110553 ⎘
ssDNA viruses; Details; Parvoviridae; Dependovirus, e.g. adenoassociated viruses Demonstrated effect
GENE THERAPY FOR JUVENILE BATTEN DISEASE
#2LIVER-SPECIFIC VIRAL PROMOTERS AND METHODS OF USING THE SAME
#3ADENO-ASSOCIATED VIRUS (AAV) DIRECTED ANTIOXIDATIVE GENE THERAPY FOR THE PREVENTION, AMELIORATION, AND/OR TREATMENT OF HEARING LOSS
#4COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#5Methods of Treating or Preventing Amyotrophic Lateral Sclerosis
#6ADENO-ASSOCIATED VIRUS DELIVERY OF CLN3 POLYNUCLEOTIDE
#7RECOMBINANT ADENO-ASSOCIATED VIRUS FOR TREATMENT OF GRN-ASSOCIATED ADULT-ONSET NEURODEGENERATION
#8ABCA4 TRANS-SPLICING MOLECULES
#9METHODS AND COMPOSITIONS FOR TREATING METABOLIC IMBALANCE IN NEURODEGENERATIVE DISEASE
#10REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED MODULATION OF ALTERNATIVE SPLICING
#11GENERATION OF NEURONS BY REPROGRAMMING OF OLIGODENDROCYTES AND OLIGODENDROCYTE PRECURSOR CELLS
#12CHIMERIC AAV AND USES THEREOF
#13METHODS OF MANUFACTURING MICRODYSTROPHIN GENE THERAPY CONSTRUCTS
#14TREATMENT OF DYSTROPHINOPATHIES WITH MICRODYSTROPHIN GENE THERAPY CONSTRUCTS
#15MICRODYSTROPHIN NUCLEIC ACID GENE THERAPY CONSTRUCTS AND USES THEREOF
#16METHOD AND MEANS TO DELIVER miRNA TO TARGET CELLS
#17TREATMENT OF OCULAR DISEASES WITH FULLY-HUMAN POST-TRANSLATIONALLY MODIFIED ANTI-VEGF Fab
#18AAV5 CAPSID WITH NON-CANONICAL AMINO ACID INCORPORATION AND USES THEREOF
#19COMPOSITIONS USEFUL IN TREATMENT OF METACHROMATIC LEUKODYSTROPHY
#20Methods for Delivery of Polynucleotides by Adeno-Associated Virus for Lysosomal Storage Disorders
#21Gene Therapy For Neurodegenerative Disorders
#22NUCLEIC ACID MOLECULES AND USES THEREOF FOR NON-VIRAL GENE THERAPY
#23HUMAN PAH EXPRESSION CASSETTE FOR TREATMENT OF PKU BY LIVER-DIRECTED GENE REPLACEMENT THERAPY
#24GENE THERAPEUTICS FOR TREATING BONE DISORDERS
#25ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
#26ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR HIGH EFFICIENCY GENOME EDITING AND METHODS THEREOF
#27METHODS OF TREATING NEURODEGENERATIVE DISORDERS
#28COMPOSITIONS AND METHODS FOR IMPEDING TRANSCRIPTION OF EXPANDED MICROSATELLITE REPEATS
#29METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#30METHODS OF RECOMBINANT ADENO-ASSOCIATED VIRUS KIDNEY ADMINISTRATION
#31METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#32METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#33COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE
#34CARDIAC CELL REPROGRAMMING WITH MYOCARDIN AND ASCL1
#35EXPRESSION VECTOR FOR CHOLESTEROL 24-HYDROLASE IN THERAPY OF POLYGLUTAMINE REPEAT SPINOCEREBELLAR ATAXIAS
#36METHODS AND COMPOSITIONS RELATING TO THE TREATMENT OF TUMORS
#37METHODS AND COMPOSITIONS FOR DELIVERY OF IMMUNOTHERAPY AGENTS ACROSS THE BLOOD-BRAIN BARRIER TO TREAT BRAIN CANCER
#38VIRAL VECTOR CONSTRUCTS FOR DELIVERY OF NUCLEIC ACIDS ENCODING CYTOKINES AND USES THEREOF FOR TREATING CANCER
#39Compositions and Methods for Treatment of Ocular Disease Associated with Angiogenesis
#40ADENO-ASSOCIATED VIRUS WITH ENGINEERED CAPSID
#41OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#42IN VIVO REPROGRAMMING OF PHOTORECEPTOR CELLS
#43ENGINEERED MUSCLE TARGETING COMPOSITIONS
#44GENE THERAPY FOR HEARING LOSS
#45COMPOSITIONS AND METHODS FOR THE TREATMENT OF TAU-RELATED DISORDERS
#46HIGH EFFICIENCY LIBRARY-IDENTIFIED AAV VECTORS
#47PRODUCTION OF RECOMBINANT AAV VECTORS FOR TREATING MUSCULAR DYSTROPHY
#48METHODS FOR EVALUATING TREATMENTS FOR BESTROPHINOPATHIES
#49AAV2-MEDIATED GENE DELIVERY OF SFASL AS A NEUROPROTECTIVE THERAPY IN GLAUCOMA
#50GENE THERAPY FOR SPINAL MUSCULAR ATROPHY
#51AAV-MEDIATED THERAPIES FOR VISION LOSS ASSOCIATED WITH FRIEDREICH’S ATAXIA
#52MODIFIED AAV CONSTRUCTS AND USES THEREOF
#53ENGINEERED CIRCULAR RNA CIRCMIR-29B AND USE THEREOF IN PREPARATION OF MEDICINE FOR TREATING MUSCLE ATROPHY
#54RECOMBINANT ADENO-ASSOCIATED VIRUS WITH MODIFIED AAV CAPSID POLYPEPTIDES
#55COMPOSITIONS, KITS AND METHODS FOR TREATMENT OF COMPLEMENT-RELATED DISORDERS
#56ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
#57ADENO-ASSOCIATED VECTORS AND VIRIONS TO TREAT GALACTOSEMIA AND METHODS OF USE AND MANUFACTURE
#58SYSTEMS AND USES THEREOF FOR THE TREATMENT OF DMD-ASSOCIATED DISEASES
#59SYSTEMIC DELIVERY OF ADENO-ASSOCIATED VIRUS VECTOR EXPRESSING GAMMA-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY
#60COMPOSITIONS AND METHODS FOR IMPROVED TREATMENT OF X-LINKED MYOTUBULAR MYOPATHY
#61Gene Therapy For Neurodegenerative Disorders
#62GENE THERAPY FOR TUBEROUS SCLEROSIS
#63VECTOR ENCODING ROD-DERIVED CONE VIABILITY FACTOR AND HUMAN IGK SIGNAL SEQUENCE
#64METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV VECTORS
#65AAV Vectors
#66Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and AAVrh74 with Reduced Liver Tropism
#67METHODS AND COMPOSITIONS FOR ADMINISTERING OTOFERLIN DUAL VECTOR SYSTEMS
#68MODIFIED AAV CAPSIDS AND USES THEREOF
#69ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR TISSUE-TARGETED EXPRESSION OF THERAPEUTIC GENES
#70Retinal Disorders
#71Therapeutic Editing
#72USE OF MULLERIAN INHIBITING SUBSTANCE (MIS) PROTEINS FOR CONTRACEPTION AND OVARIAN RESERVE PRESERVATION
#73TREATMENT OF OCULAR DISEASES WITH FULLY-HUMAN POST-TRANSLATIONALLY MODIFIED ANTI-VEGF Fab
#74METHODS AND COMPOSITIONS FOR TRANSPORT, STORAGE, AND DELIVERY OF ADENO-ASSOCIATED VIRAL VECTOR AND OTHER MOLECULES
#75AAV VIRION ENCODING NEUROTROPHIC FACTOR AND USES THEREOF
#76Compositions and Methods for Treatment of Ocular Disease Associated with Angiogenesis
#77VECTORED PROPHYLAXIS OF SARS-COV-2
#78COMPOSITIONS AND METHODS FOR TREATMENT OF CRX-MEDIATED RETINOPATHIES
#79METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS (ALS)
#80SYNTHETIC NUCLEIC ACIDS INCLUDING ASTROCYTE-DIRECTED PROMOTER CONSTRUCTS AND METHODS OF USING THE SAME
#81MUTATION-INDEPENDENT GENE KNOCK-IN THERAPY TARGETING 5' UTR
#82LIVER SPECIFIC PRODUCTION OF ENPP1 OR ENPP3
#83VECTOR-MEDIATED IMMUNE TOLERANCE IN THE EYE
#84Compositions and Methods for Treatment of Ocular Disease Associated with Angiogenesis
#85Methods of Treating Human X-Linked Retinoschisis Using Gene Therapy
#86METHODS AND COMPOSITIONS FOR TREATMENT OF CYSTIC FIBROSIS
#87COMPOSITIONS AND METHODS FOR USING ALTERNATIVE SPLICING TO CONTROL SPECIFICITY OF GENE THERAPY
#88ADENO-ASSOCIATED VIRAL VECTOR VARIANTS
#89Self-complementary AAV vectors carrying dominant negative RhoA and methods of use to treat ocular diseases
#90ABCA4 TRANS-SPLICING MOLECULES
#91Compositions and methods of treating ocular diseases
#92Adeno-associated virus with engineered capsid
#93AAV TREATMENT OF HUNTINGTON’S DISEASE
#94COMPOSITIONS FOR AND METHODS OF IMPROVING FLUID FLUX IN THE BRAIN
#95VIRAL VECTORS AND NUCLEIC ACIDS FOR REGULATED GENE THERAPY
#96TREATMENT OF LIPODYSTROPHY
#97Treatment of mucopolysaccharidosis II with recombinant human iduronate-2-sulfatase (IDS)
#98Cardiac cell reprogramming with myocardin and ASCL1
#99Antibody-evading virus vectors
#100Compositions For and Methods of Enhancing Spinal Cord Tissue Regeneration
#101SYNTHETIC PROMOTERS FOR GENE THERAPY AND PROTEIN EXPRESSION
#102Optimized expression cassettes for gene therapy
#103ABCA4 TRANS-SPLICING MOLECULES
#104ENGINEERED VIRAL CAPSIDS AND METHODS OF USE
#105Gene Therapy For Neurodegenerative Disorders
#106Compositions and methods for regulating production of a fusion protein and ribonucleic acid
#107COMPOSITIONS FOR AND METHODS OF ENHANCING TISSUE REGENERATION
#108METHODS AND COMPOSITIONS FOR TREATING TECPR2-ASSOCIATED DISEASE AND DISORDERS WITH A VIRAL VECTOR
#109NOVEL COMPOSITIONS WITH TISSUE-SPECIFIC TARGETING MOTIFS AND COMPOSITIONS CONTAINING SAME
#110ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN CHANDELIER CELLS
#111METHODS AND COMPOSITIONS FOR RESTRICTING BIODISTRIBUTION OF AAV
#112DUAL-AAV VECTOR DELIVERY OF PCDH15 AND USES THEREOF
#113METHODS AND MATERIALS FOR AMELIORATING CREATINE DEFICIENCY DISORDERS
#114Compositions and Methods for Treating Spinal Muscular Atrophy
#115LIVER DE-TARGETED RECOMBINANT AAV CAPSID PROTEINS
#116MICROGLIAL SELECTIVE GENE EXPRESSION VECTOR
#117VARIANT TXNIP COMPOSITIONS AND METHODS OF USE THEREOF FOR THE TREATMENT OF DEGENERATIVE OCULAR DISEASES
#118METHODS OF TREATING METACHROMATIC LEUKODYSTROPHY
#119ADENO-ASSOCIATED VIRAL VECTORS FOR TREATMENT OF NIEMANN-PICK DISEASE TYPE C
#120DUAL AAV VECTOR-MEDIATED DELETION OF LARGE MUTATIONAL HOTSPOT FOR TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#121VECTORIZED FACTOR XII ANTIBODIES AND ADMINISTRATION THEREOF
#122ADENO-ASSOCIATED VIRUSES FOR OCULAR DELIVERY OF GENE THERAPY
#123ENGINEERED AAV VECTORS
#124COMPOSITION INCLUDING PLGF FOR PREVENTION OR TREATMENT OF NEUROPSYCHOLOGIC DISEASE
#125GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
#126ADENO-ASSOCIATED VIRAL VECTOR VARIANTS
#127ADENO-ASSOCIATED VIRUS FOR DELIVERY OF KH902 (CONBERCEPT) AND USES THEREOF
#128CODON OPTIMIZED RPGRORF15 GENES AND USES THEREOF
#129G-PROTEIN-GATED-K+ CHANNEL-MEDIATED ENHANCEMENTS IN LIGHT SENSITIVITY IN ROD-CONE DYSTROPHY (RCD)
#130COMPOSITIONS AND METHOD OF USE OF MUTANT ACE2 DECOY VARIANTS
#131USE OF NOVEL MIRNA-BINDING SITE CASSETTES FOR ANTIGEN-PRESENTING CELL DETARGETING OF TRANSGENE EXPRESSION BY RAAV GENE THERAPY
#132AAV-VECTORS FOR USE IN GENE THERAPY OF CHOROIDEREMIA
#133TREATMENT OF RPE65-ASSOCIATED EYE DISEASES AND DISORDERS
#134NOVEL ENGINEERED CAPSID SEROTYPE OF RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR WITH ENHANCED TRANSDUCTION EFFICIENCY AND WIDESPREAD DISTRIBUTION IN THE BRAIN
#135GENE THERAPY VECTOR FOR eEF1A2 AND USES THEREOF
#136Compositions of DNA Molecules, Methods of Making Therefor, and Methods of Use Thereof
#137COMPOSITIONS AND METHODS FOR THE TREATMENT OF OCULAR DISEASES
#138ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
#139MODIFIED ADENO-ASSOCIATED VIRUS VECTORS THAT EVADE NEUTRALIZING ANTIBODIES AND USES THEREOF
#140Compositions and methods for delivery of AAV
#141COMPOSITION AND METHOD
#142OVEREXPRESSION OF LEMD2, LEMD3, OR CHMP7 AS A THERAPEUTIC MODALITY FOR TAUOPATHY
#143NOVEL AAV CAPSIDS AND COMPOSITIONS CONTAINING SAME
#144Methods of predicting ancestral virus sequences and uses thereof
#145Adeno-associated virus variant capsids and methods of use thereof
#146MODIFIED VIRAL COMPOSITIONS FOR VIRAL TRANSDUCTION
#147VIRAL PARTICLES FOR USE IN TREATING TAUOPATHIES SUCH AS ALZHEIMER'S DISEASES BY GENE THERAPY
#148ADENO-ASSOCIATED VIRAL VECTOR FOR GLUT1 EXPRESSION AND USES THEREOF
#149MICRODYSTROPHIN GENE THERAPY CONSTRUCTS AND USES THEREOF
#150GENE THERAPY FOR STXBP1 ENCEPHALOPATHY
#151GENE EDITING TO IMPROVE JOINT FUNCTION
#152METHODS AND COMPOSITIONS FOR DUAL GLYCAN BINDING AAV2.5 VECTOR
#153NRF2 Activator for Use in Treating Dilated Cardiomyopathies
#154MINI-PROMOTER
#155METHOD FOR TREATING ALZHEIMER'S DISEASE BY TARGETING MAPT GENE
#156METHODS OF MAKING AND USING A VACCINE AGAINST CORONAVIRUS
#157METHODS OF TREATING DUCHENNE MUSCULAR DYSTROPHY USING AAV MINI-DYSTROPHIN GENE THERAPY
#158COMPOSITIONS AND METHODS FOR INCREASING OR ENHANCING TRANSDUCTION OF GENE THERAPY VECTORS AND FOR REMOVING OR REDUCING IMMUNOGLOBULINS
#159REGULATORY NUCLEIC ACID SEQUENCES
#160ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#161POLYNUCLEOTIDE
#162MUSCLE-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
#163BIFUNCTIONAL BRIDGING COMPOSITIONS FOR VIRAL TRANSDUCTION
#164GENE THERAPY FOR BARDET-BIEDL SYNDROME
#165CILP-1 Inhibitors for Use in the Treatment of Dilated Cardiomyopathies
#166BBB-TARGETED GAA DELIVERED AS GENE THERAPY TREATS CNS AND MUSCLE IN POMPE DISEASE MODEL MICE
#167ADENO-ASSOCIATED VIRUS VECTOR AND USE THEREOF
#168ENGINEERING BROADLY REACTIVE CORONAVIRUS VACCINES AND RELATED DESIGNS AND USES
#169VECTORS ENCODING A GLUCOSE-6-PHOSPHATASE (G6PASE-A) FOR GENE THERAPY
#170SYSTEMS AND METHODS FOR TREATING LEVODOPA DYSKINESIA, ENHANCING MOTOR BENEFIT, AND DELAYING DISEASE PROGRESSION
#171CRISPR-MEDIATED GENOME EDITING WITH VECTORS
#172COMPOSITIONS AND METHODS FOR TREATING OCULAR DISORDERS
#173Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#174Compositions and Methods for Modifying Target RNAs
#175GENE THERAPY OF NIEMANN-PICK DISEASE TYPE C
#176GENE EDITING OF ANTICOAGULANT FACTORS
#177COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS
#178PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#179IMMUNOSUPPRESSIVE AGENTS AND VIRAL DELIVERY RE-DOSING METHODS FOR GENE THERAPY
#180COMPOSITIONS USEFUL FOR TREATMENT OF POMPE DISEASE
#181LIVING CELLS ENGINEERED WITH POLYPHENOL-FUNCTIONALIZED BIOLOGICALLY ACTIVE NANOCOMPLEXES
#182PERSISTENT MEMORY T-CELL RESPONSES TO CANCER AND INFECTIOUS-DISEASE ANTIGENS BY MANIPULATION OF AMINO ACID-CATABOLISM PATHWAYS
#183TARGETING LTBP3 FOR NONALCOHOLIC STEATOHEPATITIS (NASH)-INDUCED LIVER FIBROSIS
#184MODIFIED NUCLEIC ACIDS ENCODING ASPARTOACYLASE (ASPA) AND VECTOR FOR GENE THERAPY
#185G-PROTEIN-GATED-K+ CHANNEL-MEDIATED ENHANCEMENTS IN LIGHT SENSITIVITY IN ROD-CONE DYSTROPHY (RCD)
#186VECTORS FOR THE TREATMENT OF ACID CERAMIDASE DEFICIENCY
#187METHOD OF TREATING GENE THERAPY ASSOCIATED TOXICITY WITH ANTIBIOTICS
#188COMPOSITIONS AND METHODS FOR MODULATING RPGR EXPRESSION
#189VIRAL VECTOR DOSING PROTOCOLS
#190ANTISENSE SEQUENCES FOR TREATING AMYOTROPHIC LATERAL SCLEROSIS
#191AAV CAPSIDS VARIANTS AND USES THEREOF
#192CARDIAC CELL REPROGRAMMING WITH MICRORNAS AND OTHER FACTORS
#193CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS AND METHODS THEREFOR
#194GENE THERAPY FOR MAPLE SYRUP URINE DISEASE
#195Gene therapy for neurodegenerative disorders
#196OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#197METHODS FOR ENHANCING RECOMBINANT ADENO-ASSOCIATED VIRUS YIELD
#198COMPOSITIONS AND METHODS FOR THE TREATMENT OF SANFILIPPO DISEASE AND OTHER DISORDERS
#199GENE VECTOR CONTROL BY CARDIOMYOCYTE-EXPRESSED MICRORNAS
#200CLOSED-END DNA PRODUCTION WITH INVERTED TERMINAL REPEAT SEQUENCES
#201RAAV-MEDIATED IN VIVO DELIVERY OF SUPPRESSOR TRNAS
#202Gene therapy for neurodegenerative disorders
#203VECTORIZED ANTIBODIES (vAb) AND USES THEREOF
#204COMPOSITIONS AND METHODS FOR CIRCULAR RNA EXPRESSION
#205A PROCESS FOR PRODUCING A PLURALITY OF SUMOYLATION TARGET-SITE MODIFIED AAV VECTOR AND PRODUCT THEREOF
#206Methods and compositions for delivery of immunotherapy agents across the blood-brain barrier to treat brain cancer
#207ADENO ASSOCIATED VIRAL VECTORS
#208Therapeutic editing
#209SECRETABLE PROTEIN INDUCED IMMUNE TOLERIZATION AND TREATMENT OF AUTOIMMUNE, ALLERGIC AND OTHER DISEASES AND DISORDERS
#210COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE
#211METHOD FOR IDENTIFYING REGULATORY ELEMENTS CONFORMATIONALLY
#212TREATMENT OF DISEASES INVOLVING DEFICIENCY OF ENPP1 OR ENPP3
#213Prostate neoantigens and their uses
#214Recombinant Adeno-Associated Virus Delivery of Exon 2-Targeted U7SNRNA Polynucleotide Constructs
#215Zinc Finger Protein Transcription Factors for Treatment of Prion Disease
#216SELECTIVE EXPANSION OF GENE-TARGETED CELLS
#217EFFECTIVE DOSAGES OF AN ADENOVIRAL-BASED BIOLOGICAL DELIVERY AND EXPRESSION SYSTEM FOR USE IN THE TREATMENT OF OSTEOARTHRITIS IN HUMANS, AND COMPOSITIONS COMPRISING THE SAME
#218ENGINEERED CIRCULAR RNA CIRCMIR-29B AND USE THEREOF IN PREPARATION OF MEDICINE FOR TREATING MUSCLE ATROPHY
#219Pharmaceutical composition for diagnosing, preventing or treating liver cancer using SSU72 protein or a polynucleotide encoding the same
#220METHODS FOR TREATING PATIENTS HAVING CFH MUTATIONS WITH CFH-ENCODING VECTORS
#221VECTOR SYSTEM
#222GROWTH FACTOR RESTORATION
#223METHODS AND AAV VECTORS FOR IN VIVO TRANSDUCTION
#224ENHANCING VIRAL VECTOR-BASED GENE THERAPY
#225TREATMENT OF X-LINKED JUVENILE RETINOSCHISIS
#226PROGRAMMABLE DNA BASE EDITING BY NME2CAS9-DEAMINASE FUSION PROTEINS
#227COMPOSITIONS AND METHODS FOR TREATING AND PREVENTING MACULAR DEGENERATION
#228REGULATION OF GENE EXPRESSION BY APTAMER-MEDIATED MODULATION OF ALTERNATIVE SPLICING
#229AAV CAPSID VARIANTS FOR GENE THERAPY
#230AAV VARIANTS WITH ENHANCED TROPISM
#231Modified AAV Capsid Proteins for Treatment of Arthritic Disease
#232MPS MODIFIED PEPTIDES AND USE THEREOF
#233METHODS AND COMPOSITIONS FOR TREATING METABOLIC IMBALANCE IN NEURODEGENERATIVE DISEASE
#234ENGINEERED ADENO-ASSOCIATED VIRUS CAPSIDS
#235Use of inverted terminal repeats (ITRS) from adeno-associated virus serotypes 8 and RH.39 in gene therapy vectors
#236Adeno-associated virus variant capsids and methods of use thereof
#237ENGINEERED ITR SEQUENCES AND METHODS OF USE
#238EXPRESSION VECTOR AGAINST SEVERE ACUTE RESPIRATORY SYNDROME VIRUS SARS-COV-2
#239GENE THERAPY FOR ALZHEIMER`S DISEASE
#240Use of novel miRNA-binding site cassettes for antigen-presenting cell detargeting of transgene expression by rAAV gene therapy vectors
#241CRISPR METHODS FOR TREATING CANCERS
#242Methods and Compositions for Treating Obesity and/or Skin Disorders
#243ENGINEERED MUSCLE TARGETING COMPOSITIONS
#244COMPOSITIONS USEFUL IN TREATMENT OF METACHROMATIC LEUKODYSTROPHY
#245Peptide-Modified Hybrid Recombinant Adeno-Associated Virus Serotype Between AAV9 and AAVrh74 with Reduced Liver Tropism and Increased Muscle Transduction
#246Modified AAV capsids and uses thereof
#247OPTIMIZED GENE THERAPY TARGETING RETINAL CELLS
#248Insulin gene therapy
#249METHODS FOR TREATING NEURODEGENERATIVE DISORDERS
#250VIRUS COMPOSITIONS WITH ENHANCED SPECIFICITY IN THE BRAIN
#251INDUCING IMMUNE TOLERANCE BY rAAV VECTORS
#252Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#253OPTIMIZED SUMF1 GENES AND EXPRESSION CASSETTES AND THEIR USE
#254INTRANASAL ADMINISTRATION OF ADENO-ASSOCIATED VIRUS TO TREAT CENTRAL NERVOUS SYSTEM DISORDERS
#255PRODUCTION OF LARGE-SIZED QUASIDYSTROPHINS USING OVERLAPPING AAV VECTORS
#256COMPOSITIONS USEFUL IN TREATMENT OF RETT SYNDROME
#257INTRATHECAL AND INTRAVENOUS COMBINATION GENE THERAPY FOR THE TREATMENT OF INFANTILE BATTEN DISEASE
#258GENE THERAPY FOR TREATING OR PREVENTING VISUAL EFFECTS IN BATTEN DISEASE
#259RECOMBINANT AAV-CRUMBS HOMOLOGUE COMPOSITION AND METHODS FOR TREATING LCA-8 AND PROGRESSIVE RP
#260Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer
#261BRAIN REPAIR AFTER TRAUMATIC BRAIN INJURY THROUGH NEUROD1-MEDIATED ASTROCYTE-TO-NEURON CONVERSION
#262ADENO-ASSOCIATED VIRUS WITH ENGINEERED CAPSID
#263COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#264COMPOSITIONS AND METHODS TO TREAT BIETTI CRYSTALLINE DYSTROPHY
#265Methods and compositions for dual glycan binding AAV2.5 vector
#266COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#267RECOMBINANT ADENO-ASSOCIATED VIRUS FOR TREATMENT OF GRN-ASSOCIATED ADULT-ONSET NEURODEGENERATION
#268Targeted In Vivo Genome Modification
#269Adeno-Associated Virus Delivery of CLN3 Polynucleotide
#270AAV-MEDIATED GENE THERAPY RESTORING THE OTOFERLIN GENE
#271Compositions and methods for regulating production of a fusion protein and ribonucleic acid
#272Recombinant adeno associated virus encoding clarin-1 and uses thereof
#273Human PAH expression cassette for treatment of PKU by liver-directed gene replacement therapy
#274TREATMENT OF OCULAR NEOVASCULARIZATION USING ANTI-VEGF PROTEINS
#275Compositions of DNA molecules, methods of making therefor, and methods of use thereof
#276ASCL1 VECTOR
#277NEUROD1 COMBINATION VECTOR
#278RECOMBINANT ADENO-ASSOCIATED VIRAL VECTOR FOR GENE DELIVERY
#279METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#280Systemic delivery of adeno-associated virus vector expressing gamma-sarcoglycan and the treatment of muscular dystrophy
#281Liver-specific viral promoters and methods of using the same
#282Coronavirus immunogenic compositions and uses thereof
#283Adeno-associated virus vectors for treatment of Rett syndrome
#284THERAPEUTIC ADENO-ASSOCIATED VIRUS FOR TREATING POMPE DISEASE
#285Gene therapy for juvenile Batten disease
#286Use of neuroglobin agonist for preventing or treating mitochondrial RCCI and/or RCCIII deficiency disease
#287Method of detecting and/or identifying adeno-associated virus (AAV) sequences and isolating novel sequences identified thereby
#288INHIBITORS OF CACNA1A/ALPHA1A SUBUNIT INTERNAL RIBOSOMAL ENTRY SITE (IRES) AND METHODS OF TREATING SPINOCEREBELLAR ATAXIA TYPE 6
#289METHODS OF PREDICTING ANCESTRAL VIRUS SEQUENCES AND USES THEREOF
#290FABRY DISEASE GENE THERAPY
#291AAV treatment of Huntington's disease
#292NUCLEIC ACID CONSTRUCTS AND GENE THERAPY VECTORS FOR USE IN THE TREATMENT OF WILSON DISEASE
#293IL-10-CONTAINING VACCINES AND USES THEREOF
#294MUTANT REVERSE TETRACYCLINE TRANSACTIVATORS FOR EXPRESSION OF GENES
#295Codon optimized otoferlin AAV dual vector gene therapy
#296Compositions and methods for preventing and treating coronavirus infection—SARS-CoV-2 vaccines
#297GENE THERAPY FOR OXIDATIVE STRESS
#298Adeno-associated virus variant capsids and methods of use thereof
#299METHOD AND MEANS TO DELIVER miRNA TO TARGET CELLS
#300COMPOSITIONS AND METHODS FOR DELIVERY OF AAV