110553 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses Demonstrated effect
ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#302COMPOSITIONS AND METHODS FOR TREATING WILSON'S DISEASE
#303PREVENTION OR TREATMENT OF CARDIAC ARRHYTMIA AND SUDDEN CARDIAC DEATH
#304ENGINEERED AAV CAPSIDS WITH INCREASED TROPISM AND AAV VECTORS COMPRISING THE ENGINEERED CAPSIDS AND METHODS OF MAKING AND USING SAME
#305ANTIBODY-EVADING VIRUS VECTORS
#306ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION AND THERAPEUTIC METHODS
#307COMPOSITIONS AND METHODS FOR TREATING GM1 GANGLIOSIDOSIS AND OTHER DISORDERS
#308AAV1 VECTORS AND USES THEREOF FOR TREATMENT OF OTIC INDICATIONS
#309Method and composition for treating neuropathic pain
#310METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#311Modified AAV constructs and uses thereof
#312ACTIVATION OF YAP SIGNALING FOR SENSORY RECEPTOR REGENERATION
#313AAV-based gene therapies for treatment of autoimmune diseases
#314Vector-mediated immune tolerance in the eye
#315Compositions and methods for impeding transcription of expanded microsatellite repeats
#316COMPOSITIONS AND METHODS FOR REDUCING SPLICEOPATHY AND TREATING RNA DOMINANCE DISORDERS
#317VIRAL VECTOR FOR TREATING AUTOIMMUNE DISEASE AND DIABETES AND CONSTRUCTION METHOD AND APPLICATION THEREOF
#318CORONAVIRUS IMMUNOGENIC COMPOSITIONS AND USES THEREOF
#319TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS AND DISORDERS ASSOCIATED WITH THE SPINAL CORD
#320CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND METHODS OF USE IN GENE THERAPY OF HUMAN LIVER CANCER
#321Compositions and methods for preventing and treating coronavirus infection—SARS-CoV-2 vaccines
#322METHOD FOR TREATING TRPV1-MEDIATED DISEASES
#323Cardiac cell reprogramming with myocardin and ASCL1
#324ADENO-ASSOCIATED VIRAL VECTOR-MEDIATED GENE THERAPY FOR TREATING FRAGILE X-ASSOCIATED DISORDERS
#325TREATMENT OF DISEASES INVOLVING DEFICIENCY OF ENPP1 OR ENPP3
#326PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#327IN SITU GENE EDITING
#328GENE THERAPY VECTORS COMPRISING S/MAR SEQUENCES
#329RECOMBINANT AAVS HAVING USEFUL TRANSCYTOSIS PROPERTIES
#330Methods of Treating or Preventing Amyotrophic Lateral Sclerosis
#331COMPOSITION AND METHOD FOR TREATING COMPLEMENT-MEDIATED DISEASE
#332Compositions and methods for delivery of AAV
#333COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING CATECHOLAMINERGIC POLYMORPHIC VENTRICULAR TACHYCARDIA
#334TREATMENT OF RETINITIS PIGMENTOSA
#335ADENO-ASSOCIATED VIRUS VECTOR MEDIATED GENE THERAPY FOR OPHTHALMIC DISEASES
#336COMPOSITIONS AND METHODS FOR TREATING SPINAL MUSCULAR ATROPHY
#337Methods and compositions for dual glycan binding AAV vectors
#338SHRNA EXPRESSION CASSETTE, POLYNUCLEOTIDE SEQUENCE CARRYING SAME, AND APPLICATION THEREOF
#339Hybrid recombinant adeno-associated virus serotype between AAV9 and AAVrh74 with reduced liver tropism
#340METHODS AND COMPOSITIONS FOR TREATMENT OF HEMOPHILIA
#341Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#342Campaign-ready series of recombinant adeno-associated virus (rAAV) complementing plasmids
#343CAPSID-MODIFIED RAAV VECTORS AND METHODS OF USE
#344Cardiac cell reprogramming with myocardin and ASCL1
#345AAV-VECTORS FOR USE IN GENE THERAPY OF CHOROIDEREMIA
#346Adeno-associated virus virion for treatment of Tay-Sachs disease and Sandhoff disease
#347Gene therapeutics for treating bone disorders
#348Efficient systemic treatment of dystrophic muscle pathologies
#349AAV-based treatment of cholesterol-related disorders
#350GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
#351Use of Mullerian inhibiting substance (MIS) proteins for contraception and ovarian reserve preservation
#352TREATMENT OF SPINAL MUSCULAR ATROPHY
#353MODIFIED RAAV CAPSID PROTEIN FOR GENE THERAPY
#354Methods of treating amyotrophic lateral sclerosis (ALS)
#355High efficiency library-identified AAV vectors
#356Methods of predicting ancestral virus sequences and uses thereof
#357Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
#358AAV vectors
#359Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
#360TREATMENT OF OCULAR DISEASES WITH FULLY-HUMAN POST-TRANSLATIONALLY MODIFIED ANTI-VEGF Fab
#361NUCLEIC ACID CONSTRUCTS AND METHODS OF USE
#362Cancer treatment
#363STEM CELLS FOR TRANSPLANTATION AND MANUFACTURING METHOD THEREFOR
#364COMPOSITIONS, KITS AND METHODS FOR TREATMENT OF COMPLEMENT-RELATED DISORDERS
#365Gene therapy for ocular disorders
#366PEPTIDES AND NANOPARTICLES FOR INTRACELLULAR DELIVERY OF VIRUS
#367METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#368Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#369Methods of predicting ancestral virus sequences and uses thereof
#370GENE THERAPY CONSTRUCTS AND METHODS FOR TREATMENT OF HEARING LOSS
#371Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#372Regulation of gene expression by aptamer-mediated modulation of alternative splicing
#373AAV treatment of Huntington's disease
#374Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#375Gene therapy for tuberous sclerosis
#376TREATMENT OF ALZHEIMERS DISEASE WITH MICRO RNA AND GHRELIN
#377NUCLEIC ACID MOLECULES AND USES THEREOF FOR NON-VIRAL GENE THERAPY
#378Inhibitors of CACNA1A/ALPHA1A subunit internal ribosomal entry site (IRES) and methods of treating spinocerebellar ataxia type 6
#379Non-invasive optogenetic stimulation method to regulate glucose metabolism in the liver and brown adipose tissue
#380Methods of transfection for large cargo using poly(beta-amino esters)
#381Treatment of retinitis pigmentosa
#382Methods for delivery of polynucleotides by adeno-associated virus for lysosomal storage disorders
#383Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
#384AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#385Fusion peptides with antigens linked to short fragments of invariant chain(CD74)
#386Methods and compositions relating to the treatment of tumors
#387Zinc finger protein compositions for modulation of huntingtin (Htt)
#388ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#389Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#390Neuronal enhancers
#391AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY IN GLAUCOMA
#392Adeno-associated virus compositions for PAH gene transfer and methods of use thereof
#393AAV-based treatment of cholesterol-related disorders
#394Engineered viral vector reduces induction of inflammatory and immune responses
#395Composition and method for treating complement-mediated disease
#396Efficient systemic treatment of dystrophic muscle pathologies
#397Liver-specific constructs factor VIII expression cassettes and methods of use thereof
#398Liver-specific constructs and methods of use thereof
#399CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR
#400Recombinant AAVS having useful transcytosis properties
#401Therapeutic for treatment of diseases including the central nervous system
#402Methods of predicting ancestral virus sequences and uses thereof
#403SYSTEMIC SYNTHESIS AND REGULATION OF L-DOPA
#404Gene therapy for juvenile batten disease
#405Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#406Capsid-modified rAAV vector compositions and methods therefor
#407Hybrid dual recombinant AAV vector systems for gene therapy
#408COMPOSITIONS AND METHODS FOR REGULATABLE ANTIBODY EXPRESSION
#409Methods and compositions for dual glycan binding AAV vectors
#410TREATING CARDIOVASCULAR OR RENAL DISEASES
#411PHARMACEUTICAL COMPOSITION FOR TREATMENT OF CARDIAC FIBROSIS
#412METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#413Methods and compositions for treating metabolic imbalance in neurodegenerative disease
#414TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#415Modified AAV constructs and uses thereof
#416INTRANASAL THERAPEUTIC DELIVERY OF ADENO-ASSOCIATED VIRUS TO CENTRAL NERVOUS SYSTEM
#417Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#418TREATMENT OF RETINITIS PIGMENTOSA
#419METHODS TO TREAT CEROID LIPOFUSCINOSIS USING A RECOMBINANT ADENO-ASSOCIATED VIRUS
#420METHODS TO TREAT POMPE DISEASE USING A RECOMBINANT ADENO-ASSOCIATED VIRUS
#421ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#422High efficiency library-identified AAV vectors
#423ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#424CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS HAVING IMPROVED TRANSDUCTION EFFICIENCIES, AND METHODS OF USE
#425Treating cardiovascular or renal diseases
#426METHODS FOR TREATING NEURODEGENERATIVE DISEASES USING GENE THERAPY TO DELAY DISEASE ONSET AND PROGRESSION WHILE PROVIDING COGNITIVE PROTECTION
#427GLP-1 and use thereof in compositions for treating metabolic diseases
#428Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer
#429Adeno-associated viruses engineered for selectable tropism
#430INHIBITORS OF CACNA1A/ALPHA1A SUBUNIT INTERNAL RIBOSOMAL ENTRY SITE (IRES) AND METHODS OF TREATING SPINOCEREBELLAR ATAXIA TYPE 6
#431Products and Methods for Delivery of Polynucleotides by Adeno-Associated Virus for Lysosomal Storage Disorders
#432Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the peripheral nervous system of a subject
#433Synergistic combination of neuronal viability factors and uses thereof
#434Glucocerebrosidase gene therapy for Parkinson's disease
#435AAV-VECTORS FOR USE IN GENE THERAPY OF CHOROIDEREMIA
#436Modified AAV capsids and uses thereof
#437Treatment of ocular neovascularization using anti-VEGF proteins
#438Spinal subpial gene delivery system
#439SMAD7 gene delivery into muscle cells
#440Methods and compositions for restoration of cone function in BCM
#441Fabry disease gene therapy
#442AAV vectors with high transduction efficiency and uses thereof for gene therapy
#443Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
#444AAV treatment of Huntington's disease
#445Viral vectors for the treatment of retinal dystrophy
#446ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#447Capsid modified rAAV vectors and methods of use
#448ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#449Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#450Regulation of gene expression by aptamer-mediated modulation of alternative splicing
#451Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
#452Nucleic acid constructs and gene therapy vectors for use in the treatment of Wilson disease
#453Recombinant adeno-associated virus capsids resistant to pre-existing human neutralizing antibodies
#454AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#455METHOD FOR ENHANCED UPTAKE OF VIRAL VECTORS IN THE MYOCARDIUM
#456Adeno-associated viral vectors for treating myocilin (MYOC) glaucoma
#457Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#458Methods of predicting ancestral virus sequences and uses thereof
#459Adeno-associated viral vectors for the gene therapy of metabolic diseases
#460High-transduction-efficiency rAAV vectors, compositions, and methods of use
#461Guided injections for AAV gene transfer to muscle
#462Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#463Treating cardiovascular or renal diseases
#464Targeting peptides for directing adeno-associated viruses (AAVs)
#465Efficient systemic treatment of dystrophic muscle pathologies
#466Liver-specific constructs, factor VIII expression cassettes and methods of use thereof
#467Gene therapy for neurodegenerative disorders
#468Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#469Methods of predicting ancestral virus sequences and uses thereof
#470Replacement gene tolerizing vectors and methods of use thereof
#471METHODS AND COMPOSITIONS FOR GENE DELIVERY TO ON BIPOLAR CELLS
#472GENE THERAPY COMPOSITIONS FOR USE IN THE PREVENTION AND/OR TREATMENT OF NON-ALCOHOLIC FATTY LIVER DISEASE
#473USE OF CRISPR/CAS9 AS IN VIVO GENE THERAPY TO GENERATE TARGETED GENOMIC DISRUPTIONS IN GENES BEARING DOMINANT MUTATIONS FOR RETINITIS PIGMENTOSA
#474Methods and pharmaceutical compositions for expressing a polynucleotide of interest in the retinal pigment epithelium of a subject
#475Use of mullerian inhibiting substance (MIS) proteins for contraception and ovarian reserve preservation
#476Compositions and methods for treatment of muscular dystrophy
#477Artificial DNA-binding proteins and uses thereof
#478COMPOSITIONS AND METHODS FOR TREATING CARDIOVASCULAR DISEASES USING FOXP3
#479Methods for treating spinocerebellar ataxia type I using RPA1
#480S100 based treatment of cardiac power failure
#481Method to kill pathogenic microbes in a patient
#482Methods of predicting ancestral virus sequences and uses thereof
#483AAV-based treatment of cholesterol-related disorders
#484Recombinant AAV-crumbs homologue composition and methods for treating LCA-8 and progressive RP
#485PRODUCTS AND METHODS FOR DELIVERY OF POLYNUCLEOTIDES BY ADENO-ASSOCIATED VIRUS FOR LYSOSOMAL STORAGE DISORDERS
#486Peptides having specificity for the lungs
#487Methods for expressing a polynucleotide of interest in the retina of a subject
#488Materials and methods for the treatment of pathological neovascularization in the eye
#489Methods to treat mucopolysaccharide type I or deficiency in alpha-L-iduronidase using a recombinant adeno-associated virus encoding alpha-L-iduronidase
#490Viral vectors for the treatment of retinal dystrophy
#491Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#492STEM CELLS FOR TRANSPLANTATION AND MANUFACTURING METHOD THEREFOR
#493Recombinant adeno-associated virus delivery of exon 2-targeted U7SNRNA polynucleotide constructs
#494Compositions and methods for treating spinal muscular atrophy
#495Gene therapy for neurodegenerative disorders
#496TGIF2-induced reprogramming of hepatic cells to pancreatic progenitor cells and medical uses thereof
#497Methods and compositions for dual glycan binding AAV vectors
#498METHOD AND COMPOSITION FOR TREATING SPASTICITY
#499Methods and compositions for treating Huntington's Disease
#500Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to Friedreich ataxia
#501WIDESPREAD GENE DELIVERY OF GENE THERAPY VECTORS
#502High-transduction-efficiency RAAV vectors, compositions, and methods of use
#503Gene therapy for Alzheimer's and other neurodegenerative diseases and conditions
#504Treating cardiovascular or renal diseases
#505Treatment of ocular neovascularization using anti-VEGF proteins
#506Method of treating or retarding the development of blindness
#507Capsid-modified rAAV vector compositions and methods therefor
#508Methods for the treatment and prevention of liver disease
#509Recombinant adeno-associated virus-mediated expression of fractalkine for treatment of neuroinflammatory and neurodegenerative diseases
#510Capsid-free AAV vectors, compositions, and methods for vector production and gene delivery
#511AAV -vectors for use in gene therapy of choroideremia
#512Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use
#513Compositions, kits and methods for treatment of complement-related disorders
#514Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#515PRODUCTS AND METHODS FOR DELIVERY OF POLYNUCLEOTIDES BY ADENO-ASSOCIATED VIRUS FOR LYSOSOMAL STORAGE DISORDERS
#516METHOD OF TREATING OR RETARDING THE DEVELOPMENT OF BLINDNESS
#517METHODS FOR TREATING PARKINSON'S DISEASE AND OTHER DISORDERS OF DOPAMINERGIC NEURONS OF THE BRAIN
#518Materials and methods for the treatment of pathological neovascularization in the eye
#519ALANINE-GLYOXYLATE AMINOTRANSFERASE THERAPEUTICS
#520Adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#521Method for detecting adeno-associated virus
#522Methods and sequences to suppress primate huntington gene expression in vivo
#523Method of treating or retarding the development of blindness
#524INERT DNA SEQUENCES FOR EFFICIENT VIRAL PACKAGING AND METHODS OF USE
#525COMPOSITIONS FOR REPROGRAMMING A CELL AND USES THEREFOR
#526Tyrosine-modified recombinant rAAV vector compositions and methods for use
#527Methods for therapy of neurodegenerative disease of the brain
#528TREATING HEART FAILURE AND VENTRICULAR ARRHYTHMIAS
#529Method for enhanced uptake of viral vectors in the myocardium
#530Method of Treating or Retarding the Development of Blindness
#531Isolated nucleic acid duplex for reducing huntington gene expression
#532Methods for therapy of neurodegenerative disease of the brain
#533Truncated CMV promoters and vectors containing same
#534Method of treating or retarding the development of blindness
#535Use of fak-related non-kinase in the manufacture of a medicament for the inhibition of stenosis and restenosis
#536Method for therapy of neurodegenerative disease of the brain