MXPA05007821A - Terapia de combinacion para tratar deficiencias de proteinas. - Google Patents

Terapia de combinacion para tratar deficiencias de proteinas.

Info

Publication number
MXPA05007821A
MXPA05007821A MXPA05007821A MXPA05007821A MXPA05007821A MX PA05007821 A MXPA05007821 A MX PA05007821A MX PA05007821 A MXPA05007821 A MX PA05007821A MX PA05007821 A MXPA05007821 A MX PA05007821A MX PA05007821 A MXPA05007821 A MX PA05007821A
Authority
MX
Mexico
Prior art keywords
combination therapy
treating protein
protein deficiencies
gene therapy
deficiencies
Prior art date
Application number
MXPA05007821A
Other languages
English (en)
Inventor
Jian-Qiang Fan
Original Assignee
Sinai School Medicine
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 Sinai School Medicine filed Critical Sinai School Medicine
Publication of MXPA05007821A publication Critical patent/MXPA05007821A/es

Links

Classifications

    • 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
    • A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
    • 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
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Diabetes (AREA)
  • Hematology (AREA)
  • Obesity (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Enzymes And Modification Thereof (AREA)
  • Peptides Or Proteins (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Esta solicitud proporciona metodos para mejorar la terapia de genes al combinar la terapia de genes con chaperones de sitios especificos activos (ASSC). Los ASSC incrementan la estabilidad y eficiencia de la proteina codificada por el gen recombinante que se administra.
MXPA05007821A 2003-02-18 2004-02-18 Terapia de combinacion para tratar deficiencias de proteinas. MXPA05007821A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US44807303P 2003-02-18 2003-02-18
PCT/US2004/004909 WO2004074450A2 (en) 2003-02-18 2004-02-18 Combination therapy for treating protein deficiencies

Publications (1)

Publication Number Publication Date
MXPA05007821A true MXPA05007821A (es) 2006-04-07

Family

ID=32908532

Family Applications (1)

Application Number Title Priority Date Filing Date
MXPA05007821A MXPA05007821A (es) 2003-02-18 2004-02-18 Terapia de combinacion para tratar deficiencias de proteinas.

Country Status (9)

Country Link
US (1) US7446098B2 (es)
EP (1) EP1594514A2 (es)
JP (1) JP2006517980A (es)
CN (1) CN1750834A (es)
BR (1) BRPI0407648A (es)
CA (1) CA2516304A1 (es)
IL (1) IL169633A0 (es)
MX (1) MXPA05007821A (es)
WO (1) WO2004074450A2 (es)

Families Citing this family (28)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007137072A2 (en) 2006-05-16 2007-11-29 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2013205532B8 (en) * 2006-05-16 2015-07-16 Amicus Therapeutics, Inc. Assays for diagnosing and evaluating treatment options for fabry disease
AU2007336184A1 (en) * 2006-12-21 2008-06-26 Novo Nordisk A/S Interleukin-21 variants with altered binding to the IL-21 receptor
AU2008245578A1 (en) 2007-04-26 2008-11-06 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
US9999618B2 (en) 2007-04-26 2018-06-19 Amicus Therapeutics, Inc. Dosing regimens for the treatment of lysosomal storage diseases using pharmacological chaperones
CN102655746B (zh) * 2009-02-23 2016-08-03 联合治疗公司 亚氨基糖以及治疗病毒性疾病的方法
EP2400843B1 (en) * 2009-02-24 2015-11-11 United Therapeutics Corporation Iminosugars and methods of treating arenaviral infections
EP2416656B1 (en) 2009-04-09 2015-12-23 Amicus Therapeutics, Inc. Methods for preventing and/or treating lysosomal storage disorders
US9206457B2 (en) 2009-05-26 2015-12-08 Amicus Therapeutics, Inc. Utilization of pharmacological chaperones to improve manufacturing and purification of biologics
CN102574887A (zh) * 2009-05-26 2012-07-11 阿米库斯治疗学公司 利用药物分子伴侣改善生物制剂的生产和纯化
US8426445B2 (en) 2009-06-12 2013-04-23 United Therapeutics Corporation Iminosugars and methods of treating bunyaviral and togaviral diseases
US20110065753A1 (en) * 2009-09-04 2011-03-17 United Therapeutics Corporation Methods of treating poxviral infections
DK2473482T3 (en) * 2009-09-04 2014-05-12 United Therapeutics Corp Methods of treating orthomyxoviral infections
US20110065754A1 (en) * 2009-09-04 2011-03-17 United Therapeutics Corporation Iminosugars and methods of treating filoviral diseases
CA2778349C (en) 2009-10-19 2018-12-04 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders
RU2581058C2 (ru) 2009-10-19 2016-04-10 Амикус Терапьютикс, Инк. Новые композиции для предупреждения и/или лечения дегенеративных расстройств центральной нервной системы
AR079057A1 (es) * 2009-11-17 2011-12-21 Baylor Res Inst Triaosilceramida urinaria (gb3) como marcador de enfermedad cardiaca
EP2721151B1 (en) 2011-06-20 2017-12-06 Mount Sinai School Of Medicine Anti-tnf-therapy for the mucopolysaccharidoses and other lysosomal disorders
EP2874648A4 (en) 2012-07-17 2015-12-30 Amicus Therapeutics Inc CO-FORMULATION OF ALPHA-GALACTOSIDASE A AND 1-DEOXYGALACTONOJIRIMYCIN
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
AU2013337354A1 (en) * 2012-11-05 2015-05-21 Genzyme Corporation Compositions and methods for treating proteinopathies
KR20220119187A (ko) * 2013-05-15 2022-08-26 리젠츠 오브 더 유니버시티 오브 미네소타 중추 신경계로의 아데노-연관 바이러스 매개 유전자 전달
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
CN105622459B (zh) * 2016-01-11 2017-10-13 中国科学院化学研究所 一种制备打碗花精及其中间体的方法
MX2019004487A (es) * 2016-10-20 2020-02-07 Sangamo Therapeutics Inc Metodos y composiciones para el tratamiento de la enfermedad de fabry.
KR20190134807A (ko) 2017-04-25 2019-12-04 아미쿠스 세라퓨틱스, 인코포레이티드 중추 신경계의 퇴행성 질환 및/또는 리소솜 저장병을 예방 및/또는 치료하기 위한 신규한 조성물
EP3684938A1 (en) 2017-09-22 2020-07-29 The Trustees of the University of Pennsylvania Gene therapy for treating mucopolysaccharidosis type ii

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP0672159B1 (en) 1992-04-24 2005-12-28 Sri International Homologous sequence targeting in eukaryotic cells
WO1997010817A1 (en) 1995-09-20 1997-03-27 The Regents Of The University Of Michigan Amino ceramide-like compounds and therapeutic methods of use
US5948653A (en) 1997-03-21 1999-09-07 Pati; Sushma Sequence alterations using homologous recombination
DE69835367T2 (de) * 1997-10-29 2007-08-02 Genzyme Corp., Framingham Gentherapie für gaucher-krankheit
US6274597B1 (en) * 1998-06-01 2001-08-14 Mount Sinai School Of Medicine Of New York University Method of enhancing lysosomal α-Galactosidase A
US20040204379A1 (en) 2000-06-19 2004-10-14 Cheng Seng H. Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases

Also Published As

Publication number Publication date
EP1594514A2 (en) 2005-11-16
US20040219132A1 (en) 2004-11-04
IL169633A0 (en) 2007-07-04
WO2004074450A2 (en) 2004-09-02
JP2006517980A (ja) 2006-08-03
CA2516304A1 (en) 2004-09-02
US7446098B2 (en) 2008-11-04
CN1750834A (zh) 2006-03-22
BRPI0407648A (pt) 2006-02-21
WO2004074450A3 (en) 2005-08-25
WO2004074450A8 (en) 2004-11-04

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