21402 ⎘
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 wherein the nucleic acid is delivered as a 'naked' nucleic acid, i.e. not combined with an entity such as a cationic lipid
GENE EDITING SYSTEMS AND USES THEREOF
#2RECONSTITUTION OF LARGE GENES VIA CRE-LOX DNA RECOMBINATION IN ADENO-ASSOCIATED VIRUS VECTORS
#3ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS WITH IMPROVED RETINAL TRANSDUCTION AND USES THEREOF
#4METHODS AND SYSTEMS FOR IMPROVED NUCLEIC ACID DELIVERY VIA ULTRASOUND
#5METAL-CHELATED POLYPHENOL COMPLEX NANOPARTICLE, DRUG-LIPID PARTICLE, PREPARATION METHODS FOR THE SAME, AND USES THEREOF
#6MUTANTS OF IMMUNOGLOBULIN-DEGRADING ENZYME IDEE
#7CRISPR/Cas9-Mediated Exon-Skipping Approach for USH2A-Associated Usher Syndrome
#8Conjugated Antisense Compounds for Use in Therapy
#9NUCLEIC ACID MOLECULES AND USES THEREOF FOR NON-VIRAL GENE THERAPY
#10GENE THERAPY FOR HAPLOINSUFFICIENCY
#11TREATING METASTATIC CANCER AND MODEL SYSTEMS FOR METASTATIC DISEASE
#12TREATMENT REGIMENS FOR THERAPEUTIC EFFECTOR COMPONENTS THAT ACT VIA AN INTRACELLULAR MOLECULAR TARGET
#13USING DREADD FOR NEURONAL MODULATION IN TREATING NEURONAL DISEASES
#14METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBETA REDUCTASE
#15RAPIDLY METABOLIZED LIPID COMPOUND
#16NOVEL IMMUNOSTIMULATING VECTOR SYSTEM
#17METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#18METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#19LYMPHANGIOGENESIS-PROMOTING AGENTS
#20RESPIRATORY DELIVERY OF THERAPEUTIC AGENTS
#21SYSTEMS AND METHODS FOR NUCLEIC ACID EXPRESSION IN VIVO
#22ADMINISTRATION OF NAKED NUCLEIC ACID MOLECULE
#23Polynucleotides, Compositions, and Methods for Genome Editing
#24GENE EDITING TO IMPROVE JOINT FUNCTION
#25VARIANTS OF SIRT6 FOR USE IN PREVENTING AND/OR TREATING AGE-RELATED DISEASES
#26NON-VIRAL DNA VECTORS EXPRESSING THERAPEUTIC ANTIBODIES AND USES THEREOF
#27PHARMACEUTICAL COMPOSITION CONTAINING CELL-DERIVED NATURAL OR ARTIFICIAL NANOVESICLES LOADED WITH ANTISENSE OLIGONUCLEOTIDE-BASED DRUG
#28COMPOSITIONS USEFUL FOR TREATING SPINAL AND BULBAR MUSCULAR ATROPHY (SBMA)
#29COMPOUND, LIPOSOME, AND USES THEREOF
#30Conjugated Antisense Compounds for Use in Therapy
#31USE OF RIP1 INHIBITOR OR MLKL INHIBITOR FOR TREATING OR PREVENTING HEREDITARY RETINAL DYSTROPHY
#32NUCLEIC ACID VECTORS AND METHODS OF USE
#33OPTIMIZED FACTOR VIII GENES
#34OLIGONUCLEOTIDE COMPOSITIONS AND METHODS THEREOF
#35METHOD OF DELIVERING MRNA IN VIVO
#36ADENO-ASSOCIATED VIRUS CAPSIDS AND ENGINEERED LIGAND-GATED ION CHANNELS FOR TREATING FOCAL EPILEPSY AND NEUROPATHIC PAIN
#37METHODS OF PRECISE GENOME EDITING BY IN SITU CUT AND PASTE (ICAP)
#38ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS WITH IMPROVED RETINAL TRANSDUCTION AND USES THEREOF
#39METHODS AND COMPOSITIONS FOR NUCLEIC ACID AND PROTEIN PAYLOAD DELIVERY
#40CLOSED-ENDED DNA VECTORS AND USES THEREOF FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH)
#41METHODS AND FORMULATIONS FOR GENE THERAPY, AND FOR COMBINING GENE THERAPY WITH DITPA TREATMENT, OF ALLAN-HERNDON-DUDLEY SYNDROME
#42USING DREADD FOR NEURONAL MODULATION IN TREATING NEURONAL DISEASES
#43Gene therapy for haploinsufficiency
#44MOTOR NEURON-SPECIFIC EXPRESSION VECTORS
#45ADENO-ASSOCIATED VIRUS VECTOR FOR DWARF OPEN READING FRAME
#46Compositions and Methods for Treating Cancer with Anti-CD33 Immunotherapy
#47METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBETA REDUCTASE
#48COMPOSITIONS AND METHODS FOR REPROGRAMMING SKIN TISSUE TO HAVE INSULINOGENIC AND DELIVERY FUNCTIONS
#49Treatments for retinal disease
#50GENE THERAPY AND TARGETED DELIVERY OF CONJUGATED COMPOUNDS
#51Treating metastatic cancer and model systems for metastatic disease
#52TREATMENT OF LIVER CANCER OR LIVER FIBROSIS
#53METHODS AND SYSTEMS FOR NONINVASIVE CONTROL OF BRAIN CELLS AND RELATED VECTORS AND COMPOSITIONS
#54COMPOSITIONS AND METHODS FOR EXPRESSING FACTOR IX FOR HEMOPHILIA B THERAPY
#55COMPOSITIONS AND METHODS FOR IMPROVING VIRAL VECTOR EFFICIENCY
#56DELIVERY OF COMPOSITIONS COMPRISING CIRCULAR POLYRIBONUCLEOTIDES
#57NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING PHENYLALANINE HYDROXYLASE (PAH) THERAPEUTICS
#58DNA ANTIBODY CONSTRUCTS AND METHOD OF USING SAME
#59Treatments for retinal disease
#60GENE EDITING USING A MODIFIED CLOSED-ENDED DNA (CEDNA)
#61Methods of treating and diagnosing epilepsy
#62METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#63LYMPHANGIOGENESIS-PROMOTING AGENTS
#64Method of Treating Hepatic Steatosis
#65Methods and compositions for treating cancer by targeting the CLEC2D-KLRB1 pathway
#66METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#67Methods and compositions for nucleic acid and protein payload delivery
#68Gene therapy DNA vectors based on VTVAF17
#69OVEREXPRESSION OF FOXC1 TO TREAT CORNEAL VASCULARIZATION
#70Immunostimulating vector system
#71METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBETA REDUCTASE
#72ADENO-ASSOCIATED VIRUS VARIANT CAPSIDS AND METHODS OF USE THEREOF
#73Conjugated Antisense Compounds for Use in Therapy
#74Adeno-associated virus variant capsids and methods of use thereof
#75Gene Therapy for Muscle Improvement
#76CRISPR/Cas9-Mediated Exon-Skipping Approach for USH2A-Associated Usher Syndrome
#77Motile sperm domain containing protein 2 and inflammation
#78Method of using adeno-associated virus with variant capsid
#79Adeno-associated virus variant capsids and methods of use thereof
#80Method of Treating Insulin Resistance
#81MODRNA ENCODING SPHINGOLIPID-METABOLIZING PROTEINS
#82Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#83Wound healing through sirt1 overexpression
#84COMPOSITION FOR ENDOGENOUS PRODUCTION OF CHECKPOINT PROTEIN PRECURSORS
#85Methods of improving adeno-associated viral transduction
#86Motor neuron-specific expression vectors
#87DNA-binding domain of CRISPR system, non-fucosylated and partially fucosylated proteins, and methods thereof
#88COMPOSITIONS AND METHODS FOR TUMOR TRANSDUCTION
#89COMPOSITIONS AND METHODS FOR TREATING PAIN DISORDERS
#90METHODS FOR INCREASING PLATELET COUNT BY INHIBITING BILIVERDIN IXBETA REDUCTASE
#91METHODS AND PHARMACEUTICAL COMPOSITIONS FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#92COMPOSITIONS AND METHODS FOR DETECTION, RISK ASSESSMENT AND TREATMENT OF DIABETES, OBESITY, AND INFLAMMATION
#93MULTIGENE CONSTRUCT FOR IMMUNE-MODULATORY PROTEIN EXPRESSION AND METHODS OF USE
#94Compositions and methods for treating cancer with anti-CD33 immunotherapy
#95COMPOSITIONS AND METHODS FOR NUCLEIC ACID AND/OR PROTEIN PAYLOAD DELIVERY
#96NUCLEIC ACID MOLECULES AND USES THEREOF FOR NON-VIRAL GENE THERAPY
#97CRISPR-BASED TREATMENT OF FRIEDREICH ATAXIA
#98Lyophilized pharmaceutical compositions for naked DNA gene therapy
#99RNA for use in the treatment of ligament or tendon lesions
#100COMBINATION THERAPIES FOR ERADICATING FLAVIVIRUS INFECTIONS IN SUBJECTS
#101Transgenic mouse expressing amyloid precursor protein that has olfactory neuron degeneration
#102Gene therapy for haploinsufficiency
#103LYMPHANGIOGENESIS-PROMOTING AGENTS
#104Adipocyte-targeting non-viral gene delivery complex comprising dual plasmid vector
#105GENE THERAPY AND TARGETED DELIVERY OF CONJUGATED COMPOUNDS
#106Overexpression of FOXC1 to treat corneal vascularization
#107Adeno-associated virus variant capsids and methods of use thereof
#108Poly(histidine)-based micelles for complexation and delivery of proteins and nucleic acids
#109METHODS OF MODIFYING THE DYSTROPHIN GENE AND RESTORING DYSTROPHIN EXPRESSION AND USES THEREOF
#110MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#111MODULATION OF EXON RECOGNITION IN PRE-MRNA BY INTERFERING WITH THE SECONDARY RNA STRUCTURE
#112Optimized factor VIII genes
#113ANGIOPOIETIN-LIKE PROTEIN 8 (ANGPTL8)
#114Methods and systems for noninvasive control of brain cells and related vectors and compositions
#115Treating metastatic cancer and model systems for metastatic disease
#116Tumor suppressor TET1 and uses thereof
#117Method of treating insulin resistance
#118Compositions and methods for selective inhibition of grainyhead-like protein expression
#119Adeno-associated virus compositions for restoring HBB gene function and methods of use thereof
#120Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#121TREATMENT OF NEUROPATHY WITH DNA CONSTRUCT EXPRESSING HGF ISOFORMS WITH REDUCED INTERFERENCE FROM GABAPENTINOIDS
#122POROUS NANOPARTICLE-SUPPORTED LIPID BILAYERS (PROTOCELLS) FOR TARGETED DELIVERY AND METHODS OF USING SAME
#123Systems and Methods for Nucleic Acid Expression In Vivo
#124Using DREADD for neuronal modulation in treating neuronal diseases
#125An mRNA cancer vaccine encoding human GM-CSF fused to multiple tandem epitopes
#126Enhancing plasmin activity to prevent soft tissue calcification
#127Immunostimulating vector system
#128Systems and methods for nucleic acid expression in vivo
#129CONJUGATED ANTISENSE COMPOUNDS FOR USE IN THERAPY
#130TARGETED MRNA FOR IN VIVO APPLICATION
#131Oligonucleotide compositions and methods thereof
#132TREATMENT OF DEVELOPMENTAL SYNDROMES
#133Therapeutic targets for the correction of the human dystrophin gene by gene editing and methods of use
#134INHIBITORS FOR METHYLATION-RELATED ENZYMES HAT1 AND KAT8
#135METHODS AND PHARMACEUTICAL COMPOSITION FOR THE TREATMENT AND THE PREVENTION OF CARDIOMYOPATHY DUE TO ENERGY FAILURE
#136METHOD OF EVALUATING OR SELECTING ANDROGEN RECEPTOR ACTIVITY REGULATOR SELECTIVE TO SEBACEOUS GLANDS OR HAIR FOLLICLES
#137Compositions and methods for improving viral vector efficiency
#138Methods for increasing platelet count by inhibiting biliverdin IXβ reductase
#139Retroviral particles expressing Sirt1 embedded within PPCN
#140CELL-BASED TARGETED DELIVERY OF PSEUDONOMAS EXOTOXIN
#141Compositions and methods for treating cancer with anti-CD33 immunotherapy
#142Short non-coding protein regulatory RNAs (sprRNAs) and methods of use
#143Method effective to modulate expression of T-box protein 4 (Tbx4) for reducing progression of lung fibrosis after a lung injury
#144Motor neuron-specific expression vectors
#145Human microRNAs for treatment of malignant tumours
#146Compositions and methods for modulating FMR1 expression
#147METHOD AND COMPOSITIONS FOR REMOVING DUPLICATED COPY NUMBER VARIAIONS (CNVS) FOR GENETIC DISORDERS AND RELATED USES
#148Antibody fragments, compositions and uses thereof
#149Modified siRNA and pharmaceutical composition
#150Pharmaceutical composition for inhibiting resistance against anticancer drugs of patient suffering from ovarian cancer comprising NAG-1 inhibitor as active ingredient
#151ß2GPI GENE EXPRESSION-SUPPRESSING NUCLEIC ACID CONJUGATE
#152Adenoassociated virus vectors for the treatment of mucopolysaccharidoses
#153NON-INTEGRATING VIRAL DELIVERY SYSTEM AND METHODS OF USE THEROF
#154Methods and compositions for nucleic acid and protein payload delivery
#155DNA-based plasmid formulations and vaccines and prophylactics containing the same
#156Therapy for malignant disease
#157METHODS FOR TREATING NEURODEGENERATIVE DISEASES
#158MALAT-1, A NON-CODING RNA, IS A TARGET FOR THE REGULATION OF LEARNING AND MEMORY
#159Method for treating peripheral vascular disease using hepatocyte growth factor and stromal cell derived factor 1A
#160Methods of Inducing Exon Skipping
#161Methods of Inducing Exon Skipping
#162DNA antibody constructs and method of using same
#163Messenger RNA nanoparticles and preparation method therefor
#164RNA for use in the treatment of ligament or tendon lesions
#165Systems and methods for nucleic acid expression in vivo
#166LYMPHANGIOGENESIS-PROMOTING AGENTS
#167Nuclear localization of GLP-1 stimulates myocardial regeneration and reverses heart failure
#168GENE THERAPY FOR DIABETIC ISCHEMIC DISEASE
#169Porous nanoparticle-supported lipid bilayers (protocells) for targeted delivery and methods of using same
#170Methods and pharmaceutical compositions for the treatment and the prevention of cardiomyopathy due to Friedreich ataxia
#171Implantable liposome embedded matrix composition, uses thereof, and polycaprolactone particles as scaffolds for tissue regeneration
#172Methods and pharmaceutical compositions for the treatment of cardiomyopathy due to friedreich ataxia
#173Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#174Sustained-release lipid pre-concentrate of pharmacologically active substance and pharmaceutical composition comprising the same
#175Porous nanoparticle-supported lipid bilayers (protocells) for targeted delivery and methods of using same
#176Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#177IMPLANTABLE LIPOSOME EMBEDDED MATRIX COMPOSITION, USES THEREOF, AND POLYCAPROLACTONE PARTICLES AS SCAFFOLDS FOR TISSUE REGENERATION
#178Modulation of exon recognition in pre-mRNA by interfering RNA structure
#179LYMPHANGIOGENESIS-PROMOTING AGENTS
#180METHODS AND COMPOSITIONS FOR TARGETED DELIVERY OF GENE THERAPEUTIC VECTORS
#181GENE THERAPY FOR DIABETIC ISCHEMIC DISEASE
#182Compositions comprising GHRH and GnRH and methods of using the same
#183MATERIALS AND METHODS FOR THE DELIVERY OF BIOMOLECULES TO CELLS OF AN ORGAN
#184Decreasing cancer cell proliferation with WNT remaining active
#185Regenerating and enhancing development of muscle tissue
#186Means and method for inducing exon-skipping
#187Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#188REDUCING CULLING IN HERD ANIMALS GROWTH HORMONE RELEASING HORMONE (GHRH)
#189Gene therapy for diabetic ischemic disease
#190Genetic Immunization
#191DNA-based plasmid formulations and vaccines and prophylactics containing the same
#192Expression of zeta negative and zeta positive nucleic acids using a dystrophin gene
#193Cell proliferation inhibitory proteins and polynucleotides, antisense polynucleotides to the polynucleotides, cell proliferation inhibitors using the foregoing, cancer diagnostic agents, cancer therapeutic agents and compositions for gene therapy
#194Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#195Methods for treating or preventing angiogenesis-dependent symptoms
#196Modulation of exon recognition in pre-mRNA by interfering with the secondary RNA structure
#197Process for delivering nucleic acids to cardiac tissue
#198Nucleic acid therapy to enhance cartilage repair
#199Drugs to be used in gene therapy for recessively transmitted genetic disease
#200Intravascular delivery of non-viral nucleic acid
#201Non-viral linear DNA vectors and methods for using the same