112289 ⎘
Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
CHIMERIC PROTEINS COMPRISING MEMBRANE BOUND IL-12 WITH PROTEASE CLEAVABLE LINKERS
#2GENE THERAPY FOR JUVENILE BATTEN DISEASE
#3HYBRID AAV VECTOR ENHANCING TRANSGENE EXPRESSION IN THE LIVER
#4MODIFIED GLUCOKINASE NUCLEIC ACIDS FOR TREATING DIABETES
#5Compositions and Methods for Treating Disorders Associated with Cone Photoreceptor Cells
#6ENGINEERED NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USES THEREOF
#7ENGINEERED MUSCLE TARGETING COMPOSITIONS
#8LIVER-SPECIFIC VIRAL PROMOTERS AND METHODS OF USING THE SAME
#9NUCLEIC ACID REGULATORY ELEMENTS FOR GENE EXPRESSION IN THE LIVER AND METHODS OF USE
#10NUCLEIC ACID REGULATORY ELEMENTS FOR CONSTITUTIVE GENE EXPRESSION AND METHODS OF USE
#11COMPOSITIONS, SYSTEMS, AND METHODS FOR REDUCING ADIPOSE TISSUE
#12TARGETED GENE INSERTION FOR IMPROVED IMMUNE CELLS THERAPY
#13NUCLEIC ACID CONSTRUCTS FOR VA RNA TRANSCRIPTION
#14GENE THERAPY TREATMENT
#15COMPOSITIONS AND METHODS TO TREAT ALZHEIMER'S DISEASE AND OTHER BRAIN DISEASES
#16METHODS TO MODULATE PROTEIN TRANSLATION EFFICIENCY
#17MUSCLE-SPECIFIC EXPRESSION CASSETTES
#18GENE THERAPY FOR TREATING CITRULLENEMIA
#19COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#20ANTIBODY GENE THERAPY FOR TREATMENT AND PREVENTION OF INFECTION BY RABIES LYSSAVIRUS
#21COMPOSITIONS AND METHODS FOR TREATMENT OF CARDIAC DISORDERS
#22ENGINEERED MUSCLE TARGETING COMPOSITIONS
#23LOCO-REGIONAL PERFUSION OF A KIDNEY
#24METHODS OF DELIVERING A VIRAL VECTOR TO A KIDNEY
#25Variant AAV and Compositions, Methods and Uses for Gene Transfer to Cells, Organs and Tissues
#26COMPOSITIONS FOR AND METHODS OF EDITING THE GENOME
#27AAV CAPSID PROTEINS HAVING MUTATIONS IN THE VP1 REGION
#28METHODS AND COMPOSITIONS FOR TREATING LEUKODYSTROPHIES
#29GENE THERAPY FOR TREATING PROPIONIC ACIDEMIA
#30COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS USING OTOFERLIN DUAL VECTOR SYSTEMS
#31ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#32ENGINEERED LIVER-SPECIFIC CORE PROMOTERS AND THEIR APPLICATIONS
#33PROMOTER SEQUENCE FOR INITIATING GENE SPECIFICALLY IN MAMMALIAN MUSCLE AND USE THEREOF
#34MANUFACTURING OF THERAPEUTIC SATELLITE CELLS FOR TREATING MUSCULAR DYSTROPHY
#35AAV-MEDIATED DELIVERY OF RGS10 TO MICROGLIA
#36METHODS OF TREATING MUSCULAR DYSTROPHY
#37LINC COMPLEX INHIBITING POLYPEPTIDES
#38AAV GENE THERAPY FOR TREATING A NERVOUS SYSTEM DISEASE
#39NON-HUMAN ANIMALS EXPRESSING EXOGENOUS TERMINAL DEOXYNUCLEOTIDYLTRANSFERASE
#40GENERATION OF NEURONS BY REPROGRAMMING OF OLIGODENDROCYTES AND OLIGODENDROCYTE PRECURSOR CELLS
#41IMMUNE-CLOAKING ANTITUMOR ADENOVIRUS
#42NEURONAL PROMOTERS AND USES THEREOF
#43RETRO-AAV AND USE IN TREATING NEURODEGENERATIVE DISEASES
#44AAV-BASED GENE THERAPY FOR HEREDITARY ANTITHROMBIN DEFICIENCY
#45MICRO-DYSTROPHINS AND RELATED METHODS OF USE
#46FUSOSOME COMPOSITIONS AND USES THEREOF
#47MUSCLE TROPIC RAAV
#48REPLICATION-DEFECTIVE ARENAVIRUS VECTORS
#49SYNAPSE SURGERY TOOLS AND ASSOCIATED METHODS FOR NEURAL CIRCUIT-SPECIFIC SYNAPSE ABLATION AND MODIFICATION
#50MINIGENE THERAPY
#51ERYTHROID-SPECIFIC PROMOTER AND METHOD OF USE THEREOF
#52GENE THERAPY FOR TREATING NEURODEGENERATIVE DISEASES
#53PRODUCTS AND METHODS FOR TREATING DISEASES OR CONDITIONS ASSOCIATED WITH MUTANT OR PATHOGENIC KCNQ3 EXPRESSION
#54ENGINEERING PHOTOSYNTHETIC ELECTRON TRANSPORT CHAIN FOR IMPROVED PHOTOSYNTHETIC EFFICIENCY
#55ADENO-ASSOCIATED VIRUS VIRION FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
#56CELL CONVERSION
#57Adeno-Associated Virus Factor VIII Vectors
#58VECTORIZED LANADELUMAB AND ADMINISTRATION THEREOF
#59GENE THERAPY
#60MICRODYSTROPHIN GENE THERAPY ADMINISTRATION FOR TREATMENT OF DYSTROPHINOPATHIES
#61METHOD FOR EXPRESSING A MUSCLE-SPECIFIC GENE AND CASSETTES FOR SAME
#62Silk Gland Recombinant Expression Vector of Silkworm (Bombyx Mori) Expressing Human Epidermal Growth Factor, Preparation Method and Use Thereof
#63INHIBITORY NEURON-SPECIFIC PROMOTER
#64METHODS OF ON DEMAND IN VIVO PHOTOTAGGING
#65COMPOSITIONS AND METHODS FOR INDUCIBLE ALTERNATIVE SPLICING REGULATION OF GENE EXPRESSION
#66METHODS OF TREATING SOLID OR LYMPHATIC TUMORS BY COMBINATION THERAPY
#67MACROPHAGE-SPECIFIC PROMOTERS AND USES THEREOF
#68COMPOSITIONS AND METHODS FOR TREATING NON-AGE-ASSOCIATED HEARING IMPAIRMENT IN A HUMAN SUBJECT
#69GENE THERAPY TO IMPROVE VISION
#70NUCLEIC ACID CONSTRUCTS AND VECTORS FOR PODOCYTE SPECIFIC EXPRESSION
#71MICRO-DYSTROPHIN FOR HEART PROTECTION
#72GENE THERAPY OF HIPPO SIGNALING IMPROVES HEART FUNCTION IN A CLINICALLY RELEVANT MODEL
#73MINIMAL NEPHRIN PROMOTER
#74METHOD AND COMPOSITIONS FOR NEURONAL REPROGRAMMING
#75VECTORS FOR DELIVERY OF HUMAN GROWTH HORMONE GENE AND MULTIPLE THERAPEUTIC GENES INTO CENTRAL NERVOUS SYSTEM BY CROSSING BLOOD BRAIN BARRIER
#76EXPRESSION VECTORS COMPOSITION
#77ADENO-ASSOCIATED VIRUS COMPOSITIONS FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY
#78Vector
#79B-CELL LYMPHOMA 2-ASSOCIATED ANTHANOGENE 3 (BAG3) GENE THERAPY USING AAV VECTOR
#80HSV VECTORS
#81RAAV-CONE OPSIN COMPOSITIONS AND METHODS FOR TREATING BLUE CONE MONOCHROMACY AND COLOR BLINDNESS
#82LENTIVIRAL VECTOR
#83CAPSIDS FOR PLAKOPHILLIN-2 GENE THERAPY
#84PLAKOPHILLIN-2 GENE THERAPY TREATMENT METHODS
#85TAU BINDING COMPOUNDS
#86A GENE-BASED APPROACH TO CONFER LONG-LASTING PROTECTION FROM OPIOID USE DISORDER
#87METHODS AND COMPOSITIONS FOR TRANSDUCING LYMPHOCYTES AND REGULATING THE ACTIVITY THEREOF
#88ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN EXCITATORY CORTICAL NEURONS
#89ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN THE CEREBELLUM AND A SECONDARY CELL TYPE
#90METHODS AND MATERIALS FOR TREATING SYNGAP1-ASSOCIATED NEURODEVELOPMENTAL DISORDERS
#91TISSUE-SPECIFIC PROMOTER AND USE THEREOF
#92GENE EDITING METHODS FOR TREATING ALPHA-1 ANTITRYPSIN (AAT) DEFICIENCY
#93PROMOTER SWITCHES FOR TISSUE-SPECIFIC EXPRESSION
#94GENE THERAPY DELIVERY COMPOSITIONS AND METHODS FOR TREATING HEARING LOSS
#95CELL-TYPE SPECIFIC REGULATORY ELEMENTS FOR PHOTORECEPTORS
#96GENETIC TARGETING OF CELLULAR OR NEURONAL SUB-POPULATIONS
#97METHOD TO STIMULATE REGENERATION OF RETINAL GANGLION CELLS
#98Cochlear and/or vestibular cell specific promoter and use thereof
#99THERAPEUTIC ADENO-ASSOCIATED VIRUS USING CODON OPTIMIZED NUCLEIC ACID ENCODING ALPHA-GLUCOSIDASE (GAA) FOR TREATING POMPE DISEASE, WITH SIGNAL PEPTIDE MODIFICATIONS
#100MODIFIED MUSCLE-SPECIFIC PROMOTERS
#101METHODS AND COMPOSITIONS FOR TREATING RBM20 RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
#102Viral Vectors For Treating Neurogenic Detrusor Overactivity
#103GENE AUGMENTATION THERAPIES FOR INHERITED RETINAL DEGENERATION CAUSED BY MUTATIONS IN THE PRPF31 GENE
#104COMPOSITIONS AND METHODS FOR TREATING AND/OR PREVENTING GLYCOGEN STORAGE DISEASES
#105CRISPR/SpCas9 VARIANT AND METHODS FOR ENHANCED CORRECTION OF DUCHENNE MUSCULAR DYSTROPHY MUTATIONS
#106SEQUENCE OF ACTIVATED PROTEIN C
#107GENE THERAPY COMPOSITION AND TREATMENT OF RIGHT VENTRICULAR ARRHYTHMOGENIC CARDIOMYOPATHY
#108ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN DOPAMINERGIC NEURONS
#109SYNTHETIC CANCER-SPECIFIC PROMOTERS
#110ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#111TRANSCRIPTIONAL AND TRANSLATIONAL DUAL REGULATED ONCOLYTIC HERPES SIMPLEX VIRUS VECTORS
#112PROMOTER FOR THE EXPRESSION OF GENES IN PARTICULAR EGR2-POSITIVE CELLS IN LAYER 2/3 OF MOUSE CORTEX
#113REGULATORY ELEMENT FOR CELL TYPE SPECIFIC EXPRESSION OF GENES IN SPINAL MOTOR NEURONS
#114OPTIMIZED CLN5 GENES AND EXPRESSION CASSETTES AND THEIR USE
#115REGULATORY NUCLEIC ACID SEQUENCES
#116METHOD FOR DELIVERING RNA TO NEURONS TO TREAT HERPES INFECTIONS
#117HYBRID REGULATORY ELEMENTS
#118ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF ALPHA-SARCOGLYCAN AND THE TREATMENT OF MUSCULAR DYSTROPHY
#119ENGINEERED LIVER-SPECIFIC ENHANCERS AND THEIR APPLICATIONS
#120PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#121SCHWANN CELL-SPECIFIC PROMOTER
#122RIG1 Inhibition and Therapeutic Viral Protein Expression
#123COMPOSITIONS FOR TREATING NEUROLOGICAL DISEASE
#124VECTORS AND COMPOSITIONS FOR GENE AUGMENTATION OF CRUMBS COMPLEX HOMOLOGUE 1 (CRB1) MUTATIONS
#125PROMOTER FOR THE EXPRESSION OF GENES IN PARTICULAR CUX1-POSITIVE CELLS IN LAYER 2/3 OF MOUSE CORTEX
#126TRANSGENIC EXPRESSION CASSETTE FOR TREATING MUSCULAR DYSTROPHY
#127AAV GENE THERAPY FOR THE KIDNEY
#128Nucleic Acid-Based Compositions and Methods for Treating Small Vessel Diseases
#129VIRAL VECTOR CONSTRUCTS FOR DELIVERY OF NUCLEIC ACIDS ENCODING CYTOKINES AND USES THEREOF FOR TREATING CANCER
#130CONSTRUCTS COMPRISING NEURONAL VIABILITY FACTORS AND USES THEREOF
#131ENGINEERED MUSCLE TARGETING COMPOSITIONS
#132OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#133GENE THERAPY FOR LAMIN A-ASSOCIATED DEFICIENCIES
#134ENGINEERED MUSCLE TARGETING COMPOSITIONS
#135METHODS AND COMPOSITIONS FOR TREATING BAG-3 RELATED CARDIOMYOPATHY WITH A VIRAL VECTOR
#136METHODS AND COMPOSITIONS FOR TREATING MYBPC3 RELATED HYPERTROPHIC CARDIOMYOPATHY WITH A VIRAL VECTOR
#137RESCUING VOLTAGE-GATED SODIUM CHANNEL FUNCTION IN INHIBITORY NEURONS
#138ENHANCERS FOR DIRECTED EXPRESSION OF GENES IN NEURONAL CELL POPULATIONS, COMPOSITIONS AND METHODS THEREOF
#139SYNTHETIC RECEPTORS
#140METHODS AND MATERIALS FOR GALGT2 GENE THERAPY
#141Compositions and Methods for Cell-Specific Expression of Target Genes
#142COMPOSITIONS COMPRISING KOZAK SEQUENCES SELECTED FOR ENHANCED EXPRESSION
#143COMPOSITIONS AND METHODS FOR TREATING A MUSCULAR DYSTROPHY
#144NOCICEPTOR-SPECIFIC GENE REGULATORY ELEMENTS FOR THE TREATMENT OF PAIN
#145RETGC Gene Therapy
#146COMPOSITIONS AND METHODS FOR ADENO-ASSOCIATED (AAV) VIRUS DNASE EXPRESSION
#147AAV PARTICLES COMPRISING A LIVER-TROPIC CAPSID PROTEIN AND ALPHA-GALACTOSIDASE AND THEIR USE TO TREAT FABRY DISEASE
#148METHODS AND COMPOSITIONS FOR DIRECT LINEAGE REPROGRAMMING OF ASTROCYTES TO OLIGODENDROCYTE LINEAGE CELLS
#149AUF1 COMBINATION THERAPIES FOR TREATMENT OF MUSCLE DEGENERATIVE DISEASE
#150ENGINEERED DNA BINDING PROTEINS
#151TREATMENT OF HEREDITARY ANGIOEDEMA WITH AAV GENE THERAPY VECTORS AND THERAPEUTIC FORMULATIONS
#152PRODUCTS AND METHODS FOR MYELIN PROTEIN ZERO SILENCING AND TREATING CMT1B DISEASE
#153LIVER-SPECIFIC EXPRESSION CASSETTES, VECTORS AND USES THEREOF FOR EXPRESSING THERAPEUTIC PROTEINS
#154ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
#155GENE THERAPY FOR DUCHENNE MUSCULAR DYSTROPHY
#156EMBRYONIC CELL CULTURES AND METHODS OF USING THE SAME
#157Compositions and methods for treating non-age-associated hearing impairment in a human subject
#158TARGETED GENE THERAPY FOR DM-1 MYOTONIC DYSTROPHY
#159SYSTEMS AND METHODS FOR THE TARGETED PRODUCTION OF A THERAPEUTIC PROTEIN WITHIN A TARGET CELL
#160FRATAXIN GENE THERAPY
#161Compositions For and Methods of Editing the Genome
#162AN ENGINEERED YEAST CELL FOR THE DELIVERY OF ANTIBIOTIC-INACTIVATING ENZYMES
#163ENGINEERED ITR SEQUENCES AND METHODS OF USE
#164HYBRID PROMOTERS FOR GENE EXPRESSION IN MUSCLES AND IN THE CNS
#165TROPONIN C (TNNC1) GENE THERAPY USING AAV VECTOR
#166ANELLOVIRUS CONSTRUCTS COMPRISING RECOMBINATION SITES AND CROSS-PACKAGING OF ANELLOVECTOR PARTICLES AND GENETIC ELEMENTS
#167DIAPHRAGM-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
#168ENHANCERS DRIVING EXPRESSION IN MOTOR NEURONS
#169KCNV2 GENE THERAPY
#170Compositions and Methods for Treating Fabry Disease
#171ENHANCER SEQUENCES SPECIFIC FOR CARDIAC FIBROBLASTS AND METHODS OF USE THEREOF
#172Optimized RPE65 Promoter and Coding Sequences
#173GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
#174COMPOSITION AND METHOD FOR TREATING HEMOPHILIA B
#175ADENO-ASSOCIATED VIRAL (AAV) VECTORS FOR TISSUE-TARGETED EXPRESSION OF THERAPEUTIC GENES
#176ON DEMAND EXPRESSION OF EXOGENOUS FACTORS IN LYMPHOCYTES TO TREAT HIV
#177A shRNA, its recombinant expression vector, transformant, anti-heart failure drug, preparation method, and use as drug thereof
#178MODIFIED INSULIN AND GLUCOKINASE NUCLEIC ACIDS FOR TREATING DIABETES
#179NON-VIRAL DELIVERY OF DNA FOR PROLONGED POLYPEPTIDE EXPRESSION IN VIVO
#180ECTOPICALLY EXPRESSED TRANSCRIPTION FACTORS AND USES THEREOF
#181MODIFIED AAV CAPSIDS AND USES THEREOF
#182PRODUCTS AND METHODS FOR TREATMENT OF DYSTROPHIN-BASED MYOPATHIES USING CRISPR-CAS9 TO CORRECT DMD EXON DUPLICATIONS
#183RECOMBINANT ADENO-ASSOCIATED VIRUS PRODUCTS AND METHODS FOR TREATING DYSTROGLYCANOPATHIES AND LAMININ-DEFICIENT MUSCULAR DYSTROPHIES
#184CELL SPECIFIC GENE THERAPY DELIVERY COMPOSITIONS AND METHODS FOR TREATING HEARING LOSS
#185TRANSCRIPTION FACTOR
#186Viral Vectors For Treating Neurogenic Detrusor Overactivity
#187NEW PROMOTER SEQUENCE FOR GENE THERAPY
#188NON-VIRAL DNA VECTORS EXPRESSING THERAPEUTIC ANTIBODIES AND USES THEREOF
#189VECTORS ENCODING GENE EDITING SYSTEMS AND USES THEREOF
#190VESTIBULAR SUPPORTING CELL PROMOTERS AND USES THEREOF
#191MICRODYSTROPHIN GENE THERAPY ADMINISTRATION FOR TREATMENT OF DYSTROPHINOPATHIES
#192GM3 SYNTHASE VECTORS AND USES THEREOF
#193COMPOSITIONS AND METHODS FOR TREATING SENSORINEURAL HEARING LOSS USING STEREOCILIN DUAL VECTOR SYSTEMS
#194SYNTHETIC AAV VECTORS FOR REPEATED DELIVERY OF THERAPEUTIC GENES
#195HIGH-THROUGHPUT EXPRESSION-LINKED PROMOTER SELECTION IN EUKARYOTIC CELLS
#196EXPRESSION VECTOR FOR HUMAN NUCLEAR FACTOR ERYTHROID 2-RELATED FACTOR 2 (NRF2) AND APPLICATION THEREOF
#197METHODS OF REDOSING GENE THERAPY VECTORS
#198ARTIFICIAL REGULATORY CASSETTES FOR MUSCLE-SPECIFIC GENE EXPRESSION
#199ADENO-ASSOCIATED VIRAL VECTORS FOR TRANSDUCTION OF COCHLEA
#200GENE THERAPY
#201Gene Therapy for Retinal Disease
#202Methods of Treating Muscular Dystrophy
#203INSULIN GENE THERAPY TO TREAT DIABETES
#204ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN CLAUSTRUM NEURONS
#205ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF B-SARCOGLYCAN AND MICRORNA-29 AND THE TREATMENT OF MUSCULAR DYSTROPHY
#206METHODS AND MATERIALS FOR TREATING PROPIONIC ACIDEMIA
#207CELL SPECIFIC GENE THERAPY DELIVERY COMPOSITIONS AND METHODS FOR TREATING HEARING LOSS
#208METHODS AND COMPOSITIONS FOR REJUVENATING CNS GLIAL POPULATIONS WITH BCL11A TRANSCRIPTION FACTOR EXPRESSION
#209Noninvasive AAV Vectors For Highly Efficient Gene Delivery To The Nervous System
#210High-transducing HSV vectors
#211GENE THERAPY FOR DENT DISEASE
#212SYNTHETIC NUCLEIC ACIDS INCLUDING ASTROCYTE-DIRECTED PROMOTER CONSTRUCTS AND METHODS OF USING THE SAME
#213PROMOTER FOR SPECIFIC EXPRESSION OF GENES IN ROD PHOTORECEPTORS
#214CONFERRING DROUGHT TOLERANCE AND BIOMASS ACCUMULATION THROUGH THE PLANT-SPECIFIC RFS GENE FAMILY
#215LIVER SPECIFIC PRODUCTION OF ENPP1 OR ENPP3
#216METHODS AND COMPOSITIONS TO CONFER REGULATION TO GENE THERAPY CARGOES BY HETEROLOGOUS USE OF ALTERNATIVE SPLICING CASSETTES
#217TARGETED GENE INSERTION FOR IMPROVED IMMUNE CELLS THERAPY
#218COMPOSITIONS AND METHODS FOR TREATING CLRN1-ASSOCIATED HEARING LOSS AND/OR VISION LOSS
#219Recombinant constructs and transgenic fluorescent ornamental fish therefrom
#220Viral Vectors For Treating Neurogenic Detrusor Overactivity
#221ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN GABAERGIC NEURONS AND ASTROCYTES
#222GENE THERAPY FOR RETINAL DISEASES
#223RAPID HIGH THROUGHPUT SCREENING SYSTEM TO ASSESS POTENTIAL TREATMENTS FOR SPORADIC ALZHEIMER’S DISEASE
#224TREATMENT OF CONGENITAL STATIONARY NIGHT BLINDNESS USING GENE THERAPY
#225FUNCTIONAL NUCLEIC ACID MOLECULE AND METHOD
#226METHOD OF DESIGNING HIGHLY REGULATED LENTIVIRAL VECTORS POSSESING STRICT ENDOGENOUS REGULATION
#227COMPOSITIONS FOR AND METHODS OF IMPROVING FLUID FLUX IN THE BRAIN
#228GLOBIN GENE THERAPY FOR TREATING HEMOGLOBINOPATHIES
#229POLYNUCLEOTIDE FOR PHYSIOLOGICAL EXPRESSION IN T-CELLS
#230COMPOSITIONS AND METHODS FOR TREATING BETA-HEMOGLOBINOPATHIES
#231Optimized expression cassettes for gene therapy
#232ADENO-ASSOCIATED VIRUS (AAV) PRODUCER CELL LINE AND RELATED METHODS
#233ARTIFICIAL EXPRESSION CONSTRUCTS FOR MODULATING GENE EXPRESSION IN CHANDELIER CELLS
#234Gene Expression System
#235Compositions and methods for treating non-age-associated hearing impairment in a human subject
#236CRISPR-MEDIATED HUMAN GENOME EDITING WITH VECTORS
#237TREATMENT OF HYPERBILIRUBINEMIA
#238Compositions and methods for treating non-age-associated hearing impairment in a human subject
#239Optimized mini-dystrophin genes and expression cassettes and their use
#240BIDIRECTIONAL DUAL PROMOTER EXPRESSION VECTORS AND USES THEREOF
#241THERAPEUTIC ADENO-ASSOCIATED VIRUS DELIVERY OF FUKUTIN RELATED PROTEIN (FKRP) FOR TREATING DYSTROGLYCANOPATHY. DISORDERS INCLUDING LIMB GIRDLE 21 (LGMD21)
#242METHODS FOR TREATING NEUROLOGICAL DISEASE
#243CHIMERIC ANTIGEN RECEPTOR EXPRESSION SYSTEMS
#244GENE EDITING OF SATELLITE CELLS IN VIVO USING AAV VECTORS ENCODING MUSCLE-SPECIFIC PROMOTERS
#245MUSCLE-SPECIFIC HYBRID PROMOTER
#246Methods and compositions for transducing lymphocytes and regulating the activity thereof
#247METHODS OF TREATING CARDIAC DISORDERS AND CONGESTIVE HEART FAILURE AND ADMINISTERING AAV VECTORS
#248CONSTRUCTS, COMPOSITIONS, CELLS AND METHODS FOR INCREASED RECOMBINANT PROTEIN EXPRESSION BY TARGETED INTEGRATION AND AMPLIFICATION
#249RECOMBINANT ADENO ASSOCIATED VIRUS (RAAV) ENCODING GJB2 AND USES THEREOF
#250REGULATORY NUCLEIC ACID SEQUENCES
#251AAV VECTOR DELIVERY SYSTEMS
#252COMPOSITIONS AND METHODS FOR TREATING OR PREVENTING HEREDITARY ANGIOEDEMA
#253MODIFIED MGLUR6 PROMOTER AND METHODS OF USE
#254ISOLATED NUCLEIC ACID MOLECULE AND APPLICATION THEREOF
#255ADENO-ASSOCIATED VIRUS VECTORS FOR TREATMENT OF RETT SYNDROME
#256GENE THERAPEUTICS FOR FIBRODYSPLASIA OSSIFICANS PROGRESSIVA
#257GENE THERAPY FOR TREATING CITRULLENEMIA
#258GENE THERAPY USING NUCLEIC ACID CONSTRUCTS COMPRISING METHYL CPG BINDING PROTEIN 2 (MECP2) PROMOTER SEQUENCES
#259Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
#260METHOD FOR THE TREATMENT OF WWOX ASSOCIATED DISEASES
#261Methods for using transcription-dependent directed evolution of AAV capsids
#262Methods for using transcription-dependent directed evolution of AAV capsids
#263DNA-BINDING DOMAIN TRANSACTIVATORS AND USES THEREOF
#264MOTOR NEURON-SPECIFIC EXPRESSION VECTORS
#265TGFß THERAPY FOR OCULAR AND NEURODEGENERATIVE DISEASES
#266USE OF CIRCUTRN IN PREPARATION OF DRUG FOR TREATING HEART FAILURE, RECOMBINANT VECTOR, AND DRUG FOR TREATING HEART FAILURE
#267LDLR VARIANTS AND THEIR USE IN COMPOSITIONS FOR REDUCING CHOLESTEROL LEVELS
#268AAV VECTORS TARGETED TO THE CENTRAL NERVOUS SYSTEM
#269MINI-PROMOTER
#270MUSCLE-SPECIFIC NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USE THEREOF
#271GENE-THERAPY VECTORS FOR TREATING CARDIOMYOPATHY
#272ONCOLYTIC ADENOVIRUS WITH REPLICATION SELECTIVITY BASED ON STATUS OF P53 TRANSCRIPTIONAL ACTIVITY
#273TISSUE SELECTIVE TRANSGENE EXPRESSION
#274METHODS FOR USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
#275NUCLEIC ACID-BASED COMPOSITIONS AND METHODS FOR TREATING SMALL VESSEL DISEASES
#276AAV Vector for Disrupting Coagulation Factor-Related Gene on Liver Genome
#277COMPOSITIONS AND METHODS FOR TREATING GJB2-ASSOCIATED HEARING LOSS
#278SELF-LIMITING VIRAL VECTORS ENCODING NUCLEASES
#279VECTOR
#280METHODS AND COMPOSITIONS FOR TREATING DISEASES AND DISORDERS OF THE NERVOUS SYSTEM
#281ADENO-ASSOCIATED VIRUS VECTOR DELIVERY OF MICRO-DYSTROPHIN TO TREAT MUSCULAR DYSTROPHY
#282REDEFINING LIVESTOCK MILK BY REMOVING FOLATE RECEPTOR PROTEIN TO DECREASE FOLATE RECEPTOR AUTOIMMUNE DISORDER IN PREGNANCY AND AUTISM
#283CIRCUIT BOARD
#284ASSAY FOR DRUG DISCOVERY BASED ON IN VITRO DIFFERENTIATED CELLS
#285ARTIFICIAL EXPRESSION CONSTRUCTS FOR SELECTIVELY MODULATING GENE EXPRESSION IN NEOCORTICAL LAYER 5 GLUTAMATERGIC NEURONS
#286ENGINEERED MUSCLE TARGETING COMPOSITIONS
#287COMPOSITIONS AND METHODS FOR TREATING MACULAR DYSTROPHY
#288Transdermal Optogenetic Peripheral Nerve Stimulation
#289REGULATORY NUCLEIC ACID SEQUENCES
#290AAV NATIVE-NEURO PLATFORM AND USE FOR NEURONAL DISEASE GENE THERAPY
#291Promoter Sequences for In Vitro and In Vivo Expression of Gene Therapy Products in CD3+ Cells
#292VIRAL VECTORS FOR TREATING NEUROGENIC DETRUSOR OVERACTIVITY
#293OPTIMIZED EXPRESSION CASSETTES FOR GENE THERAPY
#294TRANSCRIPTION MODULATION IN ANIMALS USING CRISPR/CAS SYSTEMS
#295METHODS FOR USING TRANSCRIPTION-DEPENDENT DIRECTED EVOLUTION OF AAV CAPSIDS
#296KIR 7.1 GENE THERAPY VECTORS AND METHODS OF USING THE SAME
#297NOVEL THERAPEUTICAL TOOLS AND METHODS USING TEMPERATURE-SENSITIVE RECEPTORS FOR TREATING BLINDNESS
#298Compositions and methods for treating non-age-associated hearing impairment in a human subject
#299NOVEL COMBINATION OF NUCLEIC ACID REGULATORY ELEMENTS AND METHODS AND USES THEREOF
#300REPROGRAMMING THE METABOLOME TO DELAY ONSET OR TREAT NEURODEGENERATION