110540 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses Use of viral protein as therapeutic agent other than vaccine, e.g. apoptosis inducing or anti-inflammatory
METHODS FOR TREATING CANCER WITH HYPERACTIVE ADAR ENZYMES
#2VIRAL PARTICLES RETARGETED TO SKELETAL MUSCLE
#3Gene Therapy For Neurodegenerative Disorders
#4ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
#5GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
#6Gene Therapy For Neurodegenerative Disorders
#7MODIFIED AAV CAPSIDS AND USES THEREOF
#8GENE THERAPIES FOR LYSOSOMAL DISORDERS
#9TREATMENT OF COMPLEMENT-MEDIATED DISORDERS
#10METHODS OF TREATING OCULAR DISEASES USING AAV2 VARIANTS ENCODING AFLIBERCEPT
#11Genome editing approaches to treat Spinal Muscular Atrophy
#12Gene Therapy For Neurodegenerative Disorders
#13Adeno-associated virus variant capsids and methods of use thereof
#14METHODS FOR PREVENTING INDUCTION OF IMMUNE RESPONSES TO THE TRANSDUCED CELLS EXPRESSING A TRANSGENE PRODUCT AFTER OCULAR GENE THERAPY
#15PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#16CIRCUIT BOARD
#17RECOMBINANT AAV VECTORS WITH ALTERED IMMUNOGENCITY AND METHODS OF MAKING THE SAME
#18AAV NATIVE-NEURO PLATFORM AND USE FOR NEURONAL DISEASE GENE THERAPY
#19Gene therapy for neurodegenerative disorders
#20Gene therapy for neurodegenerative disorders
#21REGENERATION OF RETINAL GANGLION CELLS
#22Adeno-associated virus variant capsids and methods of use thereof
#23Modified AAV capsids and uses thereof
#24RAAV WITH CHEMICALLY MODIFIED CAPSID
#25COMPOSITIONS AND METHODS FOR THE TREATMENT OF DEGENERATIVE OCULAR DISEASES
#26MODIFIED ADENO-ASSOCIATED VIRAL VECTORS FOR USE IN GENETIC ENGINEERING
#27MODULATION OF SPTLC1 VIA RECOMBINANT ADENO-ASSOCIATED VECTORS
#28Prokaryotic-eukaryotic hybrid viral vector for delivery of large cargos of genes and proteins into human cells
#29Adeno-associated virus variant capsids and methods of use thereof
#30MODIFIED AAV CAPSID POLYPEPTIDES FOR TREATMENT OF MUSCULAR DISEASES
#31ADENO-ASSOCIATED VIRUS HAVING A VARIANT CAPSID PROTEIN, AND USE THEREOF
#32Adeno-associated virus purification methods
#33ADENO-ASSOCIATED VIRAL CAPSIDS WITH EXPANDED SIZES
#34rAAV with chemically modified capsid
#35RAAV with chemically modified capsid
#36PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#37Prokaryotic-eukaryotic hybrid viral vector for delivery of large cargos of genes and proteins into human cells
#38VIRAL VECTOR TRANSDUCTION
#39NOVEL DELIVERY OF LARGE PAYLOADS
#40GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
#41Gene therapies for lysosomal disorders
#42Gene therapy for neurodegenerative disorders
#43Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#44Gene therapies for lysosomal disorders
#45TREATMENT OF COMPLEMENT-MEDIATED DISORDERS
#46Adeno-associated virus capsid variants and methods of use thereof
#47RAAV with chemically modified capsid
#48Compositions and methods for the expression of CRISPR guide RNAS using the H1 promoter
#49Adeno-associated virus purification methods
#50Methods and compositions for treating hemophilia
#51CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#52CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#53VIRAL NANOPARTICLES AND METHODS OF USE THEREOF
#54CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#55CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#56Viral vector for the targeted transfer of genes in the brain and spinal cord
#57Adeno-associated virus polynucleotides, polypeptides and virions
#58Vector
#59Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#60Methods and compositions for genome engineering
#61METHODS OF TREATING NEURODEGENERATIVE DISORDERS
#62Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#63CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#64Modified AAV capsids and uses thereof
#65CNS targeting AAV vectors and methods of use thereof
#66METHODS AND COMPOSITIONS FOR TREATING BRAIN DISEASES
#67Methods and compositions for treating hemophilia
#68Methods of treating neurodegenerative disorders
#69Gene therapy for neurodegenerative disorders
#70Viral vector for the targeted transfer of genes in the brain and spinal cord
#71Peptides having specificity for the lungs
#72MESSENGER RNA BASED VIRAL PRODUCTION
#73Compositions and methods for the expression of CRISPR guide RNAs using the H1 promoter
#74VECTOR ENCODING THERAPEUTIC POLYPEPTIDE AND SAFETY ELEMENTS TO CLEAR TRANSDUCED CELLS
#75Gene therapy for neurodegenerative disorders
#76COMPOSITIONS AND METHODS FOR TREATING CARDIOVASCULAR DISEASES USING SMAD3
#77Methods and compositions for treating hemophilia
#78Methods and compositions for genome engineering
#79CNS targeting AAV vectors and methods of use thereof
#80Viral vectors with modified transduction profiles and methods of making and using the same
#81CNS targeting AAV vectors and methods of use thereof
#82Parvovirus methods and compositions for killing neoplastic cells
#83VECTOR ENCODING THERAPEUTIC POLYPEPTIDE AND SAFETY ELEMENTS TO CLEAR TRANSDUCED CELLS
#84Methods and compositions for treating brain diseases