TW201321515A - 重組人類naglu蛋白質及其用途 - Google Patents

重組人類naglu蛋白質及其用途 Download PDF

Info

Publication number
TW201321515A
TW201321515A TW101137865A TW101137865A TW201321515A TW 201321515 A TW201321515 A TW 201321515A TW 101137865 A TW101137865 A TW 101137865A TW 101137865 A TW101137865 A TW 101137865A TW 201321515 A TW201321515 A TW 201321515A
Authority
TW
Taiwan
Prior art keywords
rhnaglu
naglu
composition
protein
brain
Prior art date
Application number
TW101137865A
Other languages
English (en)
Chinese (zh)
Inventor
Anthony Quinn
Markley C Leavitt
Zhinan Xia
Joseph Victor Rutkowski
Original Assignee
Synageva Biopharma Corp
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Synageva Biopharma Corp filed Critical Synageva Biopharma Corp
Publication of TW201321515A publication Critical patent/TW201321515A/zh

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46Hydrolases (3)
    • A61K38/47Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7024Esters of saccharides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/0012Galenical forms characterised by the site of application
    • A61K9/0019Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/02Stomatological preparations, e.g. drugs for caries, aphtae, periodontitis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/12Antidiarrhoeals
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P17/00Drugs for dermatological disorders
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00Drugs for skeletal disorders
    • A61P19/02Drugs for skeletal disorders for joint disorders, e.g. arthritis, arthrosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/20Hypnotics; Sedatives
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P37/00Drugs for immunological or allergic disorders
    • A61P37/02Immunomodulators
    • A61P37/06Immunosuppressants, e.g. drugs for graft rejection
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/06Linear peptides containing only normal peptide links having 5 to 11 amino acids
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K7/00Peptides having 5 to 20 amino acids in a fully defined sequence; Derivatives thereof
    • C07K7/04Linear peptides containing only normal peptide links
    • C07K7/08Linear peptides containing only normal peptide links having 12 to 20 amino acids
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/24Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2474Hyaluronoglucosaminidase (3.2.1.35), i.e. hyaluronidase
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12YENZYMES
    • C12Y302/00Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
    • C12Y302/01Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
    • C12Y302/0105Alpha-N-acetylglucosaminidase (3.2.1.50)

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • General Engineering & Computer Science (AREA)
  • Immunology (AREA)
  • Biomedical Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biophysics (AREA)
  • Dermatology (AREA)
  • Hematology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Diabetes (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Transplantation (AREA)
  • Obesity (AREA)
  • Rheumatology (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Anesthesiology (AREA)
TW101137865A 2011-10-12 2012-10-12 重組人類naglu蛋白質及其用途 TW201321515A (zh)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201161546248P 2011-10-12 2011-10-12

Publications (1)

Publication Number Publication Date
TW201321515A true TW201321515A (zh) 2013-06-01

Family

ID=48082738

Family Applications (1)

Application Number Title Priority Date Filing Date
TW101137865A TW201321515A (zh) 2011-10-12 2012-10-12 重組人類naglu蛋白質及其用途

Country Status (18)

Country Link
US (3) US9579366B2 (enExample)
EP (1) EP2766477B1 (enExample)
JP (1) JP6329483B2 (enExample)
KR (1) KR20140108519A (enExample)
CN (1) CN103958676A (enExample)
AU (1) AU2012322811A1 (enExample)
BR (1) BR112014008873A2 (enExample)
CA (1) CA2852027A1 (enExample)
CL (1) CL2014000927A1 (enExample)
CO (1) CO7010783A2 (enExample)
HK (1) HK1200489A1 (enExample)
IL (1) IL232073A0 (enExample)
IN (1) IN2014MN00740A (enExample)
MX (1) MX2014004359A (enExample)
RU (1) RU2014117291A (enExample)
SG (2) SG10201510106PA (enExample)
TW (1) TW201321515A (enExample)
WO (1) WO2013055888A2 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107002095A (zh) * 2014-05-14 2017-08-01 埃斯蒂维实验室股份有限公司 用于治疗溶酶体贮积症的腺伴随病毒载体

Families Citing this family (21)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US8497246B2 (en) 2006-08-18 2013-07-30 Armagen Technologies, Inc. Methods for diagnosing and treating CNS disorders by trans-blood-brain barrier delivery of protein compositions
JP5901877B2 (ja) 2007-07-27 2016-04-13 アーメイゲン・テクノロジーズ・インコーポレイテッドArmagen Technologies, Inc. 中枢神経系のα−L−イデュロニダーゼ活性を増加させるための方法および組成物
ES2725200T3 (es) 2009-10-09 2019-09-20 Armagen Inc Métodos y composiciones para aumentar la actividad de iduronato 2-sulfatasa en el SNC
NZ715014A (en) 2010-09-09 2018-10-26 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
AU2012322811A1 (en) * 2011-10-12 2014-05-01 Synageva Biopharma Corp. Recombinant human NaGlu protein and uses thereof
EP4338797A3 (en) 2011-12-02 2024-06-12 Armagen, Inc. Methods and compositions for increasing arylsulfatase a activity in the cns
CA2912678C (en) 2013-05-15 2023-10-10 Regents Of The University Of Minnesota Adeno-associated virus mediated gene transfer to the central nervous system
TWI793159B (zh) * 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
US20170267986A1 (en) * 2014-06-25 2017-09-21 Shire Human Genetic Therapies, Inc. Generation of mannose-6-phosphate containing recombinant alpha-n-acetyl glucosaminidase
BR112017002741A2 (pt) 2014-08-11 2018-02-27 Shire Human Genetic Therapies, Inc peptídeos contendo manose-6-fosfato fundidos a enzimas lisossômicas
AU2015323966A1 (en) 2014-09-29 2017-03-16 Alexion Pharmaceuticals, Inc. Methods of treating mucopolysaccharidosis IIIB (MPSIIIB)
CN104524662B (zh) * 2014-12-31 2017-02-22 迈得医疗工业设备股份有限公司 一种医用针座组装的上料机构
US10538589B2 (en) * 2015-01-14 2020-01-21 Armagen Inc. Methods and compositions for increasing N-acetylglucosaminidase (NAGLU) activity in the CNS using a fusion antibody comprising an anti-human insulin receptor antibody and NAGLU
US20190083583A1 (en) * 2016-01-29 2019-03-21 Alexion Pharmaceuticals, Inc. Enzyme replacement therapy in mucopolysaccharidosis iiib patients
WO2017181113A1 (en) 2016-04-15 2017-10-19 The Trustees Of The University Of Pennsyvania Gene therapy for treating mucopolysaccharidosis type ii
US10633705B2 (en) * 2016-10-14 2020-04-28 Alexion Pharmaceuticals, Inc. N-acetyl-alpha-D-glucosaminidase deficiency compositions and methods
WO2019060662A1 (en) 2017-09-22 2019-03-28 The Trustees Of The University Of Pennsylvania GENE THERAPY FOR THE TREATMENT OF MUCOPOLYSACCHARIDOSE TYPE II
MA50746A (fr) 2017-10-02 2020-08-12 Denali Therapeutics Inc Protéines de fusion comprenant des enzymes d'enzymothérapie substitutive
WO2021194915A1 (en) * 2020-03-23 2021-09-30 The University Of North Carolina At Chapel Hill Aav-naglu vectors for treatment of mucopolysaccharidosis iiib
CN111718947B (zh) * 2020-06-18 2022-08-23 舒泰神(北京)生物制药股份有限公司 用于治疗ⅲa或ⅲb型粘多糖贮积症的腺相关病毒载体及用途

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6825396B2 (en) 1996-06-12 2004-11-30 Board Of Trustees Operating Michigan State University Methods for tissue specific synthesis of protein in eggs of transgenic hens
US5897998A (en) 1997-08-04 1999-04-27 The University Of Georgia Research Foundation, Inc. Method for manipulating avian eggs
US7511120B2 (en) 1997-10-16 2009-03-31 Synageva Biopharma Corp. Glycosylated G-CSF obtained from a transgenic chicken
JP2001520009A (ja) 1997-10-16 2001-10-30 ユニバーシティ・オブ・ジョージア・リサーチ・ファウンデイション・インコーポレイテッド 鳥の導入遺伝子についてのマグナム特異的プロモーターを含むベクター
US20040019923A1 (en) 1997-10-16 2004-01-29 Ivarie Robert D. Exogenous proteins expressed in avians and their eggs
US6217552B1 (en) 1999-03-01 2001-04-17 Coaxia, Inc. Medical device for selective intrathecal spinal cooling in aortic surgery and spinal trauma
US6534300B1 (en) 1999-09-14 2003-03-18 Genzyme Glycobiology Research Institute, Inc. Methods for producing highly phosphorylated lysosomal hydrolases
US7138262B1 (en) 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
US7001994B2 (en) 2001-01-18 2006-02-21 Genzyme Corporation Methods for introducing mannose 6-phosphate and other oligosaccharides onto glycoproteins
US7294507B2 (en) 2001-11-30 2007-11-13 Avigenics, Inc. Ovomucoid promoters and methods of use
US6875588B2 (en) 2001-11-30 2005-04-05 Avigenics, Inc. Ovomucoid promoter and methods of use
US6679165B2 (en) 2002-05-22 2004-01-20 Shiny Shih Window adapted to a dater
US20050142141A1 (en) * 2002-11-27 2005-06-30 Pardridge William M. Delivery of enzymes to the brain
EP1587923B1 (en) * 2003-01-22 2011-08-24 Duke University Improved constructs for expressing lysosomal polypeptides
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20050090001A1 (en) 2003-10-07 2005-04-28 Parker Stephen H. Cell lines and methods for producing proteins
US20050208090A1 (en) * 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
US20090166224A1 (en) * 2004-05-05 2009-07-02 Ziping Yang Multi-lectin affinity chromatography and uses thereof
EP1981546B1 (en) 2006-02-06 2014-06-04 The Medical Research and Infrastructure Fund of the Tel-Aviv Sourasky Medical Center Enzyme replacement therapy for treating lysosomal storage diseases
CN101410408A (zh) 2006-04-04 2009-04-15 希尔制药爱尔兰有限责任公司 用于浓缩多肽的方法
CA2671570A1 (en) 2007-01-26 2008-07-31 Synageva Biopharma Corp. Transgene expression in avians
US8431770B2 (en) * 2008-01-07 2013-04-30 Synageva Biopharma Corp. Method of producing sialytransferase-modified proteins
WO2009131698A2 (en) * 2008-04-23 2009-10-29 Iowa State University Research Foundation, Inc. PHOSPHORYLATED RECOMBINANT N-ACETYL-alpha-D- GLUCOSAMINIDASE (NaGlu) AND USES THEREOF
ES2576853T3 (es) * 2009-07-03 2016-07-11 Inserm - Institut National De La Santé Et De La Recherche Médicale Compuestos dirigidos al receptor del manosa 6-fosfato independiente de cationes
UA115648C2 (uk) * 2010-06-25 2017-12-11 Шае Хюмен Дженетік Терапіс, Інк. Доставка терапевтичних агентів до цнс
NZ702801A (en) 2010-06-25 2016-08-26 Shire Human Genetic Therapies Treatment of sanfilippo syndrome type b
WO2012012742A2 (en) * 2010-07-22 2012-01-26 Shire Human Genetic Therapies, Inc. Crystal structure of human alpha-n-acetylglucosaminidase
NZ715014A (en) 2010-09-09 2018-10-26 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
AU2012322811A1 (en) * 2011-10-12 2014-05-01 Synageva Biopharma Corp. Recombinant human NaGlu protein and uses thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN107002095A (zh) * 2014-05-14 2017-08-01 埃斯蒂维实验室股份有限公司 用于治疗溶酶体贮积症的腺伴随病毒载体

Also Published As

Publication number Publication date
BR112014008873A2 (pt) 2020-09-15
SG10201510106PA (en) 2016-01-28
US9579366B2 (en) 2017-02-28
NZ623690A (en) 2016-08-26
WO2013055888A3 (en) 2013-06-13
CN103958676A (zh) 2014-07-30
RU2014117291A (ru) 2015-11-20
US20170216412A1 (en) 2017-08-03
CA2852027A1 (en) 2013-04-18
CO7010783A2 (es) 2014-07-31
WO2013055888A2 (en) 2013-04-18
AU2012322811A1 (en) 2014-05-01
KR20140108519A (ko) 2014-09-11
EP2766477B1 (en) 2018-09-26
HK1200489A1 (en) 2015-08-07
IN2014MN00740A (enExample) 2015-05-22
CL2014000927A1 (es) 2014-11-14
US20140255383A1 (en) 2014-09-11
JP2014534962A (ja) 2014-12-25
JP6329483B2 (ja) 2018-05-23
IL232073A0 (en) 2014-05-28
MX2014004359A (es) 2014-05-28
US20130095092A1 (en) 2013-04-18
SG11201401457SA (en) 2014-05-29
EP2766477A4 (en) 2015-07-15
EP2766477A2 (en) 2014-08-20

Similar Documents

Publication Publication Date Title
JP6329483B2 (ja) 組換えヒトnagluタンパク質およびその利用
CN105214076B (zh) 溶酶体贮积病酶
IL255939A (en) Adenosocyte virus vectors for the treatment of mycopopolysaccharides
JP2018509164A (ja) 組換えGlut1アデノ随伴ウイルスベクターコンストラクトおよびGlut1発現を回復させる方法
KR101535791B1 (ko) 개량형 이듀로네이트-2-설파타제 및 이의 용도
JP2025087702A (ja) ヒト神経細胞又はグリア細胞により生産された組換えヒトイズロン酸-2-スルファターゼ(ids)によるムコ多糖症ii型の治療
IL268076B1 (en) Treatment of mucopolysaccharidoses I with human iduronidase L (IDUA) and full human glycosylations
CA3052520C (en) Mutated arylsulfatase a
US20190083583A1 (en) Enzyme replacement therapy in mucopolysaccharidosis iiib patients
US20170216413A1 (en) Methods of treating mucopolysaccharidosis iiib (mpsiiib)
KR20210034037A (ko) 완전-인간 글리코실화된 인간 알파-l-이두로니다아제 (idua)를 사용한 점액다당류증 i의 치료
US20230366887A1 (en) Methods of detecting and using biomarkers for glycogen storage diseases
NZ623690B2 (en) Recombinant human naglu protein and uses thereof
CA2978917C (en) Recombinant glut1 adeno-associated viral vector constructs and related methods for restoring glut1 expression
HK1183059B (en) Lysosomal storage disease enzyme