WO1998011206A3 - THERAPY FOR α-GALACTOSIDASE A DEFICIENCY - Google Patents

THERAPY FOR α-GALACTOSIDASE A DEFICIENCY Download PDF

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Publication number
WO1998011206A3
WO1998011206A3 PCT/US1997/016603 US9716603W WO9811206A3 WO 1998011206 A3 WO1998011206 A3 WO 1998011206A3 US 9716603 W US9716603 W US 9716603W WO 9811206 A3 WO9811206 A3 WO 9811206A3
Authority
WO
WIPO (PCT)
Prior art keywords
galactosidase
deficiency
therapy
human
gal
Prior art date
Legal status (The legal status 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 status listed.)
Ceased
Application number
PCT/US1997/016603
Other languages
French (fr)
Other versions
WO1998011206A9 (en
WO1998011206A2 (en
Inventor
Richard F Selden
Marianne Borowski
Frances P Gillespie
Carol M Kinoshita
Douglas A Treco
Melanie D Williams
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Shire Human Genetics Therapies Inc
Original Assignee
Transkaryotic Therapies Inc
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
Priority to AT97942567T priority Critical patent/ATE286120T1/en
Priority to EP97942567A priority patent/EP0935651B1/en
Priority to IL128960A priority patent/IL128960A/en
Priority to CA002265464A priority patent/CA2265464C/en
Priority to HU1000445A priority patent/HU230275B1/en
Priority to JP51400498A priority patent/JP4001925B2/en
Priority to AU44244/97A priority patent/AU4424497A/en
Priority to HU9904666A priority patent/HU227189B1/en
Priority to NZ334721A priority patent/NZ334721A/en
Priority to DE69732129T priority patent/DE69732129T2/en
Application filed by Transkaryotic Therapies Inc filed Critical Transkaryotic Therapies Inc
Priority to PL97332188A priority patent/PL190041B1/en
Priority to HK00101063.0A priority patent/HK1022173B/en
Priority to MX2014010892A priority patent/MX340738B/en
Publication of WO1998011206A2 publication Critical patent/WO1998011206A2/en
Publication of WO1998011206A9 publication Critical patent/WO1998011206A9/en
Publication of WO1998011206A3 publication Critical patent/WO1998011206A3/en
Priority to US09/266,014 priority patent/US6458574B1/en
Priority to NO19991225A priority patent/NO328443B1/en
Anticipated expiration legal-status Critical
Priority to US10/165,060 priority patent/US20030077806A1/en
Priority to US10/165,968 priority patent/US20030113894A1/en
Priority to IL184637A priority patent/IL184637A/en
Priority to NO20090583A priority patent/NO330687B1/en
Priority to US12/944,688 priority patent/US20110280856A1/en
Priority to NO20110496A priority patent/NO340408B1/en
Priority to NO20141168A priority patent/NO20141168L/en
Ceased legal-status Critical Current

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Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/14—Prodigestives, e.g. acids, enzymes, appetite stimulants, antidyspeptics, tonics, antiflatulents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P13/00—Drugs for disorders of the urinary system
    • A61P13/12—Drugs for disorders of the urinary system of the kidneys
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/04—Centrally acting analgesics, e.g. opioids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/575—Hormones
    • C07K14/61—Growth hormone [GH], i.e. somatotropin
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14—Hydrolases (3)
    • C12N9/24—Hydrolases (3) acting on glycosyl compounds (3.2)
    • C12N9/2402—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1)
    • C12N9/2465—Hydrolases (3) acting on glycosyl compounds (3.2) hydrolysing O- and S- glycosyl compounds (3.2.1) acting on alpha-galactose-glycoside bonds, e.g. alpha-galactosidase (3.2.1.22)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide

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  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Cardiology (AREA)
  • Biotechnology (AREA)
  • Neurosurgery (AREA)
  • Pain & Pain Management (AREA)
  • General Engineering & Computer Science (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Obesity (AREA)
  • Microbiology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Epidemiology (AREA)
  • Immunology (AREA)
  • Hospice & Palliative Care (AREA)
  • Biophysics (AREA)

Abstract

A therapeutic method whereby an individual suspected of having an α-galactosidase A deficiency, such as Fabry disease, is treated either with (1) human cells that have been genetically modified to overexpress and secrete human α-gal A, or (2) purified human α-gal A obtained from cultured, genetically modified human cells.
PCT/US1997/016603 1996-09-12 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY Ceased WO1998011206A2 (en)

Priority Applications (22)

Application Number Priority Date Filing Date Title
NZ334721A NZ334721A (en) 1996-09-13 1997-09-12 Purified human alpha-galactosidase A and therapeutic use
IL128960A IL128960A (en) 1996-09-13 1997-09-12 Expression and purification of glycosylated recombinant human ?? - galactosidase a and use thereof in the preparation of medicament
CA002265464A CA2265464C (en) 1996-09-13 1997-09-12 Therapy for .alpha.-galactosidase a deficiency
HU1000445A HU230275B1 (en) 1996-09-13 1997-09-12 Method for producing purified human alpha galactosidase a compositions; pharmaceutical compositions comprising said purified compositions for use in treating disorders originating from alpha galactosidase a deficiency
JP51400498A JP4001925B2 (en) 1996-09-13 1997-09-12 Treatment of α-galactosidase A deficiency
AU44244/97A AU4424497A (en) 1996-09-13 1997-09-12 Therapy for alpha-galactosidase a deficiency
HU9904666A HU227189B1 (en) 1996-09-13 1997-09-12 Therapy for alpha-galactosidase a deficiency
AT97942567T ATE286120T1 (en) 1996-09-13 1997-09-12 THERAPY FOR ALPHA GALACTOSIDASE A INSUFFICIENCY
EP97942567A EP0935651B1 (en) 1996-09-13 1997-09-12 THERAPY FOR alpha-GALACTOSIDASE A DEFICIENCY
DE69732129T DE69732129T2 (en) 1996-09-13 1997-09-12 THERAPY FOR alpha GALACTOSIDASE A INSUFFICIENCY
PL97332188A PL190041B1 (en) 1996-09-13 1997-09-12 Therapy against alpha-galactosidase a deficiency
HK00101063.0A HK1022173B (en) 1996-09-13 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY
MX2014010892A MX340738B (en) 1996-09-13 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY.
US09/266,014 US6458574B1 (en) 1996-09-12 1999-03-11 Treatment of a α-galactosidase a deficiency
NO19991225A NO328443B1 (en) 1996-09-13 1999-03-12 DNA molecule, expression construct, cultured human cell, clone cell strain, clone cell line, protein, process for producing glycosylated human alpha-galactosidase A and human alpha-galactosidase A, and use of a human cell that is genetically modified.
US10/165,060 US20030077806A1 (en) 1996-09-13 2002-06-07 Treatment of alpha-galactosidase a deficiency
US10/165,968 US20030113894A1 (en) 1996-09-13 2002-06-10 Treatment of alpha-galactosidase a deficiency
IL184637A IL184637A (en) 1996-09-13 2007-07-16 Process for preparing human alpha galactosidase a (alpha-gal a) and a therapeutic composition comprising the same
NO20090583A NO330687B1 (en) 1996-09-13 2009-02-06 Method for purification of human alpha-galactosidase A
US12/944,688 US20110280856A1 (en) 1996-09-13 2010-11-11 Treatment of alpha-galactosidase a deficiency
NO20110496A NO340408B1 (en) 1996-09-13 2011-03-31 Process for producing human alpha-galactosidase A, and pharmaceutical composition
NO20141168A NO20141168L (en) 1996-09-13 2014-09-29 Method of purifying human alpha-galactosidase A from a sample

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US71261496A 1996-09-13 1996-09-13
US08/712,614 1996-09-13

Related Child Applications (3)

Application Number Title Priority Date Filing Date
US92888196A Continuation-In-Part 1996-09-12 1996-09-12
US08/928,881 Continuation-In-Part US6083725A (en) 1996-09-13 1997-09-12 Tranfected human cells expressing human α-galactosidase A protein
US09/266,014 Continuation-In-Part US6458574B1 (en) 1996-09-12 1999-03-11 Treatment of a α-galactosidase a deficiency

Publications (3)

Publication Number Publication Date
WO1998011206A2 WO1998011206A2 (en) 1998-03-19
WO1998011206A9 WO1998011206A9 (en) 1998-07-02
WO1998011206A3 true WO1998011206A3 (en) 1998-08-13

Family

ID=24862863

Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1997/016603 Ceased WO1998011206A2 (en) 1996-09-12 1997-09-12 THERAPY FOR α-GALACTOSIDASE A DEFICIENCY

Country Status (20)

Country Link
EP (4) EP2327775A3 (en)
JP (7) JP4001925B2 (en)
KR (2) KR20050084473A (en)
CN (1) CN1268741C (en)
AT (1) ATE286120T1 (en)
AU (1) AU4424497A (en)
CA (1) CA2265464C (en)
DE (1) DE69732129T2 (en)
DK (2) DK2374876T3 (en)
ES (3) ES2581828T3 (en)
HU (2) HU227189B1 (en)
IL (2) IL128960A (en)
MX (1) MX340738B (en)
NO (4) NO328443B1 (en)
NZ (2) NZ334721A (en)
PL (1) PL190041B1 (en)
PT (1) PT1538202E (en)
RU (1) RU2179034C2 (en)
TW (1) TW585919B (en)
WO (1) WO1998011206A2 (en)

Cited By (2)

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Publication number Priority date Publication date Assignee Title
US9265780B2 (en) 1998-06-01 2016-02-23 Icahn School Of Medicine At Mount Sinai Method of enhancing lysosomal α-galactosidase A
CN106110309A (en) * 1999-03-11 2016-11-16 夏尔人类遗传性治疗公司 The treatment of α galactoside enzyme defect

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US6083725A (en) 1996-09-13 2000-07-04 Transkaryotic Therapies, Inc. Tranfected human cells expressing human α-galactosidase A protein
US6066626A (en) * 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method for treating lysosomal storage disease
EP1658857A1 (en) * 1997-10-29 2006-05-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
AU2012241170B2 (en) * 1999-03-11 2016-07-28 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2004242550B2 (en) * 1999-03-11 2008-04-03 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
AU2016250357B2 (en) * 1999-03-11 2018-11-01 Shire Human Genetic Therapies, Inc. Treatment of alpha-Galactosidase A deficiency
WO2002064799A2 (en) 1999-09-28 2002-08-22 Transkaryotic Therapies, Inc. Optimized messenger rna
EP2275559A3 (en) * 1999-09-28 2011-03-23 Shire Human Genetic Therapies, Inc. Optimized messenger RNA
AU1647501A (en) 1999-12-10 2001-06-18 Cytos Biotechnology Ag Replicon based activation of endogenous genes
ES2328446T5 (en) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Use of lysosomal acid lipase to treat atherosclerosis and associated diseases
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7138262B1 (en) * 2000-08-18 2006-11-21 Shire Human Genetic Therapies, Inc. High mannose proteins and methods of making high mannose proteins
IL140110A0 (en) * 2000-12-05 2002-02-10 Applied Research Systems Improvement of homogeneity and secretion of recombinant proteins in mammalian systems
BR122017016940B8 (en) 2002-04-25 2021-07-27 Transkaryotic Therapies Inc method for preparing a pharmaceutical composition comprising alpha-galactosidase a
JP5433133B2 (en) * 2003-01-22 2014-03-05 デューク・ユニヴァーシティ Improved construct for expressing lysosomal polypeptides
US7935336B2 (en) 2005-11-18 2011-05-03 Tokyo Metropolitan Organization For Medical Research Highly functional enzyme having α-galactosidase activity
AR059089A1 (en) 2006-01-20 2008-03-12 Genzyme Corp INTRAVENTRICULAR ADMINISTRATION OF AN ENZYME FOR LISOSOMAL STORAGE DISEASES
RS52459B (en) 2006-02-07 2013-02-28 Shire Human Genetic Therapies Inc. STABILIZED GLUCOCEREBROSIDASE COMPOSITIONS
MX361816B (en) 2006-02-09 2018-12-17 Genzyme Corp Slow intraventricular delivery.
EP2166093B1 (en) * 2007-05-18 2015-09-16 Tokyo Metropolitan Institute of Medical Science Pharmaceutical composition for enzyme replacement therapy
PL2889043T3 (en) 2008-12-16 2019-09-30 Genzyme Corporation Synthetic intermediates for preparing oligosaccharide-protein conjugates
NZ623910A (en) 2009-07-28 2016-06-24 Shire Human Genetic Therapies Compositions and methods for treating gaucher disease
MX338426B (en) 2010-04-23 2016-04-15 Synageva Biopharma Corp Lysosomal storage disease enzyme.
EP2595651B1 (en) 2010-07-19 2017-03-29 Shire Human Genetic Therapies, Inc. Mannose receptor c type 1 (mrc1) codon optimized cell line and uses thereof
DK2613798T4 (en) 2010-09-09 2018-04-16 Synageva Biopharma Corp USING LYSOSOMAL SUR LIPASE TO TREAT PATIENTS WITHOUT LYSOSOMAL SUR LIPASE
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp Methods for treating lysosomal acid lipase deficiency
EP3527274B1 (en) * 2011-11-02 2025-12-24 F. Hoffmann-La Roche AG Overload and elute chromatography
US9623090B2 (en) 2012-03-02 2017-04-18 Shire Human Genetic Therapies, Inc. Compositions and methods for treating type III gaucher disease
WO2014001509A1 (en) * 2012-06-29 2014-01-03 Consejo Superior De Investigaciones Científicas (Csic) Functionalized liposomes useful for the delivery of bioactive compounds
WO2014016873A1 (en) 2012-07-26 2014-01-30 Jcr Pharmaceuticals Co., Ltd. Method for production of recombinant human alpha-galactosidase a
JP6226435B2 (en) * 2012-07-26 2017-11-08 Ｊｃｒファーマ株式会社 Method for producing recombinant human α-galactosidase A
CN104884147A (en) 2012-10-24 2015-09-02 建新公司 Elution of Biomolecules from Multimodal Resins Using MES and MOPS as Mobile Phase Modifiers
TWI642782B (en) * 2013-10-23 2018-12-01 健臻公司 Recombinant glycoprotein and its use
US20170360900A1 (en) 2014-12-22 2017-12-21 Codexis, Inc. Human alpha-galactosidase variants
WO2016116966A1 (en) 2015-01-22 2016-07-28 Jcr Pharmaceuticals Co., Ltd. Method for purification of recombinant human alpha-galactosidase a from material containing contaminant host cell proteins
GB201508025D0 (en) * 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
EP3646899B1 (en) 2017-06-30 2024-07-31 FUJIFILM Corporation Lysosomal storage disease treatment agent
ES2716305B2 (en) 2017-12-11 2019-11-27 Fund Biomedica Galicia Sur USE OF PHARMACOLOGICAL CHAPERONS FOR THE TREATMENT OF LISOSOMAL DEPOSIT DISEASES
MX2021007552A (en) 2018-12-20 2021-08-11 Codexis Inc Human alpha-galactosidase variants.
CN111308095A (en) * 2020-03-04 2020-06-19 北京师范大学 Urine protein marker for diagnosing prostate cancer
TW202423959A (en) * 2022-08-24 2024-06-16 美商步行魚醫療公司 Compositions and methods for treatment of fabry disease

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Title
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MEDIN JA ET AL: "Correction in trans for Fabry disease: expression, secretion and uptake of alpha-galactosidase A in patient-derived cells driven by a high-titer recombinant retroviral vector.", PROC NATL ACAD SCI U S A, JUL 23 1996, 93 (15) P7917-22, UNITED STATES, XP002052041 *
TSUJI S ET AL: "Signal sequence and DNA-mediated expression of human lysosomal alpha-galactosidase A.", EUR J BIOCHEM, JUN 1 1987, 165 (2) P275-80, GERMANY, WEST, XP002052042 *

Cited By (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US9265780B2 (en) 1998-06-01 2016-02-23 Icahn School Of Medicine At Mount Sinai Method of enhancing lysosomal α-galactosidase A
CN106110309A (en) * 1999-03-11 2016-11-16 夏尔人类遗传性治疗公司 The treatment of α galactoside enzyme defect

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