[go: up one dir, main page]

AU2001269923A1 - Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases - Google Patents

Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Info

Publication number
AU2001269923A1
AU2001269923A1 AU2001269923A AU6992301A AU2001269923A1 AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1 AU 2001269923 A AU2001269923 A AU 2001269923A AU 6992301 A AU6992301 A AU 6992301A AU 2001269923 A1 AU2001269923 A1 AU 2001269923A1
Authority
AU
Australia
Prior art keywords
therapy
small molecule
lysosomal storage
storage diseases
enzyme replacement
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.)
Abandoned
Application number
AU2001269923A
Inventor
Seng H Cheng
David Meeker
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.)
Genzyme Corp
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
Publication of AU2001269923A1 publication Critical patent/AU2001269923A1/en
Abandoned legal-status Critical Current

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
    • A61K31/33—Heterocyclic compounds
    • A61K31/395—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435—Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/44—Non condensed pyridines; Hydrogenated derivatives thereof
    • A61K31/445—Non condensed piperidines, e.g. piperocaine
    • 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
    • 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
    • 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
    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Immunology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Urology & Nephrology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Vascular Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
AU2001269923A 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases Abandoned AU2001269923A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US21237700P 2000-06-19 2000-06-19
US60/212,377 2000-06-19
PCT/US2001/019579 WO2001097829A2 (en) 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Publications (1)

Publication Number Publication Date
AU2001269923A1 true AU2001269923A1 (en) 2002-01-02

Family

ID=22790749

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2001269923A Abandoned AU2001269923A1 (en) 2000-06-19 2001-06-19 Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Country Status (3)

Country Link
US (4) US20020095135A1 (en)
AU (1) AU2001269923A1 (en)
WO (1) WO2001097829A2 (en)

Families Citing this family (72)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6274597B1 (en) 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
WO2002055064A2 (en) * 2001-01-12 2002-07-18 Oxford Glycosciences (Uk) Ltd. Mucopolysaccharidosis therapies
GB9909066D0 (en) * 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
US20050032841A1 (en) * 1999-04-20 2005-02-10 Steven Walkley Therapeutic compositions and methods of treating glycolipid storage related disorders
CA2398995C (en) * 2000-02-04 2014-09-23 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related 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
US20020142299A1 (en) * 2001-01-09 2002-10-03 Davidson Beverly L. PTD-modified proteins
US8241622B2 (en) * 2001-07-13 2012-08-14 University Of Iowa Research Foundation Adeno-associated virus vectors with intravector heterologous terminal palindromic sequences
EP1336411A1 (en) * 2002-02-14 2003-08-20 Academisch Medisch Centrum bij de Universiteit van Amsterdam Compositions and methods for improving enzyme replacement therapy of lysosomal storage diseases
US20030223966A1 (en) * 2002-04-30 2003-12-04 Fraites Thomas J. Treatment for pompe disease
AU2003302724A1 (en) * 2002-08-13 2004-07-09 Mcivor, Scott, R. Methods of using vectors to treat metabolic disorders
ES2545619T3 (en) * 2003-01-31 2015-09-14 The Mount Sinai School Of Medicine Of New York University Combination therapy to treat protein deficiency disorders
US7442372B2 (en) * 2003-08-29 2008-10-28 Biomarin Pharmaceutical Inc. Delivery of therapeutic compounds to the brain and other tissues
US20090111812A1 (en) * 2004-06-14 2009-04-30 Musc Foundation For Research Development Methods for treating inflammatory disorders
MX2007014470A (en) * 2005-05-17 2008-02-06 Amicus Therapeutics Inc A method for the treatment of pompe disease using 1-deoxynojirimycin derivatives.
US20090297496A1 (en) * 2005-09-08 2009-12-03 Childrens Hospital Medical Center Lysosomal Acid Lipase Therapy for NAFLD and Related Diseases
WO2007075343A2 (en) * 2005-12-15 2007-07-05 Chemgen Corporation Enzymes for reduced immunological stress
ES2391657T3 (en) 2006-02-07 2012-11-28 Shire Human Genetic Therapies, Inc. Stabilized protein compositions that have a free thiol moiety
CA2706727A1 (en) 2007-11-29 2009-06-11 Michael S. Mcgrath Compositions and methods for treating lysosomal disorders
EP2271213A4 (en) * 2008-02-01 2011-06-29 Scripps Research Inst METHODS OF TREATING A PATHOLOGICAL CONDITION CHARACTERIZED BY DYSFUNCTION OF PROTEIN HOMEMYSTASIS
CA2718182A1 (en) * 2008-03-12 2009-09-17 Amicus Therapeutics, Inc. Treatment of pompe disease with specific pharmacological chaperones and monitoring treatment using surrogate markers
DK2318032T3 (en) 2008-06-26 2012-06-11 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
HUE060974T2 (en) 2008-12-16 2023-04-28 Genzyme Corp Synthetic intermediates for preparing oligosaccharide-protein conjugates
US20100248365A1 (en) * 2009-03-27 2010-09-30 Zacharon Pharmaceuticals, Inc. Ganglioside biosynthesis modulators
MX379016B (en) 2009-07-28 2025-03-10 Takeda Pharmaceuticals Co COMPOSITIONS AND METHODS FOR TREATING GAUCHER'S DISEASE.
SI3482767T1 (en) 2009-08-28 2022-03-31 Icahn School Of Medicine At Mount Sinai Dose escalation enzyme replacement therapy for treating acid sphingomyelinase deficiency
EP3028701B1 (en) * 2009-11-17 2019-11-13 Baylor Research Institute 1-deoxygalactonojirimycin for use in treating a cardiac disease
US8765698B2 (en) * 2010-02-03 2014-07-01 University Of Central Florida Research Foundation, Inc. Methods and products for reawakening retrocyclins
HUE062163T2 (en) 2010-04-23 2023-09-28 Alexion Pharma Inc Lysosomal storage disease enzyme
US9453847B2 (en) 2010-07-19 2016-09-27 Shire Human Genetic Therapies, Inc. Mannose receptor C type 1 (MRC1) codon optimized cell line and uses thereof
EP3650039A1 (en) 2010-09-09 2020-05-13 Alexion Pharmaceuticals, Inc. Use of lysosomal acid lipase for treating lysosomal acid lipase deficiency in patients
US9662375B2 (en) 2010-11-30 2017-05-30 Orphazyme Aps Methods for increasing intracellular activity of Hsp70
EP2675472A4 (en) 2011-02-15 2014-09-17 Synageva Biopharma Corp Methods for treating lysosomal acid lipase deficiency
WO2012112677A2 (en) * 2011-02-15 2012-08-23 Children's Hospital Medical Center Methods for treating lysosomal acid lipase deficiency
MX2013012596A (en) 2011-04-29 2014-08-21 Selecta Biosciences Inc SYNTHETIC TOLEROGENIC NANOPORTERS TO GENERATE CD8 + T REGULATORS LYMPHOCYTES.
WO2012177778A1 (en) 2011-06-20 2012-12-27 Mount Sinai School Of Medicine Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders
JP6230160B2 (en) 2012-03-02 2017-11-15 シャイア ヒューマン ジェネティック セラピーズ インコーポレイテッド Compositions and methods for treating type III Gaucher disease
WO2013134530A1 (en) 2012-03-07 2013-09-12 Amicus Therapeutics, Inc. High concentration alpha-glucosidase compositions for the treatment of pompe disease
US20150086530A1 (en) 2012-05-03 2015-03-26 Amicus Therapeutics, Inc. Dosing regimens for the treatment of pompe disease
US10155027B2 (en) 2012-07-17 2018-12-18 Amicus Therapeutics, Inc. Alpha-galactosidase A and 1-deoxygalactonojirimycin co-formulation for the treatment of fabry disease
US9694056B2 (en) 2012-07-17 2017-07-04 Amicus Therapeutics, Inc. α-galactosidase A and 1-deoxygalactonojirimycin co-formulation
EP2914281A1 (en) 2012-11-05 2015-09-09 Genzyme Corporation Compositions and methods for treating proteinopathies
BR112015027279A8 (en) 2013-05-03 2018-01-30 Selecta Biosciences Inc methods and compositions for enhancing cd4 + regulatory t cells
US20160074532A1 (en) 2014-09-07 2016-03-17 Selecta Biosciences, Inc. Methods and compositions for attenuating gene editing anti-viral transfer vector immune responses
CN107106494B (en) 2014-09-15 2021-12-21 奥菲泽米有限公司 Arilomo formulations
KR102741048B1 (en) 2014-09-30 2024-12-11 아미쿠스 세라퓨틱스, 인코포레이티드 Highly potent acid alpha-glucosidase with enhanced carbohydrates
KR102702200B1 (en) 2014-12-22 2024-09-02 코덱시스, 인코포레이티드 Human alpha-galactosidase variants
GB201508025D0 (en) 2015-05-11 2015-06-24 Ucl Business Plc Fabry disease gene therapy
CA3010205A1 (en) 2015-12-30 2017-07-06 Amicus Therapeutics, Inc. Augmented acid alpha-glucosidase for the treatment of pompe disease
US11702672B2 (en) 2016-02-08 2023-07-18 University Of Iowa Research Foundation Methods to produce chimeric adeno-associated virus/bocavirus parvovirus
MX2018010842A (en) 2016-03-07 2019-07-04 Univ Iowa Res Found Aav-mediated expression using a synthetic promoter and enhancer.
EP3433623A2 (en) * 2016-03-25 2019-01-30 Genzyme Corporation Biomarkers of proteopathies and uses thereof
KR20250017756A (en) 2016-03-30 2025-02-04 아미쿠스 세라퓨틱스, 인코포레이티드 Formulations comprising recombinant acid alpha-glucosidase
MX2018011951A (en) 2016-03-30 2019-02-13 Amicus Therapeutics Inc Method for selection of high m6p recombinant proteins.
US10898476B2 (en) 2016-04-13 2021-01-26 Orphazyme A/S Heat shock proteins and cholesterol homeostasis
HRP20210047T1 (en) 2016-04-29 2021-04-16 Orphazyme A/S, c/o COBIS A/S Arimoclomol for treating glucocerebrosidase associated disorders
JP2019519501A (en) * 2016-05-12 2019-07-11 ブライアン ピー． ハンリーＢｒｉａｎ Ｐ． ＨＡＮＬＥＹ Safe delivery of CRISPR and other gene therapeutics to the majority of somatic cells in humans and animals
WO2017205739A1 (en) 2016-05-26 2017-11-30 University Of Iowa Research Foundation cis AND trans REQUIREMENTS FOR TERMINAL RESOLUTION OF HUMAN BOCAVIRUS 1
NL2017294B1 (en) 2016-08-05 2018-02-14 Univ Erasmus Med Ct Rotterdam Natural cryptic exon removal by pairs of antisense oligonucleotides.
CA3033557A1 (en) * 2016-08-11 2018-02-15 Intrabio Limited Pharmaceutical compositions and uses directed to lysosomal storage disorders
EP3509628A1 (en) * 2016-09-09 2019-07-17 Fondazione Telethon Combined therapy for mucopolysaccharidosis type vi (maroteaux-lamy-syndrome)
US11142775B2 (en) 2017-01-13 2021-10-12 University Of Iowa Research Foundation Bocaparvovirus small noncoding RNA and uses thereof
EP3592389B1 (en) 2017-03-11 2025-05-07 Cartesian Therapeutics, Inc. Methods and compositions related to combined treatment with anti-inflammatories and synthetic nanocarriers comprising an immunosuppressant
HRP20230576T1 (en) 2017-05-15 2023-09-01 Amicus Therapeutics, Inc. Recombinant human acid alpha-glucosidase
CA3066569A1 (en) 2017-06-07 2018-12-13 Regeneron Pharmaceuticals, Inc. Compositions and methods for internalizing enzymes
JP7779653B2 (en) 2018-02-07 2025-12-03 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Methods and compositions for therapeutic protein delivery
KR20250140631A (en) 2018-05-17 2025-09-25 리제너론 파마슈티칼스 인코포레이티드 Anti-cd63 antibodies, conjugates, and uses thereof
CA3106651A1 (en) * 2018-07-18 2020-01-23 Stephen YOO Treatment of mucopolysaccharidosis i with fully-human glycosylated human alpha-l-iduronidase (idus)
CA3123598A1 (en) 2018-12-20 2020-06-25 Codexis, Inc. Human alpha-galactosidase variants
KR20230128462A (en) 2020-11-19 2023-09-05 제브라 덴마크 에이/에스 Process for preparing arimoclomol citrate and intermediates thereof
US20250163457A1 (en) * 2022-02-25 2025-05-22 Asklepios Biopharmaceutical, Inc. Therapeutic adeno-associated virus for treating pompe disease with long term cessation of gaa enzyme replacement therapy

Family Cites Families (47)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5851991A (en) * 1987-08-31 1998-12-22 The Regents Of The University Of California Therapeutic use of the retinoblastoma susceptibility gene product
US5549892A (en) * 1988-12-23 1996-08-27 Genzyme Corporation Enhanced in vivo uptake of glucocerebrosidase
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
US5272071A (en) * 1989-12-22 1993-12-21 Applied Research Systems Ars Holding N.V. Method for the modification of the expression characteristics of an endogenous gene of a given cell line
US5670488A (en) * 1992-12-03 1997-09-23 Genzyme Corporation Adenovirus vector for gene therapy
US5236938A (en) * 1991-04-30 1993-08-17 Rhone-Poulenc Inc. Pesticidal 1-aryl-5-(substituted alkylideneimino)pyrazoles
US5733761A (en) * 1991-11-05 1998-03-31 Transkaryotic Therapies, Inc. Protein production and protein delivery
WO1993025673A1 (en) * 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
US5834266A (en) 1993-02-12 1998-11-10 President & Fellows Of Harvard College Regulated apoptosis
US5836905A (en) * 1994-06-20 1998-11-17 Lemelson; Jerome H. Apparatus and methods for gene therapy
US5948767A (en) * 1994-12-09 1999-09-07 Genzyme Corporation Cationic amphiphile/DNA complexes
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
US5650096A (en) * 1994-12-09 1997-07-22 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5747471A (en) * 1994-12-09 1998-05-05 Genzyme Corporation Cationic amphiphiles containing steroid lipophilic groups for intracellular delivery of therapeutic molecules
US5939401A (en) * 1994-12-09 1999-08-17 Genzyme Corporation Cationic amphiphile compositions for intracellular delivery of therapeutic molecules
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
US5910487A (en) * 1994-12-09 1999-06-08 Genzyme Corporation Cationic amphiphiles and plasmids for intracellular delivery of therapeutic molecules
US6492332B1 (en) * 1995-12-12 2002-12-10 Omeros Corporation Irrigation solution and methods for inhibition of tumor cell adhesion, pain and inflammation
US5707618A (en) * 1995-03-24 1998-01-13 Genzyme Corporation Adenovirus vectors for gene therapy
US5756283A (en) * 1995-06-05 1998-05-26 The Trustees Of The University Of Pennsylvania Method for improved production of recombinant adeno-associated viruses for gene therapy
EP0850313B8 (en) 1995-09-08 2009-07-29 Genzyme Corporation Improved aav vectors for gene therapy
WO1997010817A1 (en) * 1995-09-20 1997-03-27 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
US5840702A (en) * 1996-03-22 1998-11-24 Uab Research Foundation Cystic fibrosis treatment
US5935936A (en) * 1996-06-03 1999-08-10 Genzyme Corporation Compositions comprising cationic amphiphiles and co-lipids for intracellular delivery of therapeutic molecules
US6177447B1 (en) * 1996-07-15 2001-01-23 Universiteit Van Amsterdam Deoxynojirimycin derivatives and their uses as glucosylceramidase inhibitors
CN1268741C (en) 1996-09-13 2006-08-09 超卡里奥迪克治疗学股份有限公司 Therapy for 'alpha'-galactosidase deficiency
US5861397A (en) 1996-10-03 1999-01-19 Vical Incorporated Piperazine based cytofectins
US5925628A (en) * 1997-03-31 1999-07-20 Genzyme Corporation Cationic amphiphiles for intracellular delivery of therapeutic molecules
US5912239A (en) * 1997-04-04 1999-06-15 Genzyme Corporation Imidazole-containing cationic amphiphiles for intracellular delivery of therapeutic molecules
US5948925A (en) * 1997-05-06 1999-09-07 Genzyme Corporation Cationic amphiphiles containing linkers derived from neutral or positively charged amino acids
US5942634A (en) * 1997-05-09 1999-08-24 Genzyme Corporation Cationic amphiphiles for cell transfections
EP2147681A1 (en) * 1997-10-29 2010-01-27 Genzyme Corporation Compositions and methods for treating lysosomal storage disease
US6465488B1 (en) * 1997-12-11 2002-10-15 Chancellor, Masters & Scholars Of The University Of Oxford Inhibition of glycolipid biosynthesis
CA2318737A1 (en) 1998-02-17 1999-08-19 Genzyme Corporation Methods for pseudoadenoviral vector production
JP2002505085A (en) 1998-02-17 2002-02-19 ジェンザイム・コーポレイション Method for producing purified AAV vector
WO1999057296A1 (en) 1998-05-01 1999-11-11 Genzyme Corporation Partially deleted adenoviral vectors
US6610703B1 (en) * 1998-12-10 2003-08-26 G.D. Searle & Co. Method for treatment of glycolipid storage diseases
GB9909064D0 (en) 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
GB0100889D0 (en) * 2001-01-12 2001-02-21 Oxford Glycosciences Uk Ltd Compounds
GB9909066D0 (en) * 1999-04-20 1999-06-16 Oxford Glycosciences Uk Ltd Therapies
ATE234626T1 (en) * 1999-07-26 2003-04-15 Searle & Co USE OF LONG CHAIN N-ALKYL DERIVATIVES OF DEOXYNOJIRIMYCIN WITH GLUCOCEREBROSIDASE ENZYMES FOR PRODUCING A MEDICATION FOR THE TREATMENT OF DISEASES RELATED TO GLYCOLIPID ACCUMULATION
ATE360082T1 (en) * 1999-11-16 2007-05-15 Genzyme Corp VECTORS AND TRANSGENES WITH REGULATORY ELEMENTS FOR GENE ADMINISTRATION IN LIVER
US20010031741A1 (en) 2000-02-17 2001-10-18 Robin Ziegler Methods for treatment of 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
ES2545619T3 (en) * 2003-01-31 2015-09-14 The Mount Sinai School Of Medicine Of New York University Combination therapy to treat protein deficiency disorders

Also Published As

Publication number Publication date
US7910545B2 (en) 2011-03-22
WO2001097829A2 (en) 2001-12-27
US20020095135A1 (en) 2002-07-18
WO2001097829A3 (en) 2002-12-27
US20070280925A1 (en) 2007-12-06
US8168587B2 (en) 2012-05-01
US20110142818A1 (en) 2011-06-16
US20120183502A1 (en) 2012-07-19

Similar Documents

Publication Publication Date Title
AU2001269923A1 (en) Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
EP1463512A4 (en) Use of p97 as an enzyme delivery system for the delivery of therapeutic lysosomal enzymes
AU1878601A (en) Enzyme
AU3818300A (en) Treating hair by targeting enzymes
AU2001227900A1 (en) Drug metabolizing enzymes
AUPR311601A0 (en) Matrix gene expression in chondrogenesis
AU2001274981A1 (en) Drug metabolizing enzymes
AUPQ262599A0 (en) Gene expression
AU2001270303A1 (en) Drug metabolizing enzymes
AU4009899A (en) Molecules targeting cd40 and tumor cells
EP1572877A3 (en) Drug metabolizing enzymes
AU2001265096A1 (en) Short-root gene, promoter, and uses thereof
AU7709300A (en) Drug discovery using gene expression profiling
AU2001270478A1 (en) Gene expression in biological conditions
AU2001255324A1 (en) Drug metabolizing enzymes
AU2732000A (en) Reverse gene therapy
AU1194201A (en) Fluorotellurite, amplifier glasses
AU2153401A (en) Gene expression in biological conditions
AU2001247444A1 (en) Erbeta-mediated gene expression
AU2001278154A1 (en) Drug metabolizing enzymes
AU2352301A (en) Gene expression in corneal diseases
AU1870000A (en) Gene therapy-2
AU2001284680A1 (en) Haplotypes of the alas2 gene
AU4552700A (en) Gene therapy
AU2002230890A1 (en) Targeted enzyme prodrug therapy