110542 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses Use of virus, viral particle or viral elements as a vector
Sub-classes:AAV CAPSID VARIANTS AND USES THEREOF
#2ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#3RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY
#4REGENERATING FUNCTIONAL NEURONS FOR TREATMENT OF SPINAL CORD INJURY AND ALS
#5ADENO-ASSOCIATED VIRUS (AAV) DELIVERY OF ANTI-FAM19A5 ANTIBODIES
#6GENE THERAPY
#7RELATIVE POTENCY ASSAY FOR VIRAL VECTOR ENCODING ISOMEROHYDROLASES
#8RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY
#9OPTIMIZATION OF HEK293 SUSPENSION PLATFORM FOR IMPROVED rAAV TITERS
#10Compositions and Methods for Immunotherapy
#11AAV CAPSID VARIANTS AND USES THEREOF
#12Targeted CRISPR Delivery Platforms
#13Targeted CRISPR Delivery Platforms
#14COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#15COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#16COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#17COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#18COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#19COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#20COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#21COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#22COMPOSITION FOR REGULATING PRODUCTION OF INTERFERING RIBONUCLEIC ACID
#23ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#24ENGINEERED MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE HEPATITIS B VIRUS GENOME
#25METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#26PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#27NEUROPEPTIDE-EXPRESSING VECTORS AND METHODS FOR THE TREATMENT OF EPILEPSY
#28AAV capsid variants and uses thereof
#29METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#30ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR TREATMENT OF PROGRANULIN ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS
#31COMPOSITIONS AND METHODS FOR THE TARGETING OF C9orf72
#32SELF-POLARIZING IMMUNE CELLS
#33ENGINEERED MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE HEPATITIS B VIRUS GENOME
#34DELIVERY OF CARD PROTEIN AS THERAPY FOR OCCULAR INFLAMMATION
#35CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES
#36MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF NERVOUS SYSTEM DYSFUNCTION
#37CONSTRUCTS AND METHODS FOR DELIVERYING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES
#38MICRORNA COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF NERVOUS SYSTEM DYSFUNCTION
#39CAS-READY MOUSE EMBRYONIC STEM CELLS AND MICE AND USES THEREOF
#40AAV TREATMENT OF HUNTINGTON’S DISEASE
#41ADENO-ASSOCIATED VARIANTS, FORMULATIONS AND METHODS FOR PULMONARY DELIVERY
#42ADENO-ASSOCIATED VIRUS CAPSIDS AND VECTORS
#43METHOD AND COMPOSITION FOR TREATING NON-ALCOHOLIC FATTY LIVER DISEASE, ASSOCIATED CONDITIONS AND SYMPTOMS
#44Adeno-Associated Virus Factor VIII Vectors, Associated Viral Particles and Therapeutic Formulations Comprising the Same
#45ASSAY FOR MEASURING POTENCY OF GENE THERAPY DRUG PRODUCT
#46Compositions and Methods for Treating Spinal Muscular Atrophy
#47Treatment of muscular dystrophies
#48ABCD1 FOR TREATMENT OF NEURODISORDERS
#49IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF MONOGENIC DISEASES, INCLUDING MUCOPOLYSACCHARIDOSIS TYPE 1H AND OTHER DISORDERS
#50METHODS AND COMPOSITIONS FOR THE PRODUCTION OF ADENO-ASSOCIATED VIRUS
#51PLASMID SYSTEM
#52Methods of predicting ancestral virus sequences and uses thereof
#53METHODS AND COMPOSITIONS FOR THE TREATMENT OF ALS
#54NOVEL CRISPR ENZYMES, METHODS, SYSTEMS AND USES THEREOF
#55THERAPEUTICS FOR THE TREATMENT OF FSHD
#56GENE CONSTRUCTS FOR SILENCING ANGIOPOIETIN-LIKE 3 (ANGPTL3) AND USES THEREOF
#57Treatment of Genetic Dilated Cardiomyopathies
#58Adeno-associated variants, formulations and methods for pulmonary delivery
#59SILENCING OF DUX4 BY RECOMBINANT GENE EDITING COMPLEXES
#60ALL-IN-ONE AAV VECTORS FOR TREATING CORONAVIRUS-INDUCED DISEASES
#61ULTRA-SENSITIVE STEP-FUNCTION OPSIN FOR MINIMALLY INVASIVE OPTOGENETIC STIMULATION
#62METHOD OF TREATMENT OF CONGENITAL MYASTHENIC SYNDROME USING DOK7 GENE OR POLYPEPTIDE
#63ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN INHIBITORY NEOCORTICAL NEURONS
#64METHODS AND COMPOSITIONS FOR NUCLEASE-MEDIATED TARGETED INTEGRATION OF TRANSGENES INTO MAMMALIAN LIVER CELLS
#65Butyrylcholinesterases having an enhanced ability to hydrolyze acyl ghrelin
#66ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#67GENOME INTEGRITY ANALYSIS OF VIRUS VECTORS
#68Delivery of card protein as therapy for ocular inflammation
#69COMPOSITIONS AND METHODS FOR IMPROVING VIRAL VECTOR EFFICIENCY
#70AAV-Mediated Targeting of MIRNA in the Treatment of X-Linked Disorders
#71METHOD FOR TREATING USHER SYNDROME AND COMPOSITION THEREOF
#72Adeno-Associated Virus Vector Delivery of Microrna-29 to Treat Muscular Dystrophy
#73OBSESSIVE-COMPULSIVE DISORDER ANIMAL MODEL AND PRODUCTION METHOD THEREOF
#74METHODS OF TREATING DEMENTIA ASSOCIATED WITH ALZHEIMER'S DISEASE WITH PROTECTIVE PROTEIN/CATHEPSIN A (PPCA)
#75Targeted CRISPR Delivery Platforms
#76METABOLIC THERAPY FOR OXIDATIVE STRESS IN THE BRAIN THROUGH TARGETED NEURONAL CATABOLISM OF N-ACETYL-ASPARTIC ACID
#77GENERATION OF NEURONS BY REPROGRAMMING OF OLIGODENDROCYTES AND OLIGODENDROCYTE PRECURSOR CELLS
#78COMPOSITIONS AND METHODS FOR TREATING LEBER'S HEREDITARY OPTIC NEUROPATHY
#79ENGINEERED MEGANUCLEASES SPECIFIC FOR RECOGNITION SEQUENCES IN THE HEPATITIS B VIRUS GENOME
#80FULLY-HUMAN POST-TRANSLATIONALLY MODIFIED ANTIBODY THERAPEUTICS
#81REGULATABLE EXPRESSION USING ADENO-ASSOCIATED VIRUS (AAV)
#82Compositions and methods for regulating production of a precursor protein
#83COMPOSITIONS AND METHODS FOR REGULATING PRODUCTION OF A PRECURSOR PROTEIN
#84Multiplex shRNA for Use in Vectors
#85CONSTRUCTS AND METHODS FOR DELIVERING MOLECULES VIA VIRAL VECTORS WITH BLUNTED INNATE IMMUNE RESPONSES
#86TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#87AAV-IDUA vector for treatment of MPS I-associated blindness
#88METHODS OF PREDICTING ANCESTRAL VIRUS SEQUENCES AND USES THEREOF
#89AAV treatment of Huntington's disease
#90Cas-ready mouse embryonic stem cells and mice and uses thereof
#91Adeno-associated virus (AAV) delivery of anti-FAM19A5 antibodies
#92DELIVERY OF CARD PROTEIN AS THERAPY FOR OCCULAR INFLAMMATION
#93AAV CAPSID PROTEINS FOR NUCLEIC ACID TRANSFER
#94RELATIVE POTENCY ASSAY FOR VIRAL VECTOR ENCODING ISOMEROHYDROLASES
#95Compositions and methods for treating Leber's hereditary optic neuropathy
#96CRISPR INTERFERENCE BASED HTT ALLELIC SUPPRESSION AND TREATMENT OF HUNTINGTON DISEASE
#97RECOMBINANT AAV VECTORS AND METHODS OF USING THE SAME
#98COMPOSITIONS AND METHODS FOR TREATING AGE-RELATED MACULAR DEGENERATION
#99Gene sequence of recombinant human type II mitochondrial dynein-like GTPase and uses thereof
#100Engineered meganucleases specific for recognition sequences in the Hepatitis B virus genome
#101Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
#102Compositions for reducing sarcolipin expression and preventing and treating muscular dystrophy and cardiomyopathy and methods of use
#103CRISPR therapy
#104REGENERATING FUNCTIONAL NEURONS FOR TREATMENT OF SPINAL CORD INJURY AND ALS
#105REGENERATING FUNCTIONAL NEURONS FOR TREATMENT OF NEUROLOGICAL DISORDERS
#106PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#107ADENO-ASSOCIATED VIRUS (AAV) SYSTEMS FOR TREATMENT OF PROGRANULIN ASSOCIATED NEURODEGENERATIVE DISEASES OR DISORDERS
#108Recombinant adeno-associated viral vector for gene delivery
#109METHODS OF GENE THERAPY
#110Treatment of muscular dystrophies
#111COMPOSITIONS AND METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
#112Methods and compositions for dual glycan binding AAV vectors
#113NEUROPEPTIDE-EXPRESSING VECTORS AND METHODS FOR THE TREATMENT OF EPILEPSY
#114Adeno-associated virus (AAV) vector having hybrid HGF gene introduced thereto
#115Bicistronic AAV vector for RNA interference in ALS
#116RECOMBINANT ADIPOSE-DERIVED STEM CELL EXPRESSING BDDHFVIII GENE, AND PREPARATION METHOD AND APPLICATION THEREOF
#117Methods of predicting ancestral virus sequences and uses thereof
#118COMPOSITIONS AND METHODS FOR EDITING RNA
#119Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#120Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
#121Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
#122Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome
#123METHODS AND COMPOSITIONS COMPRISING CRISPR-CPF1 AND PAIRED GUIDE CRISPR RNAS FOR PROGRAMMABLE GENOMIC DELETIONS
#124Metabolic therapy for oxidative stress in the brain through targeted neuronal catabolism of N-acetyl-aspartic acid
#125PEPTIDE-MEDIATED DELIVERY OF RNA-GUIDED ENDONUCLEASE INTO CELLS
#126Compositions and methods for the treatment of myotonic dystrophy
#127CRISPR/Cas 9-mediated integration of polynucleotides by sequential homologous recombination of AAV donor vectors
#128Methods of predicting ancestral virus sequences and uses thereof
#129COMPOSITIONS AND METHODS FOR CONTROLLING PAIN
#130Oxidation-resistant AAT gene therapy
#131AAV treatment of Huntington's disease
#132SELF-LIMITING CAS9 CIRCUITRY FOR ENHANCED SAFETY (SLICES) PLASMID AND LENTIVIRAL SYSTEM THEREOF
#133Adeno-associated virus Factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
#134METHODS FOR ENHANCING YIELD OF RECOMBINANT ADENO-ASSOCIATED VIRUS
#135Stem loop RNA mediated transport of mitochondria genome editing molecules (endonucleases) into the mitochondria
#136Genetic modification of the AAV capsid resulting in altered tropism and enhanced vector delivery
#137Recombinant AAV variants and uses thereof
#138Liver targeting adeno-associated viral vectors
#139Silencing of DUX4 by recombinant gene editing complexes
#140Method of making and using mammalian liver cells for treating hemophilia or lysosomal storage disorder
#141Targeted CRISPR Delivery Platforms
#142Modified soluble VEGF receptor-1 genes and vectors for gene therapy
#143Reducing abnormal accumulation of TDP-43 in motor neurons in amyotrophic lateral sclerosis and/or frontotemporal dementia using a construct encoding cyclin F
#144Pharmaceutical composition comprising AIMP2-DX2 for preventing or treating neuronal diseases and use thereof
#145SCALABLE METHODS FOR PRODUCING RECOMBINANT ADENO-ASSOCIATED VIRAL (AAV) VECTOR IN SERUM-FREE SUSPENSION CELL CULTURE SYSTEM SUITABLE FOR CLINICAL USE
#146Engineered meganucleases specific for recognition sequences in the hepatitis B virus genome
#147Butyrylcholinesterases having an enhanced ability to hydrolyze acyl ghrelin
#148CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#149CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#150Guide RNA complementary to KRAS gene, and use thereof
#151Intrathecal delivery of nucleic acid sequences encoding ABCD1 for treatment of adrenomyeloneuropathy
#152GENERATION OF NEURONS BY REPROGRAMMING OF OLIGODENDROCYTES AND OLIGODENDROCYTE PRECURSOR CELLS
#153LIGHT-CONTROLLED GENE DELIVERY WITH VIRUS VECTORS THROUGH INCORPORATION OF OPTOGENETIC PROTEINS AND GENETIC INSERTION OF NON-CONFORMATIONALLY CONSTRAINED PEPTIDES
#154CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#155CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#156Gene therapy
#157ADENO-ASSOCIATED VIRUS MEDIATED DELIVERY OF C1EI AS A THERAPY FOR ANGIOEDEMA
#158Adeno-associated virus vector delivery of microrna-29 to treat muscular dystrophy
#159Method for promoting muscle regeneration
#160Peptide-mediated delivery of RNA-guided endonuclease into cells
#161METHODS AND COMPOSITIONS FOR ATTENUATING GENE EXPRESSION MODULATING ANTI-VIRAL TRANSFER VECTOR IMMUNE RESPONSES
#162Adeno-associated virus vector delivery of microRNA-29 and micro-dystrophin to treat muscular dystrophy
#163Adeno-associated virus vector delivery of micro-dystrophin to treat muscular dystrophy
#164Methods of predicting ancestral virus sequences and uses thereof
#165AAV-IDUA vector for treatment of MPS I-associated blindness
#166NOVEL FAMILY OF RNA-PROGRAMMABLE ENDONUCLEASES AND THEIR USES IN GENOME EDITING AND OTHER APPLICATIONS
#167Adenovirus-associated viral vectors for expressing variants of tetratricopeptide repeat (TPR)-containing Rab8b interacting (TRIP8b) protein in neurons and uses thereof for treating major depressive disorder (MDD)
#168Cas-ready mouse embryonic stem cells and mice and uses thereof
#169Methods and compositions for dual glycan binding AAV vectors
#170INTRATHECAL DELIVERY OF NUCLEIC ACID SEQUENCES ENCODING ABCD1 FOR TREATMENT OF ADRENOMYELONEUROPATHY
#171Compositions and methods for improving viral vector efficiency
#172TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#173Baculovirus system for the expression of a gene therapy vector
#174AAV capsid proteins for nucleic acid transfer
#175REGULATABLE EXPRESSION USING ADENO-ASSOCIATED VIRUS (AAV)
#176Bicistronic AAV vector for RNA interference in ALS
#177NR2E1 MINI-PROMOTERS
#178PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#179CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#180Metabolic therapy for oxidative stress in the brain through targeted neuronal catabolism of N-acetyl-aspartic acid
#181Treatment of ocular neovascularization using anti-VEGF proteins
#182AAV treatment of Huntington's disease
#183Optimized liver-specific expression systems for FVIII and FIX
#184Minimally-invasive and activity-dependent control of excitable cells
#185Recombinant AAV variants and uses thereof
#186CNS targeting AAV vectors and methods of use thereof
#187Transgenic RPE cells overexpressing OTX2 for the treatment of retinal degeneration
#188Peptide-mediated delivery of RNA-guided endonuclease into cells
#189MODIFIED ADENO-ASSOCIATED VIRUS VECTOR COMPOSITIONS
#190Methods of predicting ancestral virus sequences and uses thereof
#191Targeting peptides for directing adeno-associated viruses (AAVs)
#192Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
#193Butyrylcholinesterases having an enhanced ability to hydrolyze acyl ghrelin
#194Methods of predicting ancestral virus sequences and uses thereof
#195Pharmaceutical composition and method for reducing scar formation
#196Modified factor IX, and compositions, methods and uses for gene transfer to cells, organs, and tissues
#197Adeno-associated virus mediated delivery of C1E1 as a therapy for angioedema
#198Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
#199Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
#200Compositions and methods for inhibiting NF-κB and SOD-1 to treat amyotrophic lateral sclerosis
#201Constructs and methods for delivering molecules via viral vectors with blunted innate immune responses
#202Methods of predicting ancestral virus sequences and uses thereof
#203Compositions and methods for controlling pain
#204MESSENGER RNA BASED VIRAL PRODUCTION
#205Proviral plasmids and production of recombinant adeno-associated virus
#206Compositions and methods for treating spinal muscular atrophy
#207Methods and compositions for attenuating exon skipping anti-viral transfer vector immune responses
#208Methods and compositions for attenuating anti-viral transfer vector immune responses
#209Methods for treating neurogenic disorders of the pelvic floor
#210Vectors comprising stuffer/filler polynucleotide sequences and methods of use
#211Delivery of card protein as therapy for occular inflammation
#212Methods and compositions for dual glycan binding AAV vectors
#213Modified soluble VEGF receptor-1 genes and vectors for gene therapy
#214AAV capsid proteins for nucleic acid transfer
#215Method of treating factor IX deficiency using nuclease-mediated targeted integration
#216Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
#217Modified adeno-associated virus vector compositions
#218Adeno associated virus plasmids and vectors
#219Delivery of proteins using adeno-associated virus (AAV) vectors
#220Treatment of ocular neovascularization using anti-VEGF proteins
#221METHODS AND COMPOSITIONS TO DETECT THE LEVEL OF LYSOSOMAL EXOCYTOSIS ACTIVITY AND METHODS OF USE
#222Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
#223TARGETED GENE MODIFICATION USING HYBRID RECOMBINANT ADENO-ASSOCIATED VIRUS
#224Baculovirus system for the expression of a gene therapy vector
#225Capsid-modified rAAV vector compositions and methods therefor
#226CNS targeting AAV vectors and methods of use thereof
#227Synthetic adeno-associated virus inverted terminal repeats
#228Methods for the treatment and prevention of liver disease
#229Proviral plasmids and production of recombinant adeno-associated virus
#230Metabolic therapy for oxidative stress in the brain through targeted neuronal catabolism of N-acetyl-aspartic acid
#231CNS targeting AAV vectors and methods of use thereof
#232AAV capsid proteins for nucleic acid transfer
#233Delivery of proteins using adeno-associated virus (AAV) vectors
#234AAV4 vector and uses thereof
#235Composition for regulating production of interfering ribonucleic acid
#236Composition for regulating production of interfering ribonucleic acid