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)
  • Engineering & Computer Science (AREA)
  • Medicinal Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Biomedical Technology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • General Chemical & Material Sciences (AREA)
  • Immunology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Hematology (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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (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
US6066626A (en) * 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method for treating lysosomal storage disease
CA2340276A1 (en) * 1998-08-28 2000-03-09 Duke University Adenoviruses deleted in the iva2, 100k and/or preterminal protein sequences
WO2001036603A2 (en) * 1999-11-17 2001-05-25 Avigen, Inc. Recombinant adeno-associated virus virions for the treatment of lysosomal disorders
ES2328446T5 (es) 2000-02-04 2014-02-27 Children's Hospital Research Foundation Uso de lipasa ácida lisosomal para tratar la aterosclerosis y enfermedades asociadas
WO2001060414A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Genetic modification of the lung as a portal for gene delivery
WO2001060377A2 (en) * 2000-02-17 2001-08-23 Genzyme Corporation Methods for treatment of lysosomal storage diseases using biphosphonates
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
AU2001269923A1 (en) 2000-06-19 2002-01-02 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
EP1368039A4 (de) * 2001-02-20 2006-03-29 Uab Research Foundation Aminoglycosid-behandlung für lysosomale speicherkrankheiten
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
EP1974752B1 (de) * 2001-04-30 2012-09-26 BioMarin Pharmaceutical Inc. Subzelluläres Targeting von therapeutischen Proteinen
US7560424B2 (en) 2001-04-30 2009-07-14 Zystor Therapeutics, Inc. Targeted 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
US7252799B2 (en) * 2001-08-31 2007-08-07 Clearant, Inc. Methods for sterilizing preparations containing albumin
US6749851B2 (en) 2001-08-31 2004-06-15 Clearant, Inc. Methods for sterilizing preparations of digestive enzymes
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
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
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
PT2857036T (pt) * 2003-01-31 2017-03-13 Mount Sinai School Of Medicine Of N Y Univ Terapia combinada para o tratamento de distúrbios de deficiência proteica
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
CA2553955C (en) * 2004-02-10 2012-08-28 Zystor Therapeutics, Inc. Acid alpha-glucosidase and fragments thereof
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
US7935336B2 (en) 2005-11-18 2011-05-03 Tokyo Metropolitan Organization For Medical Research Highly functional enzyme having α-galactosidase activity
ES2700048T3 (es) 2006-02-08 2019-02-13 Genzyme Corp Terapia génica par la enfermedad de Niemann-Pick tipo A
CA2669347A1 (en) * 2006-11-13 2008-05-29 Zystor Therapeutics, Inc. Methods for treating pompe disease
CN102978185B (zh) 2007-01-18 2015-02-11 建新公司 包含氨基氧基基团的寡糖及其轭合物
EP2166093B1 (de) 2007-05-18 2015-09-16 Tokyo Metropolitan Institute of Medical Science Pharmazeutische zusammensetzungen für die enzymersatztherapie
EP2211856A4 (de) 2007-11-29 2011-02-09 Hospital For Sick Children Zusammensetzungen und verfahren zur behandlung lysosomaler störungen
ES2629853T3 (es) * 2008-05-07 2017-08-16 Biomarin Pharmaceutical Inc. Péptidos de dirección lisosómica y usos de los mismos
WO2010056746A1 (en) * 2008-11-11 2010-05-20 Amicus Therapeutics, Inc. Therapy regimens, dosing regimens and stable medicaments for the treatment of pompe disease
PL2889043T3 (pl) 2008-12-16 2019-09-30 Genzyme Corporation Syntetyczne związki pośrednie do wytwarzania koniugatów oligosacharyd-białko
EP2475376B1 (de) 2009-06-17 2016-03-30 BioMarin Pharmaceutical Inc. Formulierungen für lysosomale enzyme
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
MX338426B (es) 2010-04-23 2016-04-15 Synageva Biopharma Corp Enzima de enfermedad del almacenamiento lisosomico.
DK2613798T4 (en) 2010-09-09 2018-04-16 Synageva Biopharma Corp USING LYSOSOMAL SUR LIPASE TO TREAT PATIENTS WITHOUT LYSOSOMAL SUR LIPASE
HUE030932T2 (en) 2010-11-08 2017-06-28 Amicus Therapeutics Inc Increased stability and increased catalytic activity, altered, recombinant, β-glucocerebrosidase proteins
US20120178105A1 (en) * 2011-01-10 2012-07-12 Genzyme Corporation Detection of globotriaosylceramide (glc) in human urine samples using an antibody sandwich
EP2675472A4 (de) 2011-02-15 2014-09-17 Synageva Biopharma Corp Verfahren zur behandlung von mangel an lysosomaler saurer lipase
CN103917094B (zh) 2011-03-18 2016-11-09 建新公司 葡萄糖基神经酰胺合酶抑制剂
RU2013151875A (ru) 2011-04-22 2015-05-27 Джензим Корпорейшн Модифицированная кислая альфа глюкозидаза с ускоренным процессингом
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
EP3741384A1 (de) 2011-09-07 2020-11-25 Mount Sinai School Of Medicine Ceramidase und zelldifferenzierung
CN109529028A (zh) 2012-06-01 2019-03-29 西奈山伊坎医学院 在感染的治疗和预防中的神经酰胺水平
MA37975B2 (fr) 2012-09-11 2021-03-31 Genzyme Corp Inhibiteurs de synthase de glucosylcéramide
EP2929035A1 (de) 2012-12-07 2015-10-14 Shire Human Genetic Therapies, Inc. Lipidnanopartikel zur freisetzung von mrna
WO2014160390A1 (en) 2013-03-14 2014-10-02 Icahn School Of Medicine At Mount Sinai Therapeutic acid ceramidase compositions and methods of making and using them
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
TWI642782B (zh) 2013-10-23 2018-12-01 健臻公司 重組醣蛋白及其用途
US20170360900A1 (en) 2014-12-22 2017-12-21 Codexis, Inc. Human alpha-galactosidase variants
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
CN110022904B (zh) * 2016-10-20 2024-04-19 桑格摩生物治疗股份有限公司 用于治疗法布里病的方法和组合物
NL2021840B1 (en) 2018-10-19 2020-05-13 Univ Leiden Pharmacological Chaperones For Enzyme Treatment Therapy
WO2020046132A1 (en) 2018-08-31 2020-03-05 Leiden University Pharmacological chaperones for enzyme treatment therapy
WO2020102532A1 (en) * 2018-11-15 2020-05-22 President And Fellows Of Harvard College Genetically engineered skin cells for the systemic in vivo treatment of deficient enzymes, factors or proteins
MX2021007552A (es) 2018-12-20 2021-08-11 Codexis Inc Variantes de alfa-galactosidasa humana.
US12527776B2 (en) 2019-02-04 2026-01-20 Genzyme Corporation Treatment of ciliopathies using inhibitors of glucosylceramide synthase (GCS)
PL3850089T3 (pl) 2019-02-04 2024-05-06 Freeline Therapeutics Limited Polinukleotydy
JP7686574B2 (ja) 2019-05-22 2025-06-02 マサチューセッツ インスティテュート オブ テクノロジー 環状ｒｎａ組成物及び方法
WO2021073607A1 (en) * 2019-10-17 2021-04-22 Westlake Therapeutics (Hangzhou) Co. Limited Engineering red blood cells for treatment of lysosomal storage diseases
AU2020397956A1 (en) 2019-12-04 2022-07-07 Orna Therapeutics, Inc. Circular RNA compositions and methods
EP4100009A1 (de) 2020-02-03 2022-12-14 Genzyme Corporation Verfahren zur behandlung neurologischer symptome im zusammenhang mit lysosomalen speicherkrankheiten
EP4121453A2 (de) 2020-03-20 2023-01-25 Orna Therapeutics, Inc. Zirkuläre rna-zusammensetzungen und verfahren
US20240042015A1 (en) 2020-05-19 2024-02-08 Orna Therapeutics, Inc. Circular rna compositions and methods
KR20230043024A (ko) 2020-07-24 2023-03-30 젠자임 코포레이션 벤글루스타트를 포함하는 제약 조성물
AU2022288705A1 (en) 2021-06-10 2023-12-07 Orna Therapeutics, Inc. Circular 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
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
US6071890A (en) 1994-12-09 2000-06-06 Genzyme Corporation Organ-specific targeting of cationic amphiphile/DNA complexes for gene therapy
US5719131A (en) * 1994-12-09 1998-02-17 Genzyme Corporation Cationic amphiphiles containing dialkylamine 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
US5707618A (en) 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
EP1983057A3 (de) 1995-09-08 2009-01-07 Genzyme Corporation Verbesserte AAV-Vektoren für die Gentherapie
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
PT1538202E (pt) * 1996-09-13 2014-04-22 Shire Human Genetic Therapies Produção de alga-galactosidase a humana
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
US6066626A (en) * 1997-10-29 2000-05-23 Genzyme Corporation Compositions and method 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
AU1284799A (en) 2000-03-06
IL135578A (en) 2007-03-08
IL135578A0 (en) 2001-05-20
ES2268799T3 (es) 2007-03-16
US6066626A (en) 2000-05-23
US20030087868A1 (en) 2003-05-08
AU734290B2 (en) 2001-06-07
EP1027069B1 (de) 2006-07-26
EP1027069A1 (de) 2000-08-16
DE69835367D1 (de) 2006-09-07
WO2000009153A1 (en) 2000-02-24
JP2002522509A (ja) 2002-07-23
JP2010031023A (ja) 2010-02-12
CA2305768A1 (en) 2000-02-24
IL177228A0 (en) 2006-12-10
EP2147681A1 (de) 2010-01-27
DE69835367T2 (de) 2007-08-02
US20100041151A1 (en) 2010-02-18

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