112203 ⎘
Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus
SATIATION PEPTIDE ADMINISTRATION
#2GENE THERAPY FOR AMYOTROPHIC LATERAL SCLEROSIS AND OTHER SPINAL CORD DISORDERS
#3Liver-Specific Nucleic Acid Regulatory Elements and Methods and Use Thereof
#4GENE-THERAPY VECTORS FOR TREATING CARDIOMYOPATHY
#5Immunomodulating Gene Therapy
#6PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#7Vectors for Liver-Directed Gene Therapy of Hemophilia and Methods and Use Thereof
#8NERVE REGENERATION
#9REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#10Satiation peptide administration
#11Genome editing without nucleases
#12Methods and compositions for regulation of transgene expression
#13PEPTIDES HAVING SPECIFICITY FOR THE LUNGS
#14Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
#15Genome Editing without Nucleases
#16GENE THERAPY FOR SPINAL CORD DISORDERS
#17Gene therapy for neurometabolic disorders
#18GENE THERAPY COMPOSITION FOR USE IN DIABETES TREATMENT
#19Method for inducing production of vascular endothelial growth factor
#20MODULATING PHOSPHATASE ACTIVITY IN CARDIAC CELLS
#21Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
#22Gene-therapy vectors for treating cardiomyopathy
#23Satiation peptide administration
#24MODULATING PHOSPHATASE ACTIVITY IN CARDIAC CELLS
#25Liver-specific nucleic acid regulatory elements and methods and use thereof
#26HLA Class II Deficient Cells, HLA Class I Deficient Cells Capable of Expressing HLA Class II Proteins, and Uses Thereof
#27Process for recovering viral products using functionalised chromatography media
#28Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
#29Drug composition for angiogenesis therapy
#30Methods and compositions for regulation of transgene expression
#31Method for Treating Melanoma Using a Herpes Simplex Virus and an Immune Checkpoint Inhibitor
#32Liver-specific nucleic acid regulatory elements and methods and use thereof
#33Immunomodulating gene therapy
#34PROMOTERS, EXPRESSION CASSETTES, VECTORS, KITS, AND METHODS FOR THE TREATMENT OF ACHROMATOPSIA AND OTHER DISEASES
#35Atherosclerosis inhibition via modulation of monocyte-macrophage phenotype using Apo A-I Milano gene transfer
#36Gene therapy for the treatment of a disease of retinal cone cells
#37Production of large-sized microdystrophins in an AAV-based vector configuration
#38Reagents and methods for modulating cone photoreceptor activity
#39OPTICALLY-CONTROLLED CNS DYSFUNCTION
#40Modulating phosphatase activity in cardiac cells
#41Therapeutical tools and methods for treating blindness
#42Methods and compositions for regulation of transgene expression
#43RNA interference suppression of neurodegenerative diseases and methods of use
#44Adeno-associated viral vectors for the gene therapy of metabolic diseases
#45Guided injections for AAV gene transfer to muscle
#46Liver-specific nucleic acid regulatory elements and methods and use thereof
#47Treatment of amyotrophic lateral sclerosis
#48Recombinant swine influenza virus and uses thereof
#49Genome editing without nucleases
#50SorCS1 for the treatment of obesity
#51Brown adipocyte modification
#52Capsid-mutated rAAV vectors and methods of use
#53Optically-controlled CNS dysfunction
#54Light-sensitive ion-passing molecules
#55Therapeutical tools and methods for treating blindness
#56Cell and gene based methods to improve cardiac function
#57Peptides having specificity for the lungs
#58Delivery of a therapeutic agent to the cerebral cortex by administering a viral vector by convection enhanced diffusion into the white matter of the brain
#59Gene-therapy vectors for treating cardiomyopathy
#60Vectors encoding rod-derived cone viability factor
#61Immunomodulating gene therapy
#62Gene therapy composition for use in diabetes treatment
#63SorCS1-like agent for use in the treatment of insulin resistance and diseases related thereto
#64Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same
#65Methods and compositions for regulation of transgene expression
#66Vectors for liver-directed gene therapy of hemophilia and methods and use thereof
#67Pain treatment using ERK2 inhibitors
#68Treatment of brain cancers using central nervous system mediated gene transfer of monoclonal antibodies
#69Method for treating melanoma using a herpes simplex virus and an immune checkpoint inhibitor
#70Optically controlled CNS dysfunction
#71Gene therapy for neurometabolic disorders
#72Gene therapy composition for use in diabetes treatment
#73Vectors encoding rod-derived cone viability factor
#74RNA interference suppression of neurodegenerative diseases and methods of use thereof
#75Brown adipocyte modification
#76Modified factor VIII and factor IX genes
#77Modified viral structural protein with antiviral activity
#78Homozygous and heterozygous IDH1 gene-defective cell lines derived from human colorectal cells
#79Methods for producing recombinant proteins
#80Methods and compositions for treating brain diseases
#81Reagents and methods for modulating cone photoreceptor activity
#82Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
#83Optically controlled CNS dysfunction
#84Puf-A and related compounds for treatment of retinopathies and sight-threatening ophthalmologic disorders
#85Methods and compositions for regulation of transgene expression
#86Methods and compositions for regulation of transgene expression
#87Light-sensitive ion-passing molecules
#88Light-sensitive ion-passing molecules
#89Novel Therapeutical Tools and Methods for Treating Blindness
#90Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same
#91NUCLEAR LOCALIZATION SIGNAL PEPTIDES DERIVED FROM VP2 PROTEIN OF CHICKEN ANEMIA VIRUS AND USES OF SAID PEPTIDES
#92Light-sensitive ion-passing molecules
#93Modified factor VIII and factor IX genes and vectors for gene therapy
#94Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
#95System for increasing gene expression and vector comprising the system
#96Methods for producing recombinant proteins
#97MEDICAMENT FOR THE TREATMENT AND PREVENTION OF LIVER FAILURE
#98Novel Therapeutical Tools and Methods for Treating Blindness
#99Cardiac-specific nucleic acid regulatory elements and methods and use thereof
#100Specific Antibodies To Amyloid Beta Peptide, Pharmaceutical Compositions And Methods Of Use Thereof
#101Methods for inhibiting starvation of a cell
#102REGULATED EXPRESSION SYSTEMS
#103SorCS1 for the treatment of obesity
#104Light-receiving channel rhodopsin having improved expression efficiency
#105REAGENTS AND METHODS FOR MODULATING CONE PHOTORECEPTOR ACTIVITY
#106SorCS1-like agent for use in the treatment of insulin resistance and diseases related thereto
#107DRUG COMPOSITION FOR ANGIOGENESIS THERAPY
#108COMPOSITIONS AND METHODS FOR TREATING PLASMA PROTEIN DEFICIENCY DISORDERS
#109ISOGENIC HUMAN CELL LINES COMPRISING MUTATED CANCER ALLELES AND PROCESS USING THE CELL LINES
#110VECTORS FOR DELIVERY OF LIGHT SENSITIVE PROTEINS AND METHODS OF USE
#111METHODS FOR TREATING PARKINSON'S DISEASE AND OTHER DISORDERS OF DOPAMINERGIC NEURONS OF THE BRAIN
#112Splice Switching Oligomers for TNF Superfamily Receptors and Their Use in Treatment of Disease
#113Satiation peptide administration
#114PDK inhibitor compounds and methods of use thereof
#115Pain treatment using ERK2 inhibitors
#116System for Modulating Expression of Hypothalmic Brain-Derived Neurotrophic Factor (BDNF)
#117HDAC4 nucleic acid administration to treat retinal disease
#118Porphobilinogen deaminase gene therapy
#119METHODS AND MATERIALS FOR REDUCING OR SUPPRESSING AMYLOID DEPOSITION
#120SERCA2 THERAPEUTIC COMPOSITIONS AND METHODS OF USE
#121RNA interference suppression of neurodegenerative diseases and methods of use thereof
#122Compositions and methods for detecting and modulating cell death by a translation regulated gene expression system
#123Liver-specific nucleic acid regulatory elements and methods and use thereof
#124METHOD OF TREATING HEARING LOSS USING XIAP
#125RAB3B for treatment and prevention of Parkinson's disease
#126Upregulation of opioid receptors for management
#127Immunomodulating gene therapy
#128METHODS FOR CLONING FERRETS AND TRANSGENIC FERRET MODELS FOR DISEASES
#129RECOMBINANT ANTIBODIES SPECIFIC FOR BETA-AMYLOID ENDS, DNA ENCODING AND METHODS OF USE THEREOF
#130NEW MODULATING MOLECULES FOR AN IMPROVED REGULATED EXPRESSION SYSTEM
#131Method of expanding human hepatocytes in vivo
#132Methods and compositions for regulated expression of multiple nucleic acids
#133BONE TARGETED ALKALINE PHOSPHATASE, KITS AND METHODS OF USE THEREOF
#134Compositions and methods for making therapies delivered by viral vectors reversible for safety and allele-specificity
#135Modified factor VIII and factor IX genes and vectors for gene therapy
#136ADENO-ASSOCIATED VIRUS SEROTYPE I NUCLEIC ACID SEQUENCES, VECTORS AND HOST CELLS CONTAINING SAME
#137Gene therapy for amyotrophic lateral sclerosis and other spinal cord disorders
#138Methods of inhibiting photoreceptor apoptosis
#139Myostatin inhibition for enhancing muscle and/or improving muscle function
#140Pig model for atherosclerosis
#141Therapeuting compositions comprising an RNAi agent and a neurotrophic factor and methods of use thereof
#142METHODS AND COMPOSITIONS RELATED TO APOBEC-1 EXPRESSION
#143DEVICES AND METHODS FOR DELIVERING POLYNUCLEOTIDES INTO RETINAL CELLS OF THE MACULA AND FOVEA
#144DISRUPTION OF PROGRAMMED DEATH 1 (PD-1) LIGAND TO ADJUVANT ADENO-ASSOCIATED VIRUS VECTOR VACCINES
#145Use of nucleic acid sequences for the treatment of epilepsy
#146R-RAS activity in vascular regulation
#147Splice Switching Oligomers for TNF Superfamily Receptors and their Use in Treatment of Disease
#148METHODS FOR CLONING FERRETS AND TRANSGENIC FERRET MODELS FOR DISEASES
#149Novel Gene Therapy Approach For Treating The Metabolic Disorder Obesity
#150Rescue of photoreceptors by intravitreal administration of an expression vector encoding a therapeutic protein
#151Novel Gene Therapy Approach For Treating The Metabolic Disorder Obesity
#152Methods and Compositions for Mycoplasma Toxins
#153Gene therapy for spinal cord disorders
#154RECOMBINANT ANTIBODIES SPECIFIC FOR BETA-AMYLOID ENDS, DNA ENCODING AND METHODS OF USE THEREOF
#155Dekkera/Brettanomyces Cytosine Deaminases And Their Use
#156Recombinant adeno-associated virus vector for treatment of Alzheimer disease
#157Method of delivery of nucleic acids to peripheral neurons
#158Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions
#159GENE THERAPY FOR SPINAL CORD DISORDERS
#160Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof
#161Compounds and methods to enhance rAAV transduction
#162COMPOSITIONS AND METHODS FOR THE TREATMENT OF CARDIOVASCULAR CONDITIONS
#163Anti-angiogenic methods and compositions
#164Anemia
#165Modulating phosphatase activity in cardiac cells
#166Anti-angiogenic methods and compositions
#167Anti-angiogenic methods and compositions
#168Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions vectors
#169Adeno-associated virus serotype I nucleic acid sequences, vectors and host cells containing same
#170Adeno-associated virus serotype I nucleic acid sequences, vectors and host cell containing same
#171Transgenic ungulates capable of human antibody production
#172Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
#173Compositions for treating cystic fibrosis
#174Minigene expression cassette
#175Methods and compositions for the treatment of dystonia
#176Induction of tolerance by oral administration of factor VIII and treatment of hemophilia
#177Random peptide library displayed on aav vectors
#178Adeno-associated virus-mediated survivin mutants and methods related thereto
#179Anti-angiogenic methods and compositions
#180Adeno-associated virus serotype 1 nucleic acid sequences, vectors and host cells containing same
#181Polynucleotide delivery to cardiac tissue
#182Methods for xenotopic expression of nucleus-encoded plant and protist peptides and uses thereof
#183Methods for delivering DNA to muscle cells using recombinant adeno-associated virus virions vectors
#184Compounds and methods to enhance rAAV transduction
#185DNA sequences encoding dystrophin minigenes and methods of use thereof
#186NERVE REGENERATION
#187Methods for administering recombinant adeno-associated virus virons to humans previously exposed to adeno-associated virus
#188Adeno-associated vector compositions for expression of Factor VIII
#189RAAV-neprilysin compositions and methods of use
#190Polynucleotide encoding human adenylylcyclase VI and uses thereof for enhancing cardiac function
#191Anemia
#192Methods and compositions for use in interventional pharmacogenomics
#193Adeno-associated virus-delivered ribozyme compositions and methods for the treatment of retinal diseases
#194Compositions and methods for treating cardiac dysfunction