116539 ⎘
Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2); Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1) Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase
POLYNUCLEOTIDES FOR THE TREATMENT OF DISEASE ASSOCIATED WITH GCASE DEFICIENCY
#2POLYNUCLEOTIDES
#3METHODS FOR TREATING FARBER DISEASE
#4CNS DELIVERY OF THERAPEUTIC AGENTS
#5BLOOD-BRAIN BARRIER TRANSLOCATING PEPTIDES AND RELATED MOLECULES AND METHODS OF USE THEREOF
#6TRANSFERRIN RECEPTOR-BINDING DOMAINS AND PROTEINS COMPRISING THE SAME
#7GENE THERAPY FOR GAUCHER DISEASE
#8ICAM-1 TARGETED FUSION ENZYMES
#9NOVEL IN VITRO AND IN VIVO ENRICHMENT STRATEGY TARGETING LYMPHOCYTES DERIVED FROM VECTOR TRANSDUCED HSCS FOR THERAPY OF DISORDERS
#10METHODS AND COMPOSITIONS FOR CNS DELIVERY OF ARYLSULFATASE A
#11METHODS OF TREATING GAUCHER DISEASE AND GBA-PARKINSON’S DISEASE
#12METHODS OF TREATING NEURODEGENERATIVE DISORDERS
#13COMPOSITIONS COMPRISING GLUCOCEREBROCIDASE AND METHODS OF USE THEREOF
#14PROTEIN HAVING GLUCOCEREBROSIDASE ACTIVITY AND METHOD FOR PRODUCING SAME
#15GLYCOSYLATED PROTEIN HAVING GLUCOCEREBROSIDASE ACTIVITY
#16COMPOSITIONS AND METHODS OF USING TWO-PROMOTER VECTOR FOR TREATMENT OF LYSOSOMAL STORAGE DISORDERS
#17RECOMBINANT GLUCOCEREBROSIDASE PROTEIN HAVING IMPROVED ENZYME ACTIVITY OR IMPROVED STABILITY
#18METHODS AND COMPOSITIONS FOR CNS DELIVERY OF IDURONATE-2-SULFATASE
#19AAV-BASED MODULATION OF GBA1 AND RELATED COMPOSITIONS AND USES THEREOF
#20COMPOSITONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
#21RECOMBINANT AAV VECTORS FOR TREATING NEURODEGENERATIVE DISORDERS
#22FUSION PROTEINS TARGETED TO THE CENTRAL NERVOUS SYSTEM
#23GENE THERAPY FOR NEURODEGENERATIVE DISORDERS
#24Integrated Continuous Manufacturing of Therapeutic Protein Drug Substances
#25Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders
#26ICAM-1 targeted fusion enzymes
#27POLYPEPTIDE
#28A GENETICALLY MODIFIED CELL LINE PRODUCING A RECOMBINANT GLYCOPROTEIN HAVING A MANNOSE-TERMINATED N-GLYCAN
#29SYSTEMS AND METHODS FOR PROTEIN EXPRESSION
#30GENE THERAPIES FOR LYSOSOMAL DISORDERS
#31ASSAY FOR MEASURING POTENCY OF GENE THERAPY DRUG PRODUCT
#32DOPAMINERGIC NEURONS COMPRISING MUTATIONS AND METHODS OF USE THEREOF
#33LYSOSOMAL ENZYMES MODIFIED BY CELL BASED GLYCOENGINEERING
#34Novel In Vitro and In Vivo Enrichment Strategy Targeting Lymphocytes Derived from Vector Transduced HSCS for Therapy of Disorders
#35GENE THERAPIES FOR LYSOSOMAL DISORDERS
#36A GENETICALLY MODIFIED CELL LINE PRODUCING A RECOMBINANT GLYCOPROTEIN HAVING A MANNOSE-TERMINATED N-GLYCAN
#37GENE THERAPIES FOR LYSOSOMAL DISORDERS
#38Methods and compositions for the treatment of steatosis-associated disorders
#39Methods and Compositions for the Treatment of Steatosis-Associated Disorders
#40VIRAL PARTICLES FOR USE IN TREATING TAUOPATHIES SUCH AS ALZHEIMER'S DISEASES BY GENE THERAPY
#41COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA DEFICIENCY
#42METHODS AND COMPOSITIONS FOR TREATING CYTOKINE STORM, ARDS, AND ACUTE LUNG INJURY USING BETA-GLUCOCEREBROSIDASE
#43THERMOSTABLE GLUCOCEREBROSIDASE
#44CNS DELIVERY OF THERAPEUTIC AGENTS
#45NON-VIRAL DNA VECTORS AND USES THEREOF FOR EXPRESSING GAUCHER THERAPEUTICS
#46VARIANTS OF BETA-GLUCOCEREBROSIDASE FOR USE IN TREATING GAUCHER DISEASE
#47METHODS FOR TREATING FARBER DISEASE
#48INJECTABLE HIGH CONCENTRATION PHARMACEUTICAL FORMULATIONS AND METHODS OF MANUFACTURING AND USE THEREOF
#49TRANSPOSON-BASED MODULATION OF GBA1 AND RELATED COMPOSITIONS AND USES THEREOF
#50Methods and compositions for CNS delivery of iduronate-2-sulfatase
#51Compositions and methods for treatment of GCase related disease states
#52Glycosylated lysosomal proteins, method of production and uses
#53POLYPEPTIDE-FC CONJUGATE WITH ATTENUATED IMMUNE RESPONSE
#54GLUCOCEREBROSIDASE MUTANTS
#55VIRAL PARTICLES FOR USE IN TREATING SYNUCLEINOPATHIES SUCH AS PARKINSON'S DISEASES BY GENE THERAPY
#56GENE THERAPIES FOR LYSOSOMAL DISORDERS
#57ICAM-1 targeted fusion enzymes
#58POLYNUCLEOTIDES
#59TREATMENT OF SANFILIPPO SYNDROME TYPE B
#60Methods and compositions for CNS delivery of heparan N-sulfatase
#61Modified recombinant lysosomal alpha-galactosidase A and aspartylglucoaminidase having low mannose-6-phosphate and high sialic acid
#62GLUCOCEREBROSIDASE POLYPEPTIDES
#63RECOMBINANT ADENO-ASSOCIATED VIRUS COMPOSITIONS AND METHODS FOR PRODUCING SAME
#64Methods and compositions for the treatment of cytoplasmic glycogen storage disorders
#65METHODS FOR TREATING SYMPTOMS AND DISORDERS ASSOCIATED WITH LYSOSOMAL STORAGE DISEASES
#66COMPOSITIONS AND METHODS FOR TREATING LEWY BODY DEMENTIA
#67GENE THERAPIES FOR LYSOSOMAL DISORDERS
#68METHODS AND COMPOSITIONS FOR TREATING CYTOKINE STORM, ARDS, AND ACUTE LUNG INJURY USING BETA-GLUCOCEREBROSIDASE
#69GLUCOCEREBROSIDASE GENE THERAPY
#70Methods for treating neurological symptoms associated with lysosomal storage diseases
#71Methods and compositions for the treatment of steatosis-associated disorders
#72INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
#73DELIVERING ENZYME USING AN INJECTABLE HYDROGEL DEPOT
#74Fusion proteins comprising enzyme replacement therapy enzymes
#75STRUCTURE, MANUFACTURING AND USES OF HUMAN-DERIVED CELL-PERMEABLE PEPTIDES CONJUGATED WITH SPECIFIC BIOLOGICALLY ACTIVE CARGO PEPTIDES
#76ICAM-1 targeted fusion enzymes
#77TREATMENT OF GAUCHER DISEASE
#78Gene therapies for lysosomal disorders
#79COMPOSITIONS AND METHODS FOR TREATING PARKINSON'S DISEASE
#80Methods and compositions for CNS delivery of arylsulfatase A
#81Compositions and methods for treatment of GCASE related disease states
#82Gene therapies for lysosomal disorders
#83Gene therapy for neurodegenerative disorders
#84Integrated continuous manufacturing of therapeutic protein drug substances
#85Gene therapies for lysosomal disorders
#86Gene therapies for lysosomal disorders
#87FUSION PROTEINS COMPRISING ENZYME REPLACEMENT THERAPY ENZYMES
#88Gene therapies for lysosomal disorders
#89Method for enhancing folding and transport of misfolded glucocerebrosidase
#90Gene therapies for lysosomal disorders
#91Gene therapies for lysosomal disorders
#92and enrichment strategy targeting lymphocytes derived from vector transduced HSCs for therapy of disorders
#93Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
#94CNS delivery of therapeutic agents
#95Gene therapies for lysosomal disorders
#96Substituted quinazoline compounds and uses thereof for modulating glucocerebrosidase activity
#97Methods and compositions for CNS delivery of iduronate-2-sulfatase
#98SUBSTITUTED 4-METHYL-PYRROLO[1,2-A]PYRIMIDINE-8-CARBOXAMIDE COMPOUNDS AND USES THEREOF FOR MODULATING GLUCOCEREBROSIDASE ACTIVITY
#99Fusion proteins comprising enzyme replacement therapy enzymes
#100METHODS FOR TREATING FARBER DISEASE
#101Methods and compositions for CNS delivery of heparan n-sulfatase
#102Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides
#103Substituted 4-methyl-pyrrolo[1,2-a]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
#104Delivering enzyme using an injectable hydrogel depot
#105ANTIBODY FORMULATIONS CONTAINING AMINO ACIDS
#106INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
#107Substituted quinazoline compounds and uses thereof for modulating glucocerebrosidase activity
#108Methods and compositions for the treatment of steatosis-associated disorders
#109Substituted 4-methyl-pyrrolo[1,2-A]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
#110Method for enhancing folding and transport of misfolded glucocerebrosidase
#111Compositions and methods for treatment of Gcase related disease states
#112Progranulin (PGRN) fragments and derivatives for treatment or alleviation of lysosomal storage diseases
#113Glucocerebrosidase gene therapy for Parkinson's disease
#114STABILIZED COMPOSITIONS OF PROTEINS HAVING A FREE THIOL MOIETY
#115Therapeutic fusion protein comprising an alpha-n-acetylglucosaminidase and a lysosomal targeting moiety
#116Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins and applications thereof
#117Methods and compositions for CNS delivery of arylsulfatase A
#118Enzymatic activity assays for glucocerebrosidase
#119Substituted 4-methyl-pyrrolo[1,2-A]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
#120Glycosylated lysosomal proteins, method of production and uses
#121Lysosome membrane protein 2 (LIMP-2) based peptides and related uses
#122Integrated continuous manufacturing of therapeutic protein drug substances
#123Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
#124Lysosomal protein targeting sequence and therapeutic applications of same
#125METHOD OF MAINTAINING DISEASE STABILITY IN A SUBJECT HAVING GAUCHER'S DISEASE
#126Progranulin (PGRN) and its derivatives for diagnosis and treatment of lysosomal storage diseases
#127CNS delivery of therapeutic agents
#128Methods of Producing a Secreted Protein
#129GLYCOPEGYLATION METHODS AND PROTEINS/PEPTIDES PRODUCED BY THE METHODS
#130Substituted 4-methyl-pyrrolo[1,2-A]pyrimidine-8-carboxamide compounds and uses thereof for modulating glucocerebrosidase activity
#131Substituted quinazoline compounds and uses thereof for modulating glucocerebrosidase activity
#132Method for enhancing folding and transport of misfolded glucocerebrosidase
#133Method for producing protein in plants using lighting with at least 50% red light
#134Protein Formulations Containing Amino Acids
#135Lysosomal protein targeting sequence and therapeutic applications of same
#136Treatment of synucleinopathies
#137Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
#138Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides
#139PLANT CELL CULTURE EXPRESSING HUMAN LYSOSOMAL PROTEINS AND USES THEREOF
#140Use of Polysaccharides for Promotion of Enzymatic Activity
#141GLYCOPEGYLATION METHODS AND PROTEINS/PEPTIDES PRODUCED BY THE METHODS
#142Intraventricular enzyme delivery for lysosomal storage diseases
#143COMPOSITIONS AND METHODS FOR TREATING PROTEINOPATHIES
#144Stabilized compositions of proteins having a free thiol moiety
#145Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
#146Methods for introducing mannose 6 phosphate and other oligosaccharides onto glycoproteins and applications thereof
#147Integrated continuous manufacturing of therapeutic protein drug substances
#148Method of producing recombinant proteins with mannose-terminated N-glycans
#149Gene therapy for lysosomal storage diseases
#150Compositions and methods for treating type III gaucher disease
#151COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALTERED a-SYNUCLEIN FUNCTION
#152ORAL UNIT DOSAGE FORMS AND USES OF SAME FOR THE TREATMENT OF GAUCHER DISEASE
#153Internally functionalized graphene substrates
#154Vector containing multiple nucleotide sequences for the expression of enzymes
#155Plant cell culture expressing human lysosomal proteins and uses thereof
#156Integrated continuous manufacturing of therapeutic protein drug substances
#157Compositions and methods for treating Gaucher disease
#158Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
#159Variant, recombinant beta-glucocerebrosidase proteins with increased stability and increased retained catalytic activity
#160Treatment of Sanfilippo syndrome type B by intrathecal administration of alpha-n-acetylglucosaminidase
#161Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof
#162Microorganisms genetically engineered to have modified N-glycosylation activity
#163Microorganisms genetically engineered to have modified N-glycosylation activity
#164Method of producing recombinant proteins with mannose-terminated N-glycans
#165COMPOSITIONS AND METHODS FOR PRODUCING ANTIGEN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS WITH SYNTHETIC NANOCARRIERS
#166INDUCED TOLEROGENIC DENDRITIC CELLS FOR INDUCING REGULATORY B CELLS
#167COMPOSITIONS AND METHODS RELATED TO INDUCED TOLEROGENIC DENDRITIC CELLS EXTERNALLY LOADED WITH MHC CLASS I-RESTRICTED EPITOPES
#168ALLERGEN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS FOR ALLERGY THERAPY
#169ANTIGEN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS TO REDUCE ANTIBODY RESPONSES
#170ANTIGEN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS TO REDUCE CYTOTOXIC T LYMPHOCYTE RESPONSES
#171INDUCED TOLEROGENIC DENDRITIC CELLS TO REDUCE SYSTEMIC INFLAMMATORY CYTOKINES
#172INDUCED TOLEROGENIC DENDRITIC CELLS FOR GENERATING CD8+ REGULATORY T CELLS
#173DENDRITIC CELL SUBSETS FOR GENERATING INDUCED TOLEROGENIC DENDRITIC CELLS AND RELATED COMPOSITIONS AND METHODS
#174TRANSPLANTABLE GRAFT-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS AND METHODS OF USE
#175THERAPEUTIC PROTEIN-SPECIFIC INDUCED TOLEROGENIC DENDRITIC CELLS AND METHODS OF USE
#176Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
#177Human lysosomal proteins from plant cell culture
#178Glycopegylation methods and proteins/peptides produced by the methods
#179COMPOSITIONS AND METHODS FOR THE TREATMENT OF ALTERED ALPHA-SYNUCLEIN FUNCTION
#180METHOD FOR GENERATING HYPOALLERGENIC GLYCOPROTEINS IN MUTATED OR TRANSGENIC PLANTS OR PLANT CELLS, AND MUTATED OR TRANSGENIC PLANTS AND PLANT CELLS FOR GENERATING HYPOALLERGENIC GLYCOPROTEINS
#181Method of treating gaucher disease
#182CNS delivery of therapeutic agents
#183Human lysosomal proteins from plant cell culture
#184Microorganisms genetically engineered to have modified N-glycosylation activity
#185Glycosylation of molecules
#186Human lysosomal proteins from plant cell culture
#187Stabilized compositions of proteins having a free thiol moeity
#188METHOD FOR GENERATING HYPOALLERGENIC GLYCOPROTEINS IN MUTATED OR TRANSGENIC PLANTS OR PLANT CELLS, AND MUTATED OR TRANSGENIC PLANTS AND PLANT CELLS FOR GENERATING HYPOALLERGENIC GLYCOPROTEINS
#189Production of glycosylated polypeptides in micro algae
#190Method for the Production of Human Recombinant Lysosomal Enzymes in a Cereal Endosperm
#191Treatment of synucleinopathies
#192COMPOSITIONS AND METHODS FOR TREATING GAUCHER DISEASE
#193Methods of producing a secreted protein
#194PRODUCTION OF HIGH MANNOSE PROTEINS IN PLANT CULTURE
#195Gene therapy for lysosomal storage diseases
#196Eukaryotic layered vector initiation systems
#197Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
#198Methods of treating lysosomal storage related diseases by gene therapy
#199Glycopegylation methods and proteins/peptides produced by the methods
#200Use polysaccharides for promotion of enzymatic activity
#201Production of high mannose proteins in plant culture and therapeutic uses thereof
#202Intraventricular enzyme delivery for lysosomal storage diseases
#203Method of treating gaucher disease
#204and HAC1 nucleic acids
#205Human lysosomal proteins from plant cell culture
#206Genetic modification of the lung as a portal for gene delivery
#207Stabilized compositions of proteins having a free thiol moiety
#208Partially deglycosylated glucocerebrosidase polypeptide and crystals thereof
#209High mannose proteins and methods of making high mannose proteins
#210Glycopegylation methods and proteins/peptides produced by the methods
#211Production of high mannose proteins in plant culture
#212Methods of treating lysosomal storage related diseases by gene therapy
#213In- seed expression of lysosomal enzymes
#214Glycosylated glucocerebrosidase expression in fungal hosts
#215Production of lysosomal enzymes in plants by transient expression
#216Method for recovering proteins from the interstitial fluid of plant tissues
#217Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
#218Enzymatically active recombinant glucocerebrosidase
#219Enzymatically active recombinant glucocerebrosidase