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
METHODS AND COMPOSITIONS FOR THE TREATMENT OF RARE DISEASES
#302Treatment And/Or Prevention Of DNA-Triplet Repeat Diseases Or Disorders
#303Compositions and methods of replication deficient adenoviral vectors for vaccine applications
#304Exosomes comprising therapeutic polypeptides
#305Means and methods to treat dystonia
#306Method of engineering drug-specific hypersensitive t-cells for immunotherapy by gene inactivation
#307Intrathecal delivery of recombinant adeno-associated virus 9
#308Therapeutic applications of CPF1-based genome editing
#309Methods and compositions for treatment of disorders and diseases involving RDH12
#310Compositions and methods for adeno-associated virus mediated gene expression in myofibroblast-like cells
#311NANOPARTICLES COMPRISING PROTEIN-POLYNUCLEOTIDE COMPLEXES AND FOR DELIVERING PROTEIN BASED COMPLEXES
#312METHODS AND COMPOSITIONS FOR ATTENUATING ANTI-VIRAL TRANSFER VECTOR IGM RESPONSES
#313Immunomodulatory Oncolytic Adenoviral Vectors, and Methods of Production and Use Thereof for Treatment of Cancer
#314INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS 9
#315Intrathecal delivery of recombinant Adeno-associated virus 9
#316Nipah virus envelope pseudotyped lentiviruses and methods of their use
#317Biodegradable amino-ester nanomaterials for nucleic acid delivery
#318INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS 9
#319Bacterially-derived, intact minicells that encompass plasmid-free functional nucleic acid for in vivo delivery to mammalian cells
#320Compositions and methods for detection and modulation of T cell mediated immune responses against viral vectors utilized for gene therapy
#321Immunogenic composition
#322Gene therapy for treating hemophilia B
#323Isolation of extracellular vesicles (EVs) from red blood cells for gene therapy
#324Method to enhance the efficiency of systemic AAV gene delivery to the central nervous system
#325Process for mixing nucleic acids with transfection reagents for delivery
#326Compositions comprising AAV expressing dual antibody constructs and uses thereof
#327METHODS TO REGULATE POLARIZATION AND ENHANCE FUNCTION OF CELLS
#328Sirna/nanoparticle formulations for treatment of middle-east respiratory syndrome coronaviral infection
#329Use of bacterial voltage gated ion channels for human therapies
#330Immunomodulating gene therapy
#331Adeno-associated virus vectors for treatment of glycogen storage disease
#332Means and methods for AAV gene therapy in humans
#333Piggyback Delivery of CRISPR/CAS9 RNA into Zebrafish Blood Cells
#334Composition for treatment of Crigler-Najjar syndrome
#335Targeted gene delivery to non-phagocytic mammalian cells via bacterially derived intact minicells
#336COMPOSITIONS AND METHODS FOR mRNA DELIVERY
#337Methods and Compositions for Enhancing Transduction Efficiency of Retroviral Vectors
#338Cell-based therapy for the pulmonary system
#339OLIG1 mini-promoters: PIe305
#340Nanolipoprotein particles and related compositions methods and systems for loading RNA
#341Personalized medicine therapeutic MiniCircle
#342Compounds and compositions for targeting brain injuries and methods of use thereof
#343Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#344Targeted delivery of drugs, therapeutic nucleic acids and functional nucleic acids to mammalian cells via intact killed bacterial cells
#345CAPSID-MODIFIED RAAV VECTOR COMPOSITIONS HAVING IMPROVED TRANSDUCTION EFFICIENCIES, AND METHODS OF USE
#346Capsid-modified, RAAV3 vector compositions and uses in gene therapy of human liver cancer
#347SITE-SPECIFIC DELIVERY OF NUCLEIC ACIDS BY COMBINING TARGETING LIGANDS WITH ENDOSOMOLYTIC COMPONENTS
#348PEPTIDE HAVING CELL MEMBRANE PENETRATING ACTIVITY
#349INJECTION SOLUTION FOR RNA
#350Adeno-associated viruses engineered for selectable tropism
#351Amine-containing transfection reagents and methods for making and using same
#352Engineering synthetic brain penetrating gene vectors
#353DELIVERY OF TARGET SPECIFIC NUCLEASES
#354Immunomodulatory oncolytic adenoviral vectors, and methods of production and use thereof for treatment of cancer
#355Method to achieve extended expression of DNA infused into liver
#356Use of phospholipid scramblase inhibitors for modulating inflammatory immune responses
#357Compositions and methods for treating cancer
#358Methods and compositions for treating genetic eye diseases
#359Hyperactive piggybac transposases
#360Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
#361ADENO-ASSOCIATED VIRUS VECTOR
#362Retroviral particle comprising at least two encapsidated nonviral RNAs
#363MicroRNA initiated DNAzyme motor operating in living cells
#364Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
#365Modified AAV capsids and uses thereof
#366Methods and compositions for treating a condition in a subject
#367Capsid
#368Modified serotype 28 adenoviral vectors
#369RECOMBINANT AAV VECTORS USEFUL FOR REDUCING IMMUNITY AGAINST TRANSGENE PRODUCTS
#370NON-VIRAL NANOPARTICLE-BASED DELIVERY SYSTEM
#371Method of Enhancing Delivery of Therapeutic Compounds to the Eye
#372Compositions and methods for nucleic acid transfer
#373EXCIPIENTS FOR USE IN ADENO-ASSOCIATED VIRUS PHARMACEUTICAL FORMULATIONS AND PHARMACEUTICAL FORMULATIONS MADE THEREWITH
#374Cationic lipid
#375PIV5-based amplifying virus-like particles
#376CHIMERIC VSV-G PROTEINS AS NUCLEIC ACID TRANSFER VEHICLES
#377Capsid modified rAAV vectors and methods of use
#378Extracellular vesicles for agent delivery
#379Delivering functional nucleic acids to mammalian cells via bacterially-derived, intact minicells
#380Engineered receptor/ligand system for delivery of therapeutic agents
#381Poly(Beta-Amino Ester)s With Additives for Drug Delivery
#382LIPID NANOPARTICLE COMPOSITIONS
#383Class of therapeutic protein based molecules
#384AAV-MEDIATED GENE THERAPY FOR NPHP5 LCA-CILIOPATHY
#385Efficient delivery of therapeutic molecules in vitro and in vivo
#386Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
#387TETRAVALENT TLR9 BISPECIFIC ANTIBODY
#388Genetically-modified cells comprising a modified human T cell receptor alpha constant region gene
#389METHOD FOR ENHANCED UPTAKE OF VIRAL VECTORS IN THE MYOCARDIUM
#390Cell-based therapy for the pulmonary system
#391AAV-Based Gene Therapy
#392REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
#393Nipah virus envelope pseudotyped lentiviruses and methods of their use
#394Bacterially derived intact minicells that encompass plasmid free DNA and methods of using the same
#395Artificial nucleic acid molecules for improved protein or peptide expression
#396Method for efficient delivery of therapeutic molecules in vitro and in vivo
#397Lipid nanoparticle compositions and methods for MRNA delivery
#398Magnetic control of gene delivery in vivo
#399Adeno-associated virus vectors for treatment of glycogen storage disease
#400Expression cassette for packaging and expression of variant factor VIII for the treatment of hemostasis disorders
#401Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#402POROUS NANOPARTICLE-SUPPORTED LIPID BILAYERS (PROTOCELLS) FOR TARGETED DELIVERY INCLUDING TRANSDERMAL DELIVERY OF CARGO AND METHODS THEREOF
#403INTRATHECAL DELIVERY OF RECOMBINANT ADENO-ASSOCIATED VIRUS 9
#404CARBOHYDRATE FUNCTIONALIZED CATANIONIC SURFACTANT VESICLES FOR DRUG DELIVERY
#405Method of encapsulating a nucleic acid in a lipid nanoparticle host
#406E1-MINUS ADENOVIRUSES AND USE THEREOF
#407Method for treating adenosine deaminase severe combined imunodeficiency
#408Drug delivery particle and method for producing the same
#409Isolation of novel AAV's and uses thereof
#410Recombinant AAV vectors useful for reducing immunity against transgene products
#411Hyperactive PiggyBac transposases
#412Isolation of novel AAV's and uses thereof
#413Particles comprising protamine and RNA in combination with endosome destabilizing agents
#414METHODS AND COMPOSITIONS OF CHONDRISOMES
#415Genome editing without nucleases
#416Engineering synthethic brain penetrating gene vectors
#417Adeno-associated virus factor VIII vectors, associated viral particles and therapeutic formulations comprising the same
#418Gene therapy for neurodegenerative disorders
#419Adeno-associated virus vector variants for high efficiency genome editing and methods thereof
#420Compositions comprising AAV expressing dual antibody constructs and uses thereof
#421Enhanced AAV-mediated gene transfer for retinal therapies
#422Adeno-associated virus virions with variant capsid and methods of use thereof
#423Use of liposomes in a carrier comprising a continuous hydrophobic phase for delivery of polynucleotides in vivo
#424Delivery methods and compositions for nuclease-mediated genome engineering
#425Adeno-associated virus virions with variant capsid and methods of use thereof
#426Adeno-associated virus virions with variant capsid and methods of use thereof
#427Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#428LIPIDATED GLYCOPROTEIN PARTICLES AND METHODS OF USE
#429Compositions and Methods for Yeast Extracellular Vesicles as Delivery Systems
#430COMPOSITIONS AND METHODS FOR PROVIDING ACTIVE TELOMERASE TO CELLS IN VIVO
#431Peptide having cell membrane penetrating activity
#432Method for improving survival after radiation exposure using mesenchymal stem cells as vehicles for extracellular superoxide dismutase delivery
#433Adeno-associated vectors for enhanced transduction and reduced immunogenicity
#434Liver specific delivery of messenger RNA
#435Cationic lipid
#436COMPOSITIONS AND METHODS FOR DELIVERING MESSENGER RNA
#437COMPOSITIONS AND METHODS FOR DELIVERING MESSENGER RNA
#438DRY TRANSFECTION COMPOSITIONS AND METHODS FOR MAKING AND USING THE SAME
#439Compositions and methods for the manufacture of lipid nanoparticles
#440Compositions and methods for treating endocrine, gastrointestinal or autoimmune disorders
#441Stable aqueous formulations of adenovirus vectors
#442Class of therapeutic protein based molecules
#443Delivering functional nucleic acids to mammalian cells via bacterially-derived, intact minicells
#444Lipidated glycosaminoglycan particles and their use in drug and gene delivery for diagnosis and therapy
#445Drug delivery system and method
#446Immunomodulating gene therapy
#447Bacterially-derived, intact minicells that encompass plasmid-free functional nucleic acid for in vivo delivery to mammalian cells
#448Methods And Compositions For Treatment Of Interferon-Resistant Tumors
#449System for delivering therapeutic agents into living cells and cells nuclei
#450Method of enhancing delivery of therapeutic compounds to the eye
#451COMPOSITIONS AND METHODS FOR mRNA DELIVERY
#452METHODS AND COMPOSITIONS FOR ENHANCING TRANSDUCTION EFFICIENCY OF RETROVIRAL VECTORS
#453Targeted gene delivery to non-phagocytic mammalian cells via bacterially derived intact minicells
#454Tetracycline-regulated gene expression in HSV-1 vectors
#455COMPOSITIONS AND METHODS FOR DETECTION AND MODULATION OF T CELL MEDIATED IMMUNE RESPONSES AGAINST VIRAL VECTORS UTILIZED FOR GENE THERAPY
#456Chimeric VSV-G proteins as nucleic acid transfer vehicles
#457EXCIPIENTS FOR USE IN ADENO-ASSOCIATED VIRUS PHARMACEUTICAL FORMULATIONS, AND PHARMACEUTICAL FORMULATIONS MADE THEREWITH
#458Adeno-associated virus virions with variant capsid and methods of use thereof
#459Genetically modified human umbilical cord perivascular cells for prophylaxis against or treatment of biological or chemical agents
#460Amine-containing transfection reagents and methods for making and using same
#461Modified serotype 28 adenoviral vectors
#462Matrix metalloproteinase cleavable protein polymers for cancer gene therapy
#463Stable aqueous formulations of adenovirus vectors
#464Cell-based therapy for the pulmonary system
#465Drug delivery product and methods
#466Class of therapeutic protein based molecules
#467Delivering functional nucleic acids to mammalian cells via bacterially-derived, intact minicells
#468Functionalized nanodiamonds as delivery platforms for nucleic acids
#469Covalently functionalized nanodiamond-based MALDI matrices and methods of use thereof
#470Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
#471MRNA THERAPY FOR UREA CYCLE DISORDERS
#472Pulmonary delivery of mRNA
#473MRNA therapy for Fabry disease
#474Compositions for delivery of cargo such as drugs proteins and/or genetic materials
#475Site specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#476Adeno-associated virus virions with variant capsid and methods of use thereof
#477Bacterially-derived, intact minicells that encompass plasmid-free functional nucleic acid for in vivo delivery to mammalian cells
#478Catalytic delivery nanosubstrates (CDNS) for highly efficient delivery of biomolecules
#479Uniform field magnetization and targeting of therapeutic formulations
#480Targeted delivery of drugs, therapeutic nucleic acids and functional nucleic acids to mammalian cells via intact killed bacterial cells
#481Compositions and methods for treating endocrine, gastrointestinal or autoimmune disorders
#482Vector for pulmonary delivery, inducing agent, and uses
#483COMPOSITIONS AND METHODS FOR DELIVERING NUCLEIC ACID TO A CELL
#484Capsid-modified rAAV vector compositions having improved transduction efficiencies, and methods of use
#485AAV8 vector with enhanced functional activity and methods of use thereof
#486PEGYLATED POLYPLEXES FOR POLYNUCLEOTIDE DELIVERY
#487NON-VIRAL NANOPARTICLE-BASED DELIVERY SYSTEM
#488Delivery of a gene therapy vector to the brain by convection-enhanced delivery
#489Sustained release drug delivery systems comprising a water soluble therapeutic agent and a release modifier
#490Polynucleotide constructs, pharmaceutical compositions and methods for targeted downregulations of angiogenesis and anticancer therapy
#491Methods and compositions for poxvirus A35R protein
#492Targeted integration and expression on exogenous nucleic acid sequences
#493ENVIRONMENTALLY-RESPONSIVE NANOCOMPOSITES AND METHODS OF THEIR USE
#494Liver specific delivery of messenger RNA
#495Methods of increasing efficiency of vector penetration of target tissue
#496CELL-MEDIATED GENE THERAPY FOR CANCER USING MESENCHYMAL STEM CELLS EXPRESSING A SUICIDE GENE
#497Hyperactive piggybac transposases
#498Uniform field magnetization and targeting of therapeutic formulations
#499System for delivering therapeutic agents into living cells and cells nuclei
#500Peptide conjugated, inosine-substituted antisense oligomer compound and method
#501Methods and compositions for gene therapy and GHRH therapy
#502Uniform field magnetization and targeting of therapeutic formulations
#503Sustained release drug delivery systems comprising a water soluble therapeutic agent and a release modifier
#504Uniform Field Magnetization and Targeting of Therapeutic Formulations
#505Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
#506Particle-containing complex porous materials
#507Methods and Compositions for Poxvirus A35R Protein
#508Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
#509INTACT MINICELLS AS VECTORS FOR DNA TRANSFER AND GENE THERAPY INVITRO AND INVIVO
#510system and uses thereof
#511Adeno-associated virus virions with variant capsid and methods of use thereof
#512Drug delivery nanocarriers targeted by landscape phage
#513Amine-Containing Transfection Reagents and methods for making and using same
#514COMPOSITIONS AND METHODS FOR TREATING PLASMA PROTEIN DEFICIENCY DISORDERS
#515CARBON NANOTUBES COMPLEXED WITH MULTIPLE BIOACTIVE AGENTS AND METHODS RELATED THERETO
#516LIPID FORMULATION
#517Epidermal growth factor receptor (EGFR) and methods of use in adenoviral-associated virus type 6 (AAV6) transduction
#518Liver-specific Nanocapsules and Methods of Using
#519Therapy for lysosomal enzyme deficiencies
#520E1-MINUS ADENOVIRUSES AND USE THEREOF
#521Sustained release drug delivery systems comprising a water soluble therapeutic agent and a release modifier
#522QUANTUM UNIT OF INHERITANCE VECTOR THERAPY METHOD
#523Targeted delivery of drugs, therapeutic nucleic acids and functional nucleic acids to mammalian cells via intact killed bacterial cells
#524DRUG DELIVERY VEHICLE FOR CANCER THERAPY, PROCESS FOR PRODUCING THE SAME, AND PHARMACEUTICAL PREPARATION USING THE SAME
#525Polynucleotide constructs, pharmaceutical compositions and methods for targeted downregulation of angiogenesis and anticancer therapy
#526DELIVERY OF MRNA FOR THE AUGMENTATION OF PROTEINS AND ENZYMES IN HUMAN GENETIC DISEASES
#527METHOD FOR TREATING AND PREVENTING RADIATION DAMAGE USING GENETICALLY MODIFIED MESENCHYMAL STEM CELLS
#528COMPOSITIONS AND METHODS FOR TOPICAL DELIVERY OF OLIGONUCLEOTIDES
#529Composition comprising gas-filled microcapsules for ultrasound mediated delivery
#530PROMOTERS EXHIBITING ENDOTHELIAL CELL SPECIFICITY AND METHODS OF USING SAME FOR REGULATION OF ANGIOGENESIS
#531Class of therapeutic protein based molecules
#532Genetically modified human umbilical cord perivascular cells for prophylaxis against or treatment of biological or chemical agents
#533CARBOHYDRATE FUNCTIONALIZED CATANIONIC SURFACTANT VESICLES FOR DRUG DELIVERY
#534PEGYLATED POLYPLEXES FOR POLYNUCLEOTIDE DELIVERY
#535Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
#536Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
#537NANOPARTICLE COMPOSITIONS FOR NUCLEIC ACIDS DELIVERY SYSTEM
#538Bacterially derived intact minicells that encompass plasmid free functional nucleic acid for in vivo delivery to mammalian cells
#539Immunomodulating gene therapy
#540PH sensitive biodegradable polymeric particles for drug delivery
#541OLIGONUCLEOTIDES-TRANSFERRING PREPARATIONS
#542Methods for nucleic acid transfer into cells
#543COMPOSITIONS FOR DELIVERY OF CARGO SUCH AS DRUGS PROTEINS AND/OR GENETIC MATERIALS
#544Biodegradable polyacetals for in vivo polynucleotide delivery
#545Decoy-containing pharmaceutical compositions and method of using the same
#546MIXED-CELL GENE THERAPY
#547SYN3 compositions and methods
#548Methods, compositions and drug delivery systems for intraocular delivery of siRNA molecules
#549Anti-Inflammatory Bacteria
#550Nucleic acids encoding hyperactive PiggyBac transposases
#551Methods and compositions for treatment of interferon-resistant tumors
#552Uniform field magnetization and targeting of therapeutic formulations
#553Methods and models for rapid, widespread delivery of genetic material to the CNS using non-viral, cationic lipid-mediated vectors
#554Biologic Modulations with Nanoparticles
#555αβintegrin binding RGD-lipopeptides with gene transfer activities
#556Drug delivery product and methods
#557ATTENUATED INVASIVE E.COLI STRAINS AND APPLICATIONS THEREOF AS INTRACELLULAR VECTOR FOR THERAPEUTIC MOLECULE
#558Method and formulation for treating adverse biological conditions
#559Excipients for use in adeno-associated virus pharmaceutical formulations, and pharmaceutical formulations made therewith
#560Use of liposomes in a carrier comprising a continuous hydrophobic phase for delivery of polynucleotides in vivo
#561NANOPARTICLE COMPOSITIONS FOR CONTROLLED DELIVERY OF NUCLEIC ACIDS
#562Isolation of novel AAV'S and uses thereof
#563Materials and complexes for the delivery of biologically-active materials to cells
#564Transfection complexes
#565Protein-coding RNA to correct mitochondrial dysfunction
#566PEPTIDE HAVING CELL MEMBRANE PENETRATING ACTIVITY
#567Extranuclear RNA splicing in neuronal dendrites
#568Method of producing microcapsules
#569DNA vaccines against tumor growth and methods of use thereof
#570Use of invaplex to transport functional proteins and transcriptionally active nucleic acids across mammalian cell membranes in vitro and in vivo
#571Stinging cells expressing an exogenous polynucleotide encoding a therapeutic, diagnostic or a cosmetic agent and methods compositions and devices utilizing such stinging cells or capsules derived therefrom for delivering the therapeutic, diagnostic or cosmetic agent into a tissue
#572Tyrosine-modified recombinant rAAV vector compositions and methods for use
#573System for delivering therapeutic agents into living cells and cells nuclei
#574TRAVERSAL OF NUCLEIC ACID MOLECULES THROUGH A FLUID SPACE AND EXPRESSION IN REPAIR CELLS
#575PROCESS FOR FACILITATING NUCLEIC ACID TRANSFER
#576Promoters exhibiting endothelial cell specificity and methods of using same for regulation of angiogenesis
#577Use of a truncated eIF-5A1 polynucleotide to induce apoptosis in cancer cells
#578Compositions and methods for detection and modulation of T cell mediated immune responses against viral vectors utilized for gene therapy
#579MEMBRANE(S) AND USES THEREOF
#580METHOD OF PROTECTING SENSITIVE MOLECULES FROM A PHOTO-POLYMERIZING ENVIRONMENT
#581Compositions comprising siRNA and plasmids
#582Compositions and methods for topical delivery of oligonucleotides
#583ENCAPSULATED NANOPARTICLES FOR DRUG DELIVERY
#584Methods and compositions for therapeutic use of RNA interference
#585ARPE-19 as a Platform Cell Line for Encapsulated Cell-Based Delivery
#586ARPE-19 as a Platform Cell Line for Encapsulated Cell-Based Delivery
#587Adeno-associated virus vectors
#588OLIGONUCLEOTIDES-TRANSFERRING PREPARATIONS
#589Zinc finger proteins and method for inactivating a dhfr gene in a chinese hamster ovary cell
#590Nanoencapsulation and Release of Nucleic Acids
#591LIVER-SPECIFIC NANOCAPSULES AND METHODS OF USING
#592E1-MINUS ADENOVIRUSES AND USE THEREOF
#593GENE TRANSFER METHOD
#594Method for enhanced uptake of viral vectors in the myocardium
#595GENE DELIVERY FORMULATIONS AND METHODS FOR TREATMENT OF ISCHEMIC CONDITIONS
#596METHODS AND COMPOSITIONS FOR USE OF CRL 5803 CELLS FOR EXPRESSION OF BIOTHERAPEUTICS AND ENCAPSULATED CELL-BASED DELIVERY
#597Antibody fragment-targeted immunoliposomes for systemic gene delivery
#598USE OF CELLS DERIVED FROM ADIPOSE TISSUE FOR THE PREPARATION OF AN ANTI-TUMOR MEDICAMENT
#599Complex for transferring an anionic substance into a cell
#600Materials and methods for treatment of inflammatory and cell proliferation disorders