing the function of an AAV vector
#153Compositions and methods for transient delivery of nucleases
#154Capsid modified rAAV vectors and methods of use
#155Methods and compositions for treating hemophilia
#156Satiation peptides for weight loss and altered taste sensitivity
#157Methods of predicting ancestral virus sequences and uses thereof
#158AAV Vectors Targeted to Oligodendrocytes
#159Guided injections for AAV gene transfer to muscle
#160Multiplexed shRNAs and uses thereof
#161Drug delivery particle and method for producing the same
#162Targeting peptides for directing adeno-associated viruses (AAVs)
#163Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#164Adeno-associated virus factor VIII vectors
#165Methods of predicting ancestral virus sequences and uses thereof
#166Adeno-associated virus virions with variant capsid and methods of use thereof
#167Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#168Adeno-associated virus virions with variant capsid and methods of use thereof
#169Adeno-associated virus virions with variant capsid and methods of use thereof
#170Use of mullerian inhibiting substance (MIS) proteins for contraception and ovarian reserve preservation
#171Method of Treating or Retarding the Development of Blindness
#172METHODS OF TREATING PARKINSON'S DISEASE USING VIRAL VECTORS
#173Constructs and methods for delivering molecules via viral vectors with blunted innate immune responses
#174Methods of predicting ancestral virus sequences and uses thereof
#175Methods for expressing a polynucleotide of interest in the retina of a subject
#176Capsid-modified rAAV vectors and methods of use
#177Methods and compositions for dual glycan binding AAV vectors
#178INDUCTION OF EXON SKIPPING IN EUKARYOTIC CELLS
#179Adeno-associated virus (AAV) glades, sequences, vectors containing same, and uses therefor
#180AAV variant
#181CRISPR/CAS-related methods and compositions for treating leber's congenital amaurosis 10 (LCA10)
#182AAV vectors targeted to oligodendrocytes
#183Adeno-associated virus virions with variant capsid and methods of use thereof
#184Methods and compositions for treating hemophilia
#185Method of increasing the function of an AAV vector
#186Methods and compositions for genome engineering
#187MYOSTATIN INHIBITION FOR ENHANCING MUSCLE AND/OR IMPROVING MUSCLE FUNCTION
#188Adeno-associated virus factor VIII vectors
#189Treatment of ocular neovascularization using anti-VEGF proteins
#190Method of treating or retarding the development of blindness
#191Drug delivery particle and method for producing the same
#192METHOD OF TREATING OR RETARDING THE DEVELOPMENT OF BLINDNESS
#193Adeno-associated virus virions with variant capsid and methods of use thereof
#194ADENO-ASSOCIATED VIRUS (AAV) CLADES, SEQUENCES, VECTORS CONTAINING SAME, AND USES THEREFOR
#195Method of treating or retarding the development of blindness
#196Myostatin inhibition for enhancing muscle and/or improving muscle function
#197Tyrosine-modified recombinant rAAV vector compositions and methods for use
#198Methods of treating Parkinson's disease using viral vectors
#199Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof
#200Induction of exon skipping in eukaryotic cells
#201Method of increasing the function of an AAV vector
#202Method of Treating or Retarding the Development of Blindness
#203Induction of exon skipping in eukaryotic cells
#204Method of treating or retarding the development of blindness
#205Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses therefor
#206Methods of treating parkinson's disease using viral vectors
#207Composition for regulating production of interfering ribonucleic acid