21401 ⎘
Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
Sub-classes:GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#2ENGINEERED MESENCHYMAL STEM CELL AND USE THEREOF
#3LIPID NANOPARTICLE FORMULATION COMPRISING IONIZED LIPIDS WITH BRANCHED STRUCTURE, AND USE THEREOF
#4MODIFIED LIGAND-GATED ION CHANNELS AND METHODS OF USE
#5GENE EDITING-BASED METHOD OF ATTENUATING THE BETA-AMYLOID PATHWAY
#6BISPHOSPHONATE LIPIDS, LIPID NANOPARTICLE COMPOSITIONS COMPRISING THE SAME, MINERAL TISSUE-ADSORBED COMPOSITIONS THEREOF, AND METHODS OF USE THEREOF
#7METHODS AND SYSTEMS FOR IMPROVED NUCLEIC ACID DELIVERY VIA ULTRASOUND
#8COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE
#9ARTIFICIAL NUCLEIC ACID MOLECULES FOR IMPROVED PROTEIN OR PEPTIDE EXPRESSION
#10IMPROVED IGG-DEGRADING ENZYMES AND METHODS OF USE THEREOF
#11MEANS AND METHODS FOR AAV GENE THERAPY IN HUMANS
#12SITE-SPECIFIC INTEGRATING RECOMBINANT AAV VECTORS FOR GENE THERAPY AND IMPROVED PRODUCTION METHODS
#13COMPOSITIONS AND METHODS INVOLVING ADGRG6
#14COMPOSITIONS AND METHODS FOR NUCLEIC ACID TRANSFER
#15SYSTEMS, METHODS, AND COMPOSITIONS FOR ALTERING THE EXPRESSION OF ENDOGENOUS CIRCULAR RNAS
#16MICROGLIA HAVING CAR AND USE THEREOF
#17COMPOSITIONS AND METHODS FOR TREATING METABOLIC DYSFUNCTION-ASSOCIATED STEATOTIC LIVER DISEASE
#18GENE THERAPY
#19RNA RECOGNITION COMPLEX AND USES THEREOF
#20Gene Therapy For Neurodegenerative Disorders
#21USE OF p55-GAMMA GENE AND/OR p55-GAMMA PROTEIN AS TARGET IN MAINTAINING CARDIAC IRON HOMEOSTASIS AND TREATING RELATED DISEASE
#22GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS
#23ICE-BASED LIPID NANOPARTICLE FORMULATIONS FOR DELIVERY OF MRNA
#24Materials and Methods for Treatment of Hemoglobinopathies
#25SYSTEMS AND METHODS FOR CHARACTERIZING PATHOPHYSIOLOGY
#26COMPOSITIONS AND METHODS OF THE DELIVERY OF ACTIVE AGENTS INCLUDING NUCLEIC ACIDS
#27NOVEL IONIZABLE LIPID AND LIPID NANOPARTICLE COMPOSITION USING SAME
#28Targeted CRISPR Delivery Platforms
#29Intrathecal Delivery of Recombinant Adeno-Associated Virus 9
#30Targeted CRISPR Delivery Platforms
#31Acid Degradable Solid Lipid Nanoparticles
#32COMPOSITION FOR USE IN THE TREATMENT OF FABRY DISEASE
#33ADENO-ASSOCIATED VIRUS VECTOR VARIANTS FOR HIGH EFFICIENCY GENOME EDITING AND METHODS THEREOF
#34Lipid Nanoparticle Compositions and Methods for mRNA Delivery
#35METHODS FOR IN VIVO DELIVERY AND DETECTION OF CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEAT SYSTEMS UTILIZING BACULOVIRUS VECTOR-MAGNETIC NANOPARTICLE COMPLEXES
#36CAPSID
#37PATCH FOR TARGETED DELIVERY OF AN ONCOGENIC CARGO TO A TISSUE
#38INTRASPINAL DELIVERY OF THERAPEUTIC AGENTS
#39SITE-SPECIFIC INTEGRATING RECOMBINANT AAV VECTORS FOR GENE THERAPY AND IMPROVED PRODUCTION METHODS
#40THERAPEUTIC APPLICATIONS OF CPF1-BASED GENOME EDITING
#41Lipid Nanoparticle Compositions and Methods for mRNA Delivery
#42METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES
#43ADENO-ASSOCIATED VIRUS WITH ENGINEERED CAPSID
#44GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS
#45DOSING REGIMEN FOR IL-10 ENCODING EXPRESSION CONSTRUCT
#46LIPIDS AND USES THEREOF
#47LIPID NANOPARTICLE COMPOSITIONS AND USES THEREOF
#48COMPOSITIONS AND METHODS FOR DELIVERING MOLECULES
#49NUCLEIC ACID MOLECULE FOR TREATING BIETTI CRYSTALLINE DYSTROPHY AND USE THEREOF
#50mRNA THERAPY WITH REDUCED IMMUNE REACTOGENICITY
#51COMPOSITIONS AND METHODS FOR THE TREATMENT OF AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE AND OTHER DISEASES HAVING UPREGULATED MTOR ACTIVITY
#52METHODS AND COMPOSITIONS FOR TREATING DNMT3A DEFICIENCY-ASSOCIATED DISEASES
#53GENE THERAPY
#54Gene Therapy For Neurodegenerative Disorders
#55Liver Specific Delivery of Messenger RNA
#56ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND USE FOR INHIBITING ANGIOGENESIS
#57GUIDE RNAS FOR CRISPR/CAS EDITING SYSTEMS
#58MODIFIED AAV CAPSIDS AND USES THEREOF
#59IMMUNOGENIC COMPOSITION
#60LIPID NANOPARTICLE COMPOSITIONS AND USES THEREOF
#61POLYOL-MODIFIED LIPID COMPOUND AND PREPARATION METHOD AND APPLICATION THEREOF
#62LIPID NANOMATERIALS AND USES THEREOF
#63SYNTHETIC DNA VECTORS AND METHODS OF USE
#64Materials and Methods for Treatment of Hemoglobinopathies
#65COMPOSITIONS AND METHODS FOR TREATING NGYL1 DEFICIENCY
#66VESTIBULAR SUPPORTING CELL PROMOTERS AND USES THEREOF
#67NANOLIPOPROTEIN PARTICLES AND RELATED COMPOSITIONS METHODS AND SYSTEMS FOR LOADING RNA
#68REGULATION OF GENE EXPRESSION THROUGH APTAMER-MODULATED POLYADENYLATION
#69METHODS OF DELIVERING ANIONIC AGENTS IN VIVO USING NON-VIRAL NANOPARTICLE-BASED DELIVERY SYSTEMS
#70HYPERACTIVE PIGGYBAC TRANSPOSASES
#71METHOD TO ENHANCE THE EFFICIENCY OF SYSTEMIC AAV GENE DELIVERY TO THE CENTRAL NERVOUS SYSTEM
#72ADENO-ASSOCIATED VIRUS VECTOR
#73LIVER SPECIFIC DELIVERY OF MESSENGER RNA
#74HYBRID AAV-ANELLOVECTORS
#75NOVEL CARBONYL LIPIDS AND LIPID NANOPARTICLE FORMULATIONS FOR DELIVERY OF NUCLEIC ACIDS
#76SYNTHETIC IMMUNOMODULATION WITH A CRISPR SUPER-REPRESSOR IN VIVO
#77COMPOSITIONS AND METHODS FOR USING COMBINATIONS OF ACTIN-BASED PEPTIDES TO MODULATE CELLULAR BIOACTIVITY
#78PHARMACEUTICAL COMPOSITION FOR TREATING NON-ALCOHOLIC FATTY LIVER, NON-ALCOHOLIC STEATOHEPATITIS, OR HEPATIC FIBROSIS USING SSU72 PROTEIN OR A POLYNUCLEOTIDE ENCODING THE SAME
#79Adeno-Associated Virus Factor VIII Vectors, Associated Viral Particles and Therapeutic Formulations Comprising the Same
#80Gene Therapy For Neurodegenerative Disorders
#81COMPOSITIONS FOR FACILITATING MEMBRANE FUSION AND USES THEREOF
#82POLYNUCLEOTIDES ENCODING INTERLEUKIN-12 (IL12) AND USES THEREOF
#83GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#84Mesothelioma Gene Therapy
#85MOLONEY MURINE LEUKEMIA VIRUS-BASED SELF-INACTIVATING VECTOR AND APPLICATIONS THEREOF
#86COMPOSITIONS AND USES THEREOF FOR TREATMENT OF ANGELMAN SYNDROME
#87GENE THERAPIES FOR NEURODEGENERATIVE DISEASE
#88ENGINEERED AAV VECTORS
#89Lipid nanoparticle compositions and methods for mRNA delivery
#90Compositions and Methods of Treatment
#91METHODS FOR MODULATING LEVEL OF EXPRESSION FROM GENE THERAPY EXPRESSION CASSETTE
#92ARC-BASED CAPSIDS AND USES THEREOF
#93LIPIDIC COMPOUNDS COMPRISING AT LEAST ONE TERMINAL RADICAL OF FORMULA -NH-CX-A OR -NH-CX-NH-A, COMPOSITIONS CONTAINING THEM AND USES THEREOF
#94GENE EDITING-BASED METHOD OF ATTENUATING THE BETA-AMYLOID PATHWAY
#95LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
#96Targeted DNA demethylation method, fusion protein and application thereof preliminary class
#97Lipid nanoparticle compositions and methods for MRNA delivery
#98Lipid nanoparticle compositions and methods for MRNA delivery
#99NIPAH VIRUS ENVELOPE PSEUDOTYPED LENTIVIRUSES AND METHODS OF THEIR USE
#100COMPOSITIONS AND METHODS FOR ENHANCING DONOR OLIGONUCLEOTIDE-BASED GENE EDITING
#101GENE THERAPY EXPRESSION SYSTEM ALLEVIATING CARDIAC TOXICITY OF FKRP
#102PLURIPOTENT STEM CELL AND DERIVATIVE THEREOF
#103COMPOSITIONS FOR TREATING FRIEDREICH’S ATAXIA
#104Immunomodulating Gene Therapy
#105EXOSOMES COMPRISING THERAPEUTIC POLYPEPTIDES
#106Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#107GENE THERAPY OF NIEMANN-PICK DISEASE TYPE C
#108COMPOSITION AND METHODS FOR TREATMENT OF PRIMARY CILIARY DYSKINESIA
#109GENOMIC INSULATOR ELEMENT EXHIBITING ENHANCER BLOCKING ACTIVITIES IN LYMPHOCYTES AND USES THEREOF
#110AMINE-CONTAINING TRANSFECTION REAGENTS AND METHODS FOR MAKING AND USING SAME
#111COMPOSITIONS USEFUL FOR TREATMENT OF POMPE DISEASE
#112COMPOSITIONS AND METHODS FOR DELIVERING MESSENGER RNA
#113MODULATING OPSIN SIGNALING LIFETIME FOR OPTOGENETIC APPLICATIONS
#114METHODS AND COMPOSITIONS FOR ATTENUATING ANTI-VIRAL TRANSFER VECTOR IGM RESPONSES
#115Gene therapy for neurodegenerative disorders
#116NOVEL USE OF ASPIRIN COMPOUND IN INCREASING NUCLEIC ACID EXPRESSION
#117Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
#118PSEUDOTYPED RECOMBINANT LYSSAVIRUSES FOR GENE THERAPY
#119Tricine and Citric Acid Lipids
#120Gene therapy for neurodegenerative disorders
#121METHODS AND COMPOSITIONS FOR DELIVERY OF AGENTS ACROSS THE BLOOD-BRAIN BARRIER
#122ADENO-ASSOCIATED VIRUS FORMULATIONS
#123ARC-BASED CAPSIDS AND USES THEREOF
#124Methods for Treating Parkinson's Disease
#125COMPOSITIONS AND METHODS FOR RESTORING AND MAINTAINING THE DYSTROPHIN-ASSOCIATED PROTEIN COMPLEX (DAPC)
#126Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
#127SYSTEMS AND METHODS FOR LIPID NANOPARTICLE DELIVERY OF GENE EDITING MACHINERY
#128Pharmaceutical composition for treating non-alcoholic fatty liver, non-alcoholic steatohepatitis, or hepatic fibrosis using Ssu72 protein or a polynucleotide encoding the same
#129Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
#130Zinc Finger Protein Transcription Factors for Treatment of Prion Disease
#131FORMULATION FOR DELIVERY OF LUBRICIN GENE
#132METHOD OF TRANSACTIVATING A HOMOLOGOUS GENE OF A GENE OF INTEREST AND AN IN VITRO METHOD OF DIAGNOSING A DISEASE
#133USE OF SUMF2 GENE AS A GENE THERAPY TARGET FOR PREVENTING AND/OR TREATING ALLERGIC ASTHMA ATTACK AND REDUCING AIRWAY HYPERRESPONSIVENESS
#134Means and methods for AAV gene therapy in humans
#135Targeted CRISPR Delivery Platforms
#136Compositions and methods of treatment
#137METHODS OF MODULATING HAIR FOLLICLE STEM CELL QUIESCENCE BY MODULATING DERMAL NICHE ACTIVATOR GAS6
#138Lipid nanoparticle compositions and methods for mRNA delivery
#139GENE THERAPY EXPRESSION SYSTEM ALLEVIATING CARDIAC TOXICITY OF FKRP
#140LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
#141METHODS AND COMPOSITIONS FOR INCREASING TRANSDUCTION EFFICIENCY WITH CELL MEMBRANE FUSION PROTEINS
#142MODULATION OF UBIQUITIN CARBOXY-TERMINAL HYDROLASE LIGASE 1 (UCHL1) EXPRESSION FOR TREATING NEUROLOGICAL DISEASE, DISORDERS, AND INJURIES ASSOCIATED WITH UPPER MOTOR NEURONS
#143COMPOSITIONS AND METHODS FOR TREATING NERVOUS SYSTEM INJURIES
#144PHARMACEUTICAL COMPOSITION COMPRISING VACCINIA VIRUS AND HYDROXYUREA AS ACTIVE INGREDIENT FOR TREATMENT OF CANCER
#145COMPOSITIONS AND METHODS FOR ENHANCED DELIVERY OF AGENTS
#146MODIFIED ADENO-ASSOCIATED VIRUS (AAV) PARTICLES FOR GENE THERAPY
#147GENE THERAPY FOR TREATING HEMOPHILIA B
#148METHODS AND COMPOSITIONS FOR REDUCING GENE OR NUCLEIC ACID THERAPY-RELATED IMMUNE RESPONSES
#149COMPOSITIONS AND METHODS FOR TREATING ALPHA THALASSEMIA
#150Methods And Compositions For Enhancing AAV-Mediated Homologous Recombination Using Ribonucleotide Reductase Inhibitors
#151COMPOSITIONS AND METHODS FOR TREATING CEP290-ASSOCIATED DISEASE
#152LIPID COMPOSITION
#153Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#154COMPOSITIONS AND METHODS FOR mRNA DELIVERY
#155Adeno-Associated Virus Virions with Variant Capsid and Methods of Use Thereof
#156Modified AAV capsids and uses thereof
#157Composition for treatment of Crigler-Najjar syndrome
#158Methods for efficient delivery of therapeutic molecules in vitro and in vivo
#159METHODS AND COMPOSITIONS COMPRISING ENHANCED TARGETED IMMUNE GENE THERAPY FOR THE TREATMENT OF CANCER
#160VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A
#161MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
#162REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
#163USE OF Dvl3-DEP PEPTIDE IN PREPARATION OF MEDICAMENT FOR REPAIRING SERTOLI CELL INJURY IN TESTES
#164FOCUSED ULTRASOUND FOR NON-INVASIVE FOCAL GENE DELIVERY TO THE MAMMALIAN BRAIN
#165GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#166Anti-miRNA carrier conjugated with a peptide binding to a cancer cell surface protein and use thereof
#167GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#168COMPOSITIONS AND METHODS FOR TREATING CANCER
#169Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
#170Methods and Compositions for Treatment of Disorders and Diseases Involving RDH12
#171LIVER SPECIFIC DELIVERY OF MESSENGER RNA
#172LIVER SPECIFIC DELIVERY OF MESSENGER RNA
#173LIVER SPECIFIC DELIVERY OF MESSENGER RNA
#174Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes
#175Lipid nanoparticle compositions and methods for mRNA delivery
#176LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
#177Lipid nanoparticle compositions and methods for MRNA delivery
#178ADENO-ASSOCIATED VIRUS VECTOR
#179Lipid nanoparticle compositions and methods for mRNA delivery
#180Methods and compositions for delivery of agents across the blood-brain barrier
#181ARC-BASED CAPSIDS AND USES THEREOF
#182Drug Delivery Particle and Method for Producing the Same
#183Lipid nanoparticle compositions and methods for mRNA delivery
#184Gene editing-based method of attenuating the beta-amyloid pathway
#185Endogenous Gag-based capsids and uses thereof
#186IMMUNOMODULATORY ONCOLYTIC ADENOVIRAL VECTORS, AND METHODS OF PRODUCTION AND USE THEREOF FOR TREATMENT OF CANCER
#187GENE THERAPY FOR NEURONAL CEROID LIPOFUSCINOSES
#188INHALATION POWDER MEDICINE, EVALUATION METHOD THEREOF, AND USE THEREOF
#189Synthetic immunomodulation with a CRISPR super-repressor in vivo
#190Lipid nanoparticle formulations for delivery of MRNA
#191Endogenous Gag-based capsids and uses thereof
#192Lipid Vesicle for Oral Drug Delivery
#193COMPOSITIONS AND METHODS FOR ENHANCING DONOR OLIGONUCLEOTIDE-BASED GENE EDITING
#194USE OF LIPOSOMES IN A CARRIER COMPRISING A CONTINUOUS HYDROPHOBIC PHASE FOR DELIVERY OF POLYNUCLEOTIDES IN VIVO
#195METHODS AND COMPOSITIONS FOR TREATMENT OF INTERFERON-RESISTANT TUMORS
#196RECOMBINANT AAV VECTORS USEFUL FOR REDUCING IMMUNITY AGAINST TRANSGENE PRODUCTS
#197Vector for gene silencing and replacement and methods of use thereof
#198INJECTION SOLUTION FOR RNA
#199RETROVIRAL PARTICLE COMPRISING AT LEAST TWO ENCAPSIDATED NONVIRAL RNAS
#200Lipid nanoparticle compositions and methods for mRNA delivery
#201Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#202ENGINEERED RECEPTOR/LIGAND SYSTEM FOR DELIVERY OF THERAPEUTIC AGENTS
#203RNA-GUIDED NUCLEIC ACID MODIFYING ENZYMES AND METHODS OF USE THEREOF
#204Genome editing without nucleases
#205METHODS AND COMPOSITIONS FOR DELIVERY OF AGENTS ACROSS THE BLOOD-BRAIN BARRIER
#206MODIFIED SEROTYPE 28 ADENOVIRAL VECTORS
#207CAPSID-MODIFIED, RAAV3 VECTOR COMPOSITIONS AND METHODS OF USE IN GENE THERAPY OF HUMAN LIVER CANCER
#208COMPLEX FOR THE DELIVERY OF CAS9 PROTEINS AND GUIDE RNA TO CELLS
#209Adeno-associated virus vector
#210Intrathecal delivery of recombinant adeno-associated virus 9
#211FUSOSOME COMPOSITIONS AND USES THEREOF
#212INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS 9
#213Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
#214Modified ligand-gated ion channels and methods of use
#215Isolation of novel AAV's and uses thereof
#216Programmable assembly of virus composites for receptor-targeted gene delivery
#217Compositions for transfecting resistant cell types
#218Cell-based therapy for the pulmonary system
#219Method for improving retroviral transduction and gene editing in hematopoietic stem cells using cyclosporine h (CsH)
#220Methods of delivering anionic agents in vivo using non-viral nanoparticle-based delivery systems
#221COMPOSITIONS AND METHODS FOR NUCLEIC ACID TRANSFER
#222Intrathecal Delivery of Recombinant Adeno-Associated Virus 9
#223METHOD AND COMPOSITION FOR ENDOGENOUS PRODUCTION OF CONSTITUTIVELY ACTIVATED RECEPTORS, AND RECEPTORS WITH BROADER BINDING RANGES OR HIGHER AFFINITY THAN NATIVE RECEPTORS
#224Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#225AAV vectors encoding clarin-1 or GJB2 and uses thereof
#226Lipid nanoparticle compositions and methods for mRNA delivery
#227Dry Transfection Compositions and Methods for Making and Using the Same
#228Conjugates of Guide RNA-Cas Protein Complex
#229CAPSID-MODIFIED RAAV VECTORS AND METHODS OF USE
#230Genome Editing without Nucleases
#231SYNTHETIC DNA VECTORS AND METHODS OF USE
#232GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
#233Multi-armed polyrotaxane platform for protected nucleic acid delivery
#234Polynucleotides encoding interleukin-12 (IL12) and uses thereof
#235Compounds and compositions for targeting brain injuries and methods of use thereof
#236TETRAVALENT TLR9 BISPECIFIC ANTIBODY
#237METHODS AND COMPOSITIONS FOR INHIBITION OF INNATE IMMUNE RESPONSE ASSOCIATED WITH AAV TRANSDUCTION
#238Lipid nanoparticle compositions and methods for mRNA delivery
#239Variant AAV capsids for intravitreal delivery
#240ARTIFICIAL NUCLEIC ACID MOLECULES FOR IMPROVED PROTEIN OR PEPTIDE EXPRESSION
#241METHODOLOGY FOR IDENTIFYING GENE DELIVERY VECTORS WITH RETINAL CELL SPECIFICITY IN NON-HUMAN PRIMATE
#242MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
#243Echovirus for treatment of tumors
#244AAV CAPSID DESIGNS
#245COMPOSITIONS AND METHODS FOR DELIVERING MESSENGER RNA
#246Adeno-associated virus variant capsids and use for inhibiting angiogenesis
#247METHOD OF ENHANCING DELIVERY OF THERAPEUTIC COMPOUNDS TO THE EYE
#248ADENO-ASSOCIATED VIRUS VIRIONS WITH VARIANT CAPSID AND METHODS OF USE THEREOF
#249ISOLATION OF EXTRACELLULAR VESICLES (EVS) FROM RED BLOOD CELLS FOR GENE THERAPY
#250Compositions comprising AAV expressing dual antibody constructs and uses thereof
#251EXTRACELLULAR VESICLES FOR AGENT DELIVERY
#252Collagen-mimetic peptide mediated delivery of nucleic acid carriers for efficient delivery from collagen
#253Mesothelioma Gene Therapy
#254Ice-based lipid nanoparticle formulation for delivery of mRNA
#255ENGINEERING SYNTHETIC BRAIN PENETRATING GENE VECTORS
#256Hyperactive piggybac transposases
#257LIPID NANOPARTICLE COMPOSITIONS AND METHODS FOR MRNA DELIVERY
#258Cationic lipid
#259Capsid
#260Amine-containing transfection reagents and methods for making and using same
#261Adeno-associated virus Factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
#262Compositions for facilitating membrane fusion and uses thereof
#263Ice-based lipid nanoparticle formulation for delivery of mRNA
#264Regulation of gene expression through aptamer-modulated polyadenylation
#265GENE EDITING OF CAR-T CELLS FOR THE TREATMENT OF T CELL MALIGNANCIES WITH CHIMERIC ANTIGEN RECEPTORS
#266Methods and Compositions for Treatment of Interferon-Resistant Tumors
#267AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#268Targeted CRISPR Delivery Platforms
#269Intrathecal delivery of recombinant adeno-associated virus 9
#270Method of making peptide-tagged PEGylated chitosan nanoparticles
#271Compositions comprising AAV expressing dual antibody constructs and uses thereof
#272Lipid nanoparticle compositions and methods for mRNA delivery
#273Method of Encapsulating a Nucleic Acid in a Lipid Nanoparticle Host
#274Endosomolytic agents for gene therapy
#275Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency
#276Isolation of novel AAV's and uses thereof
#277Nanoparticles comprising protein-polynucleotide complexes and for delivering protein based complexes
#278Intrathecal delivery of recombinant adeno-associated virus 9
#279COMPOSITIONS AND METHODS FOR THE MANUFACTURE OF LIPID NANOPARTICLES
#280Intrathecal delivery of recombinant adeno-associated virus 9
#281Enhanced Gene Delivery to Natural Killer Cells, Hematopoietic Stem Cells and Macrophages
#282Vector for gene silencing and replacement and methods of use thereof
#283Poly(cyclic imino ether)s
#284Use of liposomes in a carrier comprising a continuous hydrophobic phase for delivery of polynucleotides in vivo
#285Adeno-associated virus virions with variant capsid
#286Gene editing-based method of attenuating the beta-amyloid pathway
#287Adeno-associated virus particle with mutated capsid and methods of use thereof
#288Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#289MATERIALS AND METHODS FOR TREATMENT OF HEMOGLOBINOPATHIES
#290PULMONARY TARGETED CAS9/CRISPR FOR IN VIVO EDITING OF DISEASE GENES
#291Compositions and methods for treating CEP290-associated disease
#292GENETICALLY-MODIFIED CELLS COMPRISING A MODIFIED HUMAN T CELL RECEPTOR ALPHA CONSTANT REGION GENE
#293Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#294LIVER SPECIFIC DELIVERY OF MESSENGER RNA
#295Lipid nanoparticle compositions and methods for mRNA delivery
#296Gene therapy
#297Dependent Component Genome Editing Gene Drives
#298Lipid nanoparticle compositions and methods for mRNA delivery
#299Modified ligand-gated ion channels and methods of use
#300METHODS FOR PREVENTING OR TREATING FIBROTIC DISEASES