116569 ⎘
Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2); Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1) L-Iduronidase (3.2.1.76)
COMPOSITIONS AND METHODS COMPRISING PROGRAMMABLE SNRNAS FOR RNA EDITING
#2AAV-IDUA VECTOR FOR TREATMENT OF MPS I
#3OLIGOSACCHARIDES COMPRISING AN AMINOOXY GROUP AND CONJUGATES THEREOF
#4GLYCOPROTEIN HAVING REDUCED NUMBER OF MANNOSE-6-PHOSPHATES
#5PLANT LECTINS AS CARRIERS OF ASSOCIATED DRUG SUBSTANCES INTO ANIMAL AND HUMAN CELLS
#6IDURONIDASE STABILIZERS AND USES THEREOF
#7COMPOSITIONS AND METHODS FOR INTERNALIZING ENZYMES
#8METHODS FOR TREATING INHERITED METABOLIC DISORDERS
#9Lyophilized Pharmaceutical Compositions Comprising A Lipid Nanoparticle
#10FUSION PROTEINS COMPRISING ALPHA-L-IDURONIDASE ENZYMES AND METHODS
#11NON-VIRAL B CELL ENGINEERING METHODS
#12CD40L ANTAGONIST AND USES THEREOF
#13METHODS AND COMPOSITIONS FOR INCREASING N-ACETYLGLUCOSAMINIDASE ACTIVITY IN THE CNS
#14Lyophilized Pharmaceutical Compositions Comprising a Lipid Nanoparticle
#15Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders
#16METHODS FOR PREVENTING CARDIAC OR SKELETAL DEFECTS IN DISEASES INCLUDING MUCOPOLYSACCHARIDOSES
#17AAV9-MEDIATED GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
#18Pharmaceutical Composition for Treatment of Mucopolysaccharidosis Type 1
#19Method for Producing Antibody-Lysosomal Enzyme Fusion Protein
#20TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY-HUMAN GLYCOSYLATED HUMAN ALPHA-L-IDURONIDASE (IDUA)
#21METHODS FOR THE IMPROVED RECOVERY OF SECRETED COMPOUNDS IN A HAIRY ROOTS-BASED EXPRESSION SYSTEM
#22GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
#23IN UTERO AND POSTNATAL GENE EDITING AND THERAPY FOR TREATMENT OF MONOGENIC DISEASES, INCLUDING MUCOPOLYSACCHARIDOSIS TYPE 1H AND OTHER DISORDERS
#24METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEUROLOGIC DISEASE
#25IMPLANTABLE CELL CHAMBER DEVICE AND USES THEREOF
#26MATERIALS AND METHODS FOR MITIGATING IMMUNE-SENSITIZATION
#27Methods and compositions for the treatment of steatosis-associated disorders
#28Methods and Compositions for the Treatment of Steatosis-Associated Disorders
#29METHODS AND COMPOSITIONS FOR INCREASING ALPHA-L-IDURONIDASE ACTIVITY IN THE CNS
#30TARGETING THE HUMAN CCR5 LOCUS AS A SAFE HARBOR FOR THE EXPRESSION OF THERAPEUTIC PROTEINS
#31ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#32COMBINATION THERAPY FOR TREATING MPS1
#33Plant lectins as carriers of associated drug substances into animal and human cells
#34ENGINEERED TARGET SPECIFIC NUCLEASES
#35COMPOSITIONS FOR DRG-SPECIFIC REDUCTION OF TRANSGENE EXPRESSION
#36GENE THERAPY FOR MUCOPOLYSACCHARIDOSIS, TYPE I
#37INTRANASAL ADMINISTRATION OF ADENO-ASSOCIATED VIRUS TO TREAT CENTRAL NERVOUS SYSTEM DISORDERS
#38OLIGOSACCHARIDES COMPRISING AN AMINOOXY GROUP AND CONJUGATES THEREOF
#39Cleavable lipids
#40B CELLS FOR IN VIVO DELIVERY OF THERAPEUTIC AGENTS AND DOSAGES THEREOF
#41METHOD FOR IMPROVING NEUROLOGICAL FUNCTION IN MPSI AND MPSII AND OTHER NEUROLOGICAL DISORDERS
#42Anti-Human Transferrin Receptor Antibody Permeating Blood-Brain Barrier
#43AAV-IDUA vector for treatment of MPS I-associated blindness
#44Methods and compositions for the treatment of cytoplasmic glycogen storage disorders
#45ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#46TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY-HUMAN GLYCOSYLATED HUMAN ALPHA-L-IDURONIDASE (IDUA)
#47Methods and compositions for the treatment of steatosis-associated disorders
#48GENE THERAPY FOR TREATING MUCOPOLYSACCHARIDOSIS TYPE I
#49INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
#50DELIVERING ENZYME USING AN INJECTABLE HYDROGEL DEPOT
#51STRUCTURE, MANUFACTURING AND USES OF HUMAN-DERIVED CELL-PERMEABLE PEPTIDES CONJUGATED WITH SPECIFIC BIOLOGICALLY ACTIVE CARGO PEPTIDES
#52COMPOSITIONS AND METHODS FOR TREATING MPSI
#53Method for the treatment of mucopolysaccharidosis type I
#54Cleavable lipids
#55Methods and compositions for the treatment of lysosomal storage diseases
#56USE OF P97 AS AN ENZYME DELIVERY SYSTEM FOR THE DELIVERY OF THERAPEUTIC LYSOSOMAL ENZYMES
#57AAV9-mediated gene therapy for treating mucopolysaccharidosis type I
#58POLYPEPTIDES
#59Oligosaccharides comprising an aminooxy group and conjugates thereof
#60Methods and compositions for increasing alpha-L-iduronidase activity in the CNS
#61TREATMENT OF MUCOPOLYSACCHARIDOSIS I WITH FULLY-HUMAN GLYCOSYLATED HUMAN ALPHA-L-IDURONIDASE (IDUA)
#62Cleavable lipids
#63ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#64METHOD FOR IMPROVING NEUROLOGICAL FUNCTION IN MPSI AND MPSII AND OTHER NEUROLOGICAL DISORDERS
#65Engineered target specific nucleases
#66Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides
#67Delivering enzyme using an injectable hydrogel depot
#68Compositions and methods for treating MPSI
#69INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES
#70AAV-IDUA vector for treatment of MPS I-associated blindness
#71MATERIALS AND METHODS FOR MITIGATING IMMUNE-SENSITIZATION
#72Methods and compositions for the treatment of steatosis-associated disorders
#73Gene therapy for treating mucopolysaccharidosis type I
#74Engineered enzyme for enzyme replacement therapy
#75INTRANASAL THERAPEUTIC DELIVERY OF ADENO-ASSOCIATED VIRUS TO CENTRAL NERVOUS SYSTEM
#76METHODS TO TREAT CEROID LIPOFUSCINOSIS USING A RECOMBINANT ADENO-ASSOCIATED VIRUS
#77METHODS TO TREAT POMPE DISEASE USING A RECOMBINANT ADENO-ASSOCIATED VIRUS
#78ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#79ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#80MODIFIED LYSOSOMAL PROTEIN AND PRODUCTION THEREOF
#81THERAPEUTIC COMPOSITIONS OF ALPHA-L-IDURONIDASE, IDURONATE-2-SULFATASE, AND ALPHA-GALACTOSIDASE A AND METHODS OF USE THEREOF
#82Anti-human transferrin receptor antibody permeating blood-brain barrier
#83Cleavable lipids
#84Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
#85Methods to treat mucopolysaccharidosis type II or deficiency in iduronate-2-sulfatase using a recombinant adeno-associated virus (AAV) vector encoding iduronate-2-sulfatase
#86Intranasal administration of guanidinylated aminoglycosides
#87ADENO-ASSOCIATED VIRUS FOR THERAPEUTIC DELIVERY TO CENTRAL NERVOUS SYSTEM
#88ADENO-ASSOCIATED VIRUS MEDIATED GENE TRANSFER TO THE CENTRAL NERVOUS SYSTEM
#89Use of P97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
#90Cleavable Lipids
#91Plant lectins as carriers of associated drug substances into animal and human cells
#92METHODS AND COMPOSITIONS FOR THE TREATMENT OF NEUROLOGIC DISEASE
#93Treatment of lysosomal storage diseases
#94Methods and compositions for increasing α-L-iduronidase activity in the CNS
#95Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
#96Structure, manufacturing and uses of human-derived cell-permeable peptides conjugated with specific biologically active cargo peptides
#97Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
#98Methods to treat mucopolysaccharide type I or deficiency in alpha-L-iduronidase using a recombinant adeno-associated virus encoding alpha-L-iduronidase
#99Plant lectins as carriers of associated drug substances into animal and human cells
#100Method of treating mucopolysaccharidosis type I or II
#101Intranasal administration of guanidinylated aminoglycosides
#102Compositions and methods for treating MPSI
#103Methods for assaying alpha-L-iduronidase enzymatic activity
#104Intraventricular enzyme delivery for lysosomal storage diseases
#105Methods and compositions for increasing α-L-iduronidase activity in the CNS
#106Enhancing Vegetative Protein Production in Transgenic Plants Using Seed Specific Promoters
#107TARGETED ENZYME COMPOUNDS AND USES THEREOF
#108Assay for measuring enzymatic modification of a substrate by a glycoprotein having enzymatic activity
#109Oligosaccharides comprising an aminooxy group and conjugates thereof
#110Cleavable lipids
#111Targeted delivery of proteins across the blood-brain barrier
#112Intranasal delivery of AAV encoding therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
#113Treatment of alpha-L-iduronidase (IDUA) related diseases by inhibition of natural antisense transcript to IDUA
#114Intranasal delivery of therapeutic enzymes to the central nervous system for the treatment of lysosomal storage diseases
#115Methods for assaying α-L-iduronidase enzymatic activity
#116Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
#117Methods for assaying alpha-L-iduronidase enzymatic activity
#118Methods of treating lysosomal storage related diseases by gene therapy
#119Oligosaccharides comprising an aminooxy group and conjugates thereof
#120Enhancing vegetative protein production in transgenic plants using seed specific promoters
#121Intraventricular enzyme delivery for lysosomal storage diseases
#122Methods and compositions for increasing α-L-iduronidase activity in the CNS
#123Production Of A Protein Localized To The Endoplasmic Reticulum Protein Bodies In A Transgenic Plant And Seed
#124Genetic modification of the lung as a portal for gene delivery
#125MAPC administration for the treatment of lysosomal storage disorders
#126Recombinant alpha-L-iduronidase, methods for producing and purifying the same and methods for treating diseases caused by deficiencies thereof
#127Vector for expressing alpha-N-acetyl-galactosaminidase and method of treating MPS I by stereotactic injection into the brain of a mammal
#128Methods of treating lysosomal storage related diseases by gene therapy
#129Methods for producing and purifying recombinant alpha-L-iduronidase
#130Methods of treating diseases caused by deficiencies of recombinant α-L-iduronidase
#131Methods for treating diseases caused by deficiencies of recombinant alpha-L-iduronidase
#132Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
#133Delivery of enzymes to the brain
#134Compounds and methods for assaying activity of lysosomal storage diseases