112319 ⎘
Vectors comprising a special translation-regulating system cell or tissue specific
TISSUE SPECIFIC SYNTHETIC PROMOTERS AND CIRCUITS AND USE THEREOF
#2COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#3ENGINEERED POLYNUCLEOTIDES FOR CELL SELECTIVE EXPRESSION
#4NPP1 Fusion Proteins
#5METHODS OF TREATING NON-SYNDROMIC SENSORINEURAL HEARING LOSS
#6APTAMER AND RIBOZYME EQUILIBRIUM SHIFTING (ARES) RNA CIRCUITS AND USES THEREOF
#7Compositions for Treating Pathological Calcification Conditions, and Methods Using Same
#8TRANSCRIPTIONAL AND TRANSLATIONAL DUAL REGULATED ONCOLYTIC HERPES SIMPLEX VIRUS VECTORS
#9VECTOR CONSTRUCTS FOR DELIVERY OF NUCLEIC ACIDS ENCODING THERAPEUTIC ANTI-IGF-1R ANTIBODIES AND METHODS OF USING THE SAME
#10METHODS AND COMPOSITIONS RELATING TO THE TREATMENT OF TUMORS
#11COMPOSITIONS COMPRISING KOZAK SEQUENCES SELECTED FOR ENHANCED EXPRESSION
#12PROTEIN DEGRADATION SYSTEM, COMPONENTS, AND METHODS
#13IMPROVED NUCLEIC ACID SEQUENCE FOR CELL TYPE SPECIFIC EXPRESSION
#14COMPOSITIONS AND METHODS FOR TREATING RIGHT VENTRICLE DYSFUNCTION
#15REGULATORY SYSTEM FOR EXPRESSION OF A GENE OF INTEREST IN A TARGET CELL AND METHOD OF USE THEREOF
#16Gene Expression System
#17ANTI-IDIOTYPE COMPOSITIONS AND METHODS OF USE THEREOF
#18IMMUNOENGINEERED PLURIPOTENT CELLS
#19CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#20GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A
#21Self-Complementary Adeno-Associated Virus Vector and its Use in Treatment of Muscular Dystrophy
#22FUSOGENIC LIPID NANOPARTICLES AND METHODS FOR THE MANUFACTURE AND USE THEREOF FOR THE TARGET CELL-SPECIFIC PRODUCTION OF A THERAPEUTIC PROTEIN AND FOR THE TREATMENT OF A DISEASE, CONDITION, OR DISORDER ASSOCIATED WITH A TARGET CELL
#23PROSTATE TARGETING ADENO-ASSOCIATED VIRUS SEROTYPE VECTORS
#24PLATFORM FOR THE INDUCTION & MAINTENANCE OF GROUND STATE PLURIPOTENCY
#25VIRAL VECTORS ENCODING RECOMBINANT FVIII VARIANTS WITH INCREASED EXPRESSION FOR GENE THERAPY OF HEMOPHILIA A
#26REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
#27HYBRID PROMOTERS FOR MUSCLE EXPRESSION
#28COMPOSITIONS AND METHODS FOR SELECTIVE GENE REGULATION
#29Compositions for treating pathological calcification conditions, and methods using same
#30GENE THERAPY FOR OCULAR DISORDERS
#31Methods of treating non-syndromic sensorineural hearing loss
#32PROMOTER FOR THE SPECIFIC EXPRESSION OF GENES IN MÜLLER CELLS
#33GENERATING GABAergic NEURONS IN BRAINS
#34Isolation of novel AAV's and uses thereof
#35COMPOSITIONS AND METHODS FOR TREATING AGE-RELATED MACULAR DEGENERATION
#36Gene expression system
#37MICRORNA REGULATED EXPRESSION VECTORS, METHODS OF MAKING, AND USES THEREOF
#38Efficient systemic treatment of dystrophic muscle pathologies
#39Methods for treating pseudoxanthoma elasticum (PXE)
#40EXPRESSION VECTORS AND RELATED METHODS OF DELIVERY OF Na/K ATPASE/Src RECEPTOR COMPLEX ANTAGONISTS
#41Compositions and methods of dual poison-antidote meiotic drivers
#42Transcriptionally targeted and CpG-free plasmid for theranostic gene therapy
#43Compositions for treating pathological calcification conditions, and methods using same
#44MODIFIED UBE3A GENE FOR A GENE THERAPY APPROACH FOR ANGELMAN SYNDROME
#45GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A
#46Gene therapy for ocular disorders
#47Compositions and methods for improving plastid transformation efficiency in higher plants
#48Stable gene transfer to proliferating cells
#49IMMUNOENGINEERED PLURIPOTENT CELLS
#50Polynucleotides and vectors for the expression of transgenes
#51Methods and compositions relating to the treatment of tumors
#52Expression constructs, fusogenic lipid-based nanoparticles and methods of use thereof
#53Isolation of novel AAV's and uses thereof
#54CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#55CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#56Tumor-specific adenovirus vectors and therapeutic uses
#57Compositions for treating pathological calcification conditions, and methods using same
#58Platform for the induction and maintenance of ground state pluripotency
#59Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#60CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#61CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#62Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#63Efficient systemic treatment of dystrophic muscle pathologies
#64TISSUE SELECTIVE TRANSGENE EXPRESSION
#65Tissue selective transgene expression
#66Tissue selective transgene expression
#67Tissue selective transgene expression
#68Prostate-targeting adeno-associated virus serotype vectors
#69Tumor suppressor SALL1 as a therapeutic agent for treating cancer
#70Plants having increased tolerance to herbicides
#71CNS TARGETING AAV VECTORS AND METHODS OF USE THEREOF
#72Promoter for the specific expression of genes in Müller cells
#73Compositions for treating pathological calcification conditions, and methods using same
#74Compositions for treating pathological calcification conditions, and methods using same
#75Compositions for treating pathological calcification conditions, methods, and host cells using the same
#76TERMINAL MODIFICATIONS OF POLYNUCLEOTIDES
#77CNS targeting AAV vectors and methods of use thereof
#78Compositions for treating pathological calcification conditions, and methods using same
#79Compositions for treating pathological calcification conditions, and methods using same
#80REGULATABLE ADENO-ASSOCIATED VIRUS (AAV) VECTOR
#81Generating GABAergic neurons in brains
#82Expression cassette for packaging and expression of variant factor VIII for the treatment of hemostasis disorders
#83Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#84Viral vectors encoding recombinant FVIII variants with increased expression for gene therapy of hemophilia A
#85METHOD TO SELECTIVELY TARGET CANCEROUS CELLS FOR GENETIC MANIPULATION
#86Stable Gene Transfer to Proliferating Cells
#87GENE EXPRESSION SYSTEM
#88Isolation of novel AAV's and uses thereof
#89Isolation of novel AAV's and uses thereof
#90Efficient systemic treatment of dystrophic muscle pathologies
#91NPP1 FUSION PROTEINS
#92Synthetic regulation of gene expression
#93Non-viral episomal suicide construct
#94Methods of modulating smooth muscle cell proliferation and differentiation
#95Alpha amylase mutant inhibitors isolated from phaseolus vulgaris with properties of controlling insect pests, compositions containing such mutants, and method of using thereof
#96CNS targeting AAV vectors and methods of use thereof
#97Synthetic regulation of gene expression
#98CNS targeting AAV vectors and methods of use thereof
#99Methods of modulating smooth muscle cell proliferation and differentiation
#100Detection and destruction of cancer cells using programmed genetic vectors
#101Isolation of novel AAV'S and uses thereof
#102Diabetes Model Animal
#103Cre-lox based gene knockdown constructs and methods of use thereof
#104GENE THERAPY FOR NIEMANN-PICK DISEASE TYPE A
#105Method of treating fibrosis
#106Tmprss2 Regulatory Sequences and Uses Thereof
#107METHOD OF TREATING FIBROSIS
#108Method for treating fibrosis