ATE333893T1 - Gentherapie für gaucher-krankheit - Google Patents

Gentherapie für gaucher-krankheit

Info

Publication number
ATE333893T1
ATE333893T1 AT98956290T AT98956290T ATE333893T1 AT E333893 T1 ATE333893 T1 AT E333893T1 AT 98956290 T AT98956290 T AT 98956290T AT 98956290 T AT98956290 T AT 98956290T AT E333893 T1 ATE333893 T1 AT E333893T1
Authority
AT
Austria
Prior art keywords
gene therapy
gaucher disease
biologically active
cells
vector
Prior art date
Application number
AT98956290T
Other languages
English (en)
Inventor
Nelson S Yew
Robin J Ziegler
Seng H Cheng
Original Assignee
Genzyme 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 Genzyme Corp filed Critical Genzyme Corp
Application granted granted Critical
Publication of ATE333893T1 publication Critical patent/ATE333893T1/de

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • 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
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2217/00—Genetically modified animals
    • A01K2217/07—Animals genetically altered by homologous recombination
    • A01K2217/075—Animals genetically altered by homologous recombination inducing loss of function, i.e. knock out
    • 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
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/022—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from an adenovirus

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Biomedical Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Microbiology (AREA)
  • Immunology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
AT98956290T 1997-10-29 1998-10-29 Gentherapie für gaucher-krankheit ATE333893T1 (de)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US6352797P 1997-10-29 1997-10-29

Publications (1)

Publication Number Publication Date
ATE333893T1 true ATE333893T1 (de) 2006-08-15

Family

ID=22049811

Family Applications (1)

Application Number Title Priority Date Filing Date
AT98956290T ATE333893T1 (de) 1997-10-29 1998-10-29 Gentherapie für gaucher-krankheit

Country Status (10)

Country Link
US (3) US6066626A (de)
EP (2) EP1027069B1 (de)
JP (2) JP2002522509A (de)
AT (1) ATE333893T1 (de)
AU (1) AU734290B2 (de)
CA (1) CA2305768A1 (de)
DE (1) DE69835367T2 (de)
ES (1) ES2268799T3 (de)
IL (3) IL135578A0 (de)
WO (1) WO2000009153A1 (de)

Families Citing this family (89)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5362442A (en) * 1993-07-22 1994-11-08 2920913 Canada Inc. Method for sterilizing products with gamma radiation
US20040067157A1 (en) * 1993-07-22 2004-04-08 Clearant, Inc. Methods for sterilizing biological materials
US6705323B1 (en) * 1995-06-07 2004-03-16 Conceptus, Inc. Contraceptive transcervical fallopian tube occlusion devices and methods
WO2000009153A1 (en) * 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
CN1342206A (zh) * 1998-08-28 2002-03-27 杜克大学 在IVa2,100K和/或preterminal protein序列上有缺失的腺病毒
US6582692B1 (en) * 1999-11-17 2003-06-24 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
CA2398995C (en) 2000-02-04 2014-09-23 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
WO2001060377A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
JP2003533437A (ja) * 2000-02-17 2003-11-11 ジェンザイム・コーポレイション 遺伝子デリバリーのための門戸としての肺の遺伝的修飾
US20040086420A1 (en) * 2000-03-23 2004-05-06 Macphee Martin J. Methods for sterilizing serum or plasma
US20040204379A1 (en) * 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
WO2001097829A2 (en) * 2000-06-19 2001-12-27 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7723296B2 (en) 2001-01-18 2010-05-25 Genzyme Corporation Methods for introducing mannose-6-phosphate and other oligosaccharides onto glycoproteins and its application thereof
US7749971B2 (en) * 2001-02-20 2010-07-06 Bedwell David M Aminoglycoside treatment for lysosomal storage diseases
US6682695B2 (en) * 2001-03-23 2004-01-27 Clearant, Inc. Methods for sterilizing biological materials by multiple rates
US20040005309A1 (en) * 2002-05-29 2004-01-08 Symbiontics, Inc. Targeted therapeutic proteins
US7629309B2 (en) * 2002-05-29 2009-12-08 Zystor Therapeutics, Inc. Targeted therapeutic proteins
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted therapeutic proteins
AU2002256423B2 (en) * 2001-04-30 2008-07-24 Zystor Therapeutics, Inc. Subcellular targeting of therapeutic proteins
US6946126B2 (en) * 2001-06-04 2005-09-20 Duke University Replicating adenovirus vectors
US6696060B2 (en) * 2001-06-14 2004-02-24 Clearant, Inc. Methods for sterilizing preparations of monoclonal immunoglobulins
US6946098B2 (en) 2001-08-10 2005-09-20 Clearant, Inc. Methods for sterilizing biological materials
US20030031584A1 (en) * 2001-08-10 2003-02-13 Wilson Burgess Methods for sterilizing biological materials using dipeptide stabilizers
US6749851B2 (en) 2001-08-31 2004-06-15 Clearant, Inc. Methods for sterilizing preparations of digestive enzymes
US7252799B2 (en) * 2001-08-31 2007-08-07 Clearant, Inc. Methods for sterilizing preparations containing albumin
US20030095890A1 (en) * 2001-09-24 2003-05-22 Shirley Miekka Methods for sterilizing biological materials containing non-aqueous solvents
US20110091353A1 (en) * 2001-09-24 2011-04-21 Wilson Burgess Methods for Sterilizing Tissue
US6783968B2 (en) 2001-09-24 2004-08-31 Clearant, Inc. Methods for sterilizing preparations of glycosidases
US20030185702A1 (en) * 2002-02-01 2003-10-02 Wilson Burgess Methods for sterilizing tissue
US20030072761A1 (en) * 2001-10-16 2003-04-17 Lebowitz Jonathan Methods and compositions for targeting proteins across the blood brain barrier
US20030124023A1 (en) * 2001-12-21 2003-07-03 Wilson Burgess Method of sterilizing heart valves
US20030180181A1 (en) * 2002-02-01 2003-09-25 Teri Greib Methods for sterilizing tissue
US7090836B2 (en) * 2002-06-21 2006-08-15 Institut Pasteur Vector for expressing α-L-iduronidase and method of treating MPS I by stereotactic injection into the brain of a mammal
US20040013562A1 (en) * 2002-07-18 2004-01-22 Wilson Burgess Methods for sterilizing milk.
US6908591B2 (en) * 2002-07-18 2005-06-21 Clearant, Inc. Methods for sterilizing biological materials by irradiation over a temperature gradient
CN102586205A (zh) * 2003-01-31 2012-07-18 纽约大学西奈山医学院 酶和酶的活性部位特异性陪伴分子的组合
US7446098B2 (en) * 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US20040258666A1 (en) * 2003-05-01 2004-12-23 Passini Marco A. Gene therapy for neurometabolic disorders
BRPI0416216A (pt) * 2003-11-12 2006-12-26 Amicus Therapeutics Inc composto de glicoimidazol e polihidroxiciclohexenila e método para sintetizar composto de glicoimidazol e polihidroxiciclohexenila, métodos para inibir glicocerebrosidase, para aumentar a atividade de glicocerebrosidase e para estabilizar glicocerebrosidade, composição farmacêutica, métodos para inibir uma -glicosidade, e para tratar doença de gaucher, derivado de glicoimidazol e derivados de polihidroxiciclohexilamina
JP4914224B2 (ja) * 2004-02-10 2012-04-11 バイオマリン ファーマシューティカル インコーポレイテッド 酸性αグルコシダーゼおよびそのフラグメント
US8889127B2 (en) * 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
BRPI0618583A2 (pt) 2005-11-18 2011-09-06 Tokyo Metropolitan Org Med Res enzima altamente funcional tendo especifidade ao substrato modificada
EP1986661B1 (de) 2006-02-08 2018-08-29 Genzyme Corporation Gentherapie für die niemann-pick-krankheit vom typ a
EP2099523A2 (de) * 2006-11-13 2009-09-16 ZyStor Therapeutics , Inc. Verfahren zur behandlung von pompe-krankheit
CA2675968C (en) 2007-01-18 2019-05-21 Genzyme Corporation Oligosaccharides comprising an aminooxy group and conjugates thereof
JP5507242B2 (ja) 2007-05-18 2014-05-28 公益財団法人東京都医学総合研究所 酵素補充療法用医薬組成物
WO2009073564A1 (en) 2007-11-29 2009-06-11 The Hospital For Sick Children Compositions and methods for treating lysosomal disorders
US20110223147A1 (en) 2008-05-07 2011-09-15 Zystor Therapeutics, Inc. Lysosomal targeting peptides and uses thereof
US20100119502A1 (en) * 2008-11-11 2010-05-13 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
SI2889043T1 (sl) 2008-12-16 2019-08-30 Genzyme Corporation Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein
ES2758827T3 (es) 2009-06-17 2020-05-06 Biomarin Pharm Inc Formulaciones para enzimas lisosómicas
WO2011028941A2 (en) 2009-09-04 2011-03-10 The United States Of America, As Represented By The Secretary Department Of Health & Human Services Disabling autophagy as a treatment for lysosomal storage diseases
EP2561069B1 (de) 2010-04-23 2017-03-08 Alexion Pharmaceuticals, Inc. Enzym für die lysosomale speicherkrankheit
NZ715014A (en) 2010-09-09 2018-10-26 Synageva Biopharma Corp Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
DK2638152T3 (en) 2010-11-08 2016-12-12 Amicus Therapeutics Inc VARIANTE, RECOMBINANT BETA-glucocerebrosidase PROTEINS WITH INCREASED STABILITY AND INCREASED RETAINED CATALYTIC ACTIVITY
US20120178105A1 (en) * 2011-01-10 2012-07-12 Genzyme Corporation Detection of globotriaosylceramide (glc) in human urine samples using an antibody sandwich
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
MY173998A (en) 2011-03-18 2020-03-03 Genzyme Corp Glucosylceramide synthase inhibitors
EP2699676A1 (de) 2011-04-22 2014-02-26 Genzyme Corporation Modifizierte saure alpha-glukosidase mit beschleunigter verarbeitung
PT2717893T (pt) 2011-06-08 2019-08-20 Translate Bio Inc Composições de nanopartículas lipídicas e métodos para transferência de arnm
GB201115280D0 (en) * 2011-09-05 2011-10-19 Alligator Bioscience Ab Antibodies, uses and methods
DK2753346T3 (da) 2011-09-07 2020-05-25 Sinai School Medicine Ceramidase og celledifferentiering
EP2854910B1 (de) 2012-06-01 2020-04-15 Icahn School of Medicine at Mount Sinai Ceramidspiegel bei der behandlung und vorbeugung von infektionen
JOP20130273B1 (ar) 2012-09-11 2021-08-17 Genzyme Corp مثبطات انزيم (سينثاز) غلوكوسيل سيراميد
WO2014089486A1 (en) 2012-12-07 2014-06-12 Shire Human Genetic Therapies, Inc. Lipidic nanoparticles for mrna delivering
EP3659619A1 (de) 2013-03-14 2020-06-03 Icahn School of Medicine at Mount Sinai Therapeutische säure-ceramidase-zusammensetzungen und verfahren zur herstellung und verwendung davon
JO3713B1 (ar) 2013-03-15 2021-01-31 Genzyme Corp أشكال ملح (s)-كوينوكليدين-3-يل(2-(2-(4-فلوروفينيل)ثيازول-4-يل)بروبان-2-يل)كارباميت
TWI793159B (zh) 2013-10-23 2023-02-21 美商健臻公司 重組醣蛋白及其用途
FI3237621T3 (fi) 2014-12-22 2023-06-01 Codexis Inc Ihmisen alfa-galaktosidaasivariantteja
US9981021B1 (en) 2015-04-09 2018-05-29 Kinetiq, Inc. Subcutaneous therapeutic enzyme formulations, uses, and methods for generating thereof
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
JP7042263B2 (ja) * 2016-10-20 2022-03-25 サンガモ セラピューティクス， インコーポレイテッド ファブリー病の治療のための方法および組成物
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
US20220001027A1 (en) * 2018-11-15 2022-01-06 President And Fellows Of Harvard College Genetically Engineered Skin Cells for the Systemic In Vivo Treatment of Deficient Enzymes, Factors or Proteins
EP3898960A4 (de) 2018-12-20 2022-11-30 Codexis, Inc. Varianten von menschlicher alpha-galactosidase
PT3920912T (pt) 2019-02-04 2025-09-09 Genzyme Corp Tratamento de ciliopatias usando inibidores da glucosilceramida sintase (gcs)
SG11202108502UA (en) 2019-02-04 2021-09-29 Freeline Therapeutics Ltd Polynucleotides
AU2020280105A1 (en) 2019-05-22 2022-01-20 Massachusetts Institute Of Technology Circular RNA compositions and methods
WO2021073607A1 (en) * 2019-10-17 2021-04-22 Westlake Therapeutics (Hangzhou) Co. Limited Engineering red blood cells for treatment of lysosomal storage diseases
JP2023504568A (ja) 2019-12-04 2023-02-03 オルナ セラピューティクス インコーポレイテッド 環状ｒｎａ組成物及び方法
EP4100009A1 (de) 2020-02-03 2022-12-14 Genzyme Corporation Verfahren zur behandlung neurologischer symptome im zusammenhang mit lysosomalen speicherkrankheiten
JP2023518295A (ja) 2020-03-20 2023-04-28 オルナ セラピューティクス インコーポレイテッド 環状ｒｎａ組成物及び方法
WO2021236855A1 (en) 2020-05-19 2021-11-25 Orna Therapeutics, Inc. Circular rna compositions and methods
CA3186766A1 (en) 2020-07-24 2022-01-27 Danielle Combessis Pharmaceutical compositions comprising venglustat
IL308746A (en) 2021-06-10 2024-03-01 Orna Therapeutics Inc Cyclic RNA compositions and methods
US12297285B2 (en) 2022-06-24 2025-05-13 Orna Therapeutics, Inc. Circular RNA encoding chimeric antigen receptors targeting BCMA
AU2024269222A1 (en) 2023-05-05 2025-10-09 Orna Therapeutics, Inc. Circular rna compositions and methods
WO2026006593A1 (en) 2024-06-28 2026-01-02 Orna Therapeutics, Inc. Synthetic internal ribosome entry sites

Family Cites Families (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4647447A (en) 1981-07-24 1987-03-03 Schering Aktiengesellschaft Diagnostic media
US5139941A (en) 1985-10-31 1992-08-18 University Of Florida Research Foundation, Inc. AAV transduction vectors
US5672344A (en) 1987-12-30 1997-09-30 The Regents Of The University Of Michigan Viral-mediated gene transfer system
US5236838A (en) * 1988-12-23 1993-08-17 Genzyme Corporation Enzymatically active recombinant glucocerebrosidase
US5179023A (en) * 1989-03-24 1993-01-12 Research Corporation Technologies, Inc. Recombinant α-galactosidase a therapy for Fabry disease
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5670488A (en) 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5401650A (en) * 1990-10-24 1995-03-28 The Mount Sinai School Of Medicine Of The City University Of New York Cloning and expression of biologically active α-galactosidase A
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
US5252479A (en) 1991-11-08 1993-10-12 Research Corporation Technologies, Inc. Safe vector for gene therapy
US5911983A (en) * 1992-06-26 1999-06-15 University Of Pittsburgh Gene therapy for Gaucher disease using retroviral vectors
US5872005A (en) * 1994-11-03 1999-02-16 Cell Genesys Inc. Packaging cell lines for adeno-associated viral vectors
US5719131A (en) * 1994-12-09 1998-02-17 Genzyme Corporation Cationic amphiphiles containing dialkylamine lipophilic groups for intracellular delivery of therapeutic molecules
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US5767099A (en) 1994-12-09 1998-06-16 Genzyme Corporation Cationic amphiphiles containing amino acid or dervatized amino acid groups for intracellular delivery of therapeutic molecules
US6071890A (en) 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
CA2230758A1 (en) 1995-09-08 1997-03-13 Genzyme Corporation Improved aav vectors for gene therapy
US5962313A (en) * 1996-01-18 1999-10-05 Avigen, Inc. Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
AU4352997A (en) 1996-08-30 1998-03-19 Genzyme Corporation Inhibition of primary and/or secondary immune response to repeat adenoviral vector administration using cd40l specific antibodies
HU227189B1 (en) * 1996-09-13 2010-10-28 Transkaryotic Therapies Therapy for alpha-galactosidase a deficiency
US5981275A (en) * 1997-04-14 1999-11-09 Genzyme Corporation Transgene expression system for increased persistence
GB9708526D0 (en) * 1997-04-25 1997-06-18 Royal Free Hosp School Med Eukaryotic gene expression cassette and uses thereof
WO2000009153A1 (en) * 1997-10-29 2000-02-24 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US5879005A (en) * 1998-02-26 1999-03-09 Baca; Ron Wargame playing system
CN105246194B (zh) 2011-11-15 2018-07-03 昂宝电子（上海）有限公司 用于各种操作模式中的恒流控制的led照明系统和方法
US9807841B2 (en) 2012-07-12 2017-10-31 Hubbell Incorporated Circuit for expanding the dimming range of an LED lamp
KR101690305B1 (ko) 2014-10-16 2016-12-27 주식회사 솔루엠 컨버터
CN104409036B (zh) 2014-12-16 2017-01-11 上海天马微电子有限公司 一种显示面板和显示装置

Also Published As

Publication number Publication date
DE69835367D1 (de) 2006-09-07
EP1027069B1 (de) 2006-07-26
WO2000009153A1 (en) 2000-02-24
IL177228A0 (en) 2006-12-10
DE69835367T2 (de) 2007-08-02
JP2002522509A (ja) 2002-07-23
US20030087868A1 (en) 2003-05-08
EP1027069A1 (de) 2000-08-16
ES2268799T3 (es) 2007-03-16
IL135578A0 (en) 2001-05-20
JP2010031023A (ja) 2010-02-12
AU734290B2 (en) 2001-06-07
US20100041151A1 (en) 2010-02-18
IL135578A (en) 2007-03-08
EP2147681A1 (de) 2010-01-27
US6066626A (en) 2000-05-23
CA2305768A1 (en) 2000-02-24
AU1284799A (en) 2000-03-06

Similar Documents

Publication Publication Date Title
DE69835367D1 (de) Gentherapie für gaucher-krankheit
WO2001049830A3 (en) Improved lysosomal enzymes and lysosomal enzyme activators
PT1083899E (pt) Derivados de 1-desoxi-galactonojirimicina e respectiva utilização no tratamento de doenças de acumulação lisossomal, por intensificação da alfa-galactosidase a lisossomal
DE69840165D1 (de) Genkonstrukt das für ein heterologes "prodrug"-aktivierendes enzym und für ein zellgerichtes teil kodiert
WO2001019955A3 (en) Lysosomal targeting pathway enzymes
IL118201A0 (en) Erythropoietin free of animal proteins processes for production and use thereof
DE69514234D1 (de) Oberflächenlokalisierte expression von enzymen in gengesteuerter prodrugtherapie
ATE376054T1 (de) Diacylglyzerin o-acyltransferase
ZA9711146B (en) Novel genes coding for amino acid deacetylase with specificity for N-acetyl-L-phosphinotricin, their isolation and use.
WO2002031134A3 (en) Novel serine protease genes related to dppiv
IL68366A (en) Process for expressing a gene encoding a protein comprising the enzymatic portion of human urokinase in a microorganism or cell culture
IL181695A0 (en) Methods for cultivating cells and propagating viruses
EP0967271A4 (de) Neue carbonyl-reduktase, gene welche diese kodieren und methoden zu deren verwendung
WO2002008404A3 (en) Production of lysosomal enzymes in plants by transient expression
DE69939426D1 (de) Diacylglycerin o-acyltransferase
DE122011000008I1 (de) Herstellung enzymatischer aktiver glukocerebrosidase von rekombinanten zellen.
ZA971158B (en) Nucleic acid constructs for the cell cycle-regulated expression of genes, cells containing such constructs and their use for preparing drugs.
ZA948987B (en) Therapy
DE3751280D1 (de) Enzyme als landwirtschaftliche chemische Hilfsmittel.
DE69532485D1 (de) Trehalose-freisetzendes Enzym, dafür kodierende DNA, Herstellung und Verwendung
WO2000068396A3 (en) Bacillus circulans xylanase mutants
DE69827317D1 (de) C3-Pflanzen, welche photosynthetische Enzyme von C4-Pflanzen exprimieren
JPS6430588A (en) Foreign dna developing promoter n methylotroph bacteria, its production and use
WO2001077307A3 (en) Expression system for efficiently producing clinically effective lysosomal enzymes (glucocerebrosidase)
BG105779A (en) Human antibiotic proteins

Legal Events

Date Code Title Description
RER Ceased as to paragraph 5 lit. 3 law introducing patent treaties