ATE360082T1 - Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber - Google Patents
Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leberInfo
- Publication number
- ATE360082T1 ATE360082T1 AT00982132T AT00982132T ATE360082T1 AT E360082 T1 ATE360082 T1 AT E360082T1 AT 00982132 T AT00982132 T AT 00982132T AT 00982132 T AT00982132 T AT 00982132T AT E360082 T1 ATE360082 T1 AT E360082T1
- Authority
- AT
- Austria
- Prior art keywords
- liver
- transgenes
- vectors
- regulatory elements
- elements
- Prior art date
Links
- 210000004185 liver Anatomy 0.000 title abstract 4
- 108700019146 Transgenes Proteins 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- 230000001105 regulatory effect Effects 0.000 title 1
- 239000013598 vector Substances 0.000 title 1
- 108010088751 Albumins Proteins 0.000 abstract 1
- 102000009027 Albumins Human genes 0.000 abstract 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 abstract 1
- 239000003623 enhancer Substances 0.000 abstract 1
- 230000002085 persistent effect Effects 0.000 abstract 1
Classifications
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
-
- 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
- A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
-
- 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
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
-
- 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
- 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
- C12N2710/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA dsDNA viruses
- C12N2710/00011—Details
- C12N2710/10011—Adenoviridae
- C12N2710/10311—Mastadenovirus, e.g. human or simian adenoviruses
- C12N2710/10341—Use of virus, viral particle or viral elements as a vector
- C12N2710/10343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
-
- 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
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/15—Vector systems having a special element relevant for transcription chimeric enhancer/promoter combination
-
- 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
- C12N2830/00—Vector systems having a special element relevant for transcription
- C12N2830/80—Vector systems having a special element relevant for transcription from vertebrates
- C12N2830/85—Vector systems having a special element relevant for transcription from vertebrates mammalian
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biotechnology (AREA)
- Biomedical Technology (AREA)
- Zoology (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Biochemistry (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Infusion, Injection, And Reservoir Apparatuses (AREA)
- Medicines Containing Plant Substances (AREA)
Applications Claiming Priority (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US16586699P | 1999-11-16 | 1999-11-16 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE360082T1 true ATE360082T1 (de) | 2007-05-15 |
Family
ID=22600808
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT00982132T ATE360082T1 (de) | 1999-11-16 | 2000-11-15 | Vektoren und transgene mit regulatorischen elementen zur genverabreichung in leber |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US7312324B2 (de) |
| EP (2) | EP1232276B8 (de) |
| AT (1) | ATE360082T1 (de) |
| AU (1) | AU1920201A (de) |
| DE (1) | DE60034478T2 (de) |
| ES (1) | ES2284545T3 (de) |
| WO (1) | WO2001036620A2 (de) |
Families Citing this family (48)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| 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 |
| US7858367B2 (en) * | 2002-04-30 | 2010-12-28 | Duke University | Viral vectors and methods for producing and using the same |
| HUE055861T2 (hu) | 2004-04-21 | 2021-12-28 | Alexion Pharma Inc | Csontbejuttató konjugátumok és azok alkalmazási módszere fehérjék csonthoz kötéséhez |
| US20070081984A1 (en) | 2005-10-11 | 2007-04-12 | Shunji Tomatsu | Compositions and methods for treating hypophosphatasia |
| TWI293307B (en) * | 2004-09-30 | 2008-02-11 | Ind Tech Res Inst | A liver-specific chimeric regulatory sequence and use thereof |
| EP1986661B1 (de) | 2006-02-08 | 2018-08-29 | Genzyme Corporation | Gentherapie für die niemann-pick-krankheit vom typ a |
| PL2158319T4 (pl) | 2007-05-11 | 2016-04-29 | Alexion Pharma Inc | Fosfataza alkaliczna kierowana do kości, zestawy i zastosowanie |
| DK2282764T3 (da) * | 2008-04-22 | 2019-10-14 | Vib Vzw | Leverspecifikke nukleinsyreregulerende elementer samt fremgangsmåder og anvendelser heraf |
| SI2889043T1 (sl) | 2008-12-16 | 2019-08-30 | Genzyme Corporation | Sintetične vmesne spojine za pripravo konjugatov oligosaharid-protein |
| BR112012027765A2 (pt) | 2010-04-30 | 2019-09-24 | Enobia Pharma Inc | métodos, composições e kits para tratamento de distúrbios de mineralização da matriz. |
| US9266939B2 (en) | 2010-12-27 | 2016-02-23 | Alexion Pharmaceuticals, Inc. | Compositions comprising natriuretic peptides and methods of use thereof |
| SG11201401605QA (en) | 2011-10-19 | 2014-09-26 | Alexion Pharma Holding | Compositions comprising alkaline phosphatase and/or natriuretic peptide and methods of use thereof |
| US10052366B2 (en) | 2012-05-21 | 2018-08-21 | Alexion Pharmaceuticsl, Inc. | Compositions comprising alkaline phosphatase and/or natriuretic peptide and methods of use thereof |
| WO2016007873A1 (en) | 2014-07-11 | 2016-01-14 | The Regents Of The University Of Michigan | Compositions and methods for treating craniosynostosis |
| GB201420139D0 (en) | 2014-11-12 | 2014-12-24 | Ucl Business Plc | Factor IX gene therapy |
| CA2967851C (en) | 2014-12-05 | 2024-02-27 | Alexion Pharmaceuticals, Inc. | Treating seizure with recombinant alkaline phosphatase |
| AU2016211447B2 (en) | 2015-01-28 | 2021-09-23 | Alexion Pharmaceuticals, Inc. | Methods of treating a subject with an alkaline phosphatase deficiency |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| EP3337894A1 (de) | 2015-08-17 | 2018-06-27 | Alexion Pharmaceuticals, Inc. | Herstellung von alkalischen phosphatasen |
| JP6868617B2 (ja) | 2015-09-28 | 2021-05-12 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | 低ホスファターゼ血症の組織非特異的アルカリホスファターゼ（ｔｎｓａｌｐ）酵素補充療法に有効な投薬計画の特定 |
| US11400140B2 (en) | 2015-10-30 | 2022-08-02 | Alexion Pharmaceuticals, Inc. | Methods for treating craniosynostosis in a patient |
| US11065306B2 (en) | 2016-03-08 | 2021-07-20 | Alexion Pharmaceuticals, Inc. | Methods for treating hypophosphatasia in children |
| KR20220162816A (ko) | 2016-04-01 | 2022-12-08 | 알렉시온 파마슈티칼스, 인코포레이티드 | 알칼리성 포스파타아제로 근육 약화의 치료 |
| WO2017173395A1 (en) | 2016-04-01 | 2017-10-05 | Alexion Pharmaceuticals, Inc. | Methods for treating hypophosphatasia in adolescents and adults |
| WO2017180857A1 (en) | 2016-04-15 | 2017-10-19 | The Trustees Of The University Of Pennsylvania | Gene therapy for treating hemophilia a |
| US10988744B2 (en) | 2016-06-06 | 2021-04-27 | Alexion Pharmaceuticals, Inc. | Method of producing alkaline phosphatase |
| WO2018035420A1 (en) | 2016-08-18 | 2018-02-22 | Alexion Pharmaceuticals, Inc. | Methods for treating tracheobronchomalacia |
| JP2020512363A (ja) | 2017-03-31 | 2020-04-23 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | 成人及び青年における低ホスファターゼ症（ｈｐｐ）を治療する方法 |
| CA3057142A1 (en) | 2017-04-03 | 2018-10-11 | American Gene Technologies International Inc. | Compositions and methods for treating phenylketonuria |
| KR102636351B1 (ko) * | 2017-05-19 | 2024-02-15 | 엔코디드 테라퓨틱스, 인크. | 고활성 조절 요소 |
| KR20200057766A (ko) * | 2017-10-02 | 2020-05-26 | 아메리칸 진 테크놀로지스 인터내셔널 인코포레이티드 | 페닐케톤뇨증을 치료하기 위한 프로모터 및 인핸서 조합을 포함하는 벡터 |
| SG11202004926WA (en) | 2017-12-01 | 2020-06-29 | Encoded Therapeutics Inc | Engineered dna binding proteins |
| US10610606B2 (en) | 2018-02-01 | 2020-04-07 | Homology Medicines, Inc. | Adeno-associated virus compositions for PAH gene transfer and methods of use thereof |
| US20190256867A1 (en) | 2018-02-01 | 2019-08-22 | Homology Medicines, Inc. | Adeno-associated virus compositions for restoring pah gene function and methods of use thereof |
| CN112041437A (zh) | 2018-02-19 | 2020-12-04 | 同源药物公司 | 用于恢复f8基因功能的腺相关病毒组合物和其使用方法 |
| JP2021519590A (ja) | 2018-03-30 | 2021-08-12 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | 糖タンパク質の製造 |
| WO2020033867A2 (en) | 2018-08-10 | 2020-02-13 | Alexion Pharmaceuticals, Inc. | Methods of treating neurofibromatosis type 1 and related conditions with alkaline phosphatase |
| US10842885B2 (en) | 2018-08-20 | 2020-11-24 | Ucl Business Ltd | Factor IX encoding nucleotides |
| GB201813528D0 (en) | 2018-08-20 | 2018-10-03 | Ucl Business Plc | Factor IX encoding nucleotides |
| WO2020081979A1 (en) * | 2018-10-19 | 2020-04-23 | Memorial Sloan-Kettering Cancer Center | Low dose radiation conditioning for immunotherapy |
| WO2020102723A1 (en) * | 2018-11-16 | 2020-05-22 | Encoded Therapeutics, Inc. | Compositions and methods for treating wilson's disease |
| JP7796645B2 (ja) | 2019-12-09 | 2026-01-09 | アレクシオン ファーマシューティカルズ， インコーポレイテッド | アルカリホスファターゼポリペプチド及びその使用方法 |
| TW202140791A (zh) | 2020-01-13 | 2021-11-01 | 美商霍蒙拉奇醫藥公司 | 治療苯酮尿症之方法 |
| BR112022014808A2 (pt) | 2020-01-31 | 2022-09-20 | Beth Israel Deaconess Medical Ct Inc | Composições e métodos para vacinas para prevenir e tratar infecção pelo coronavírus-sars-cov-2 |
| CN111088282B (zh) * | 2020-03-23 | 2020-08-28 | 上海安民生物技术有限公司 | Aavs1和h11安全港位点在重组表达蛋白中的应用 |
| TW202208632A (zh) | 2020-05-27 | 2022-03-01 | 美商同源醫藥公司 | 用於恢復ｐａｈ基因功能的腺相關病毒組成物及其使用方法 |
| CA3173631A1 (en) | 2021-02-12 | 2022-08-18 | Walter C. Voegtli | Alkaline phosphatase polypeptides and methods of use thereof |
Family Cites Families (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| IT1265794B1 (it) * | 1992-09-07 | 1996-12-03 | Angeletti P Ist Richerche Bio | Mammiferi transgenici non umani con un oncogene sotto il controllo di un promotore inducibile fegato-specifico |
| DE4339922C1 (de) * | 1993-09-03 | 1994-10-06 | Max Planck Gesellschaft | Vektor für Leber-Gentherapie |
| US6268212B1 (en) * | 1993-10-18 | 2001-07-31 | Amgen Inc. | Tissue specific transgene expression |
| DE4407859C1 (de) * | 1994-03-04 | 1995-03-02 | Max Planck Gesellschaft | Vektor für die leberspezifische Gentherapie |
| US5965541A (en) * | 1995-11-28 | 1999-10-12 | Genvec, Inc. | Vectors and methods for gene transfer to cells |
| DE19525900C1 (de) * | 1995-07-15 | 1996-12-12 | Max Planck Gesellschaft | Leberspezifischer Adenovirus-Expressionsvektor |
| US6521225B1 (en) * | 1996-09-06 | 2003-02-18 | Chiron Corporation | AAV vectors |
| US20020155095A1 (en) * | 1996-10-18 | 2002-10-24 | Tattanahalli L. Nagabhushan | Methods and compositions for delivery and expression of interferon-a nucleic acids |
| JP2002517180A (ja) * | 1997-12-05 | 2002-06-18 | ジ・イミユーン・リスポンス・コーポレーシヨン | 増大された発現を示す新規ベクターおよび遺伝子 |
| EP1045919A1 (de) * | 1998-01-16 | 2000-10-25 | Genzyme Corporation | Promotorelemente für andauernde genexpression |
-
2000
- 2000-11-15 AT AT00982132T patent/ATE360082T1/de active
- 2000-11-15 ES ES00982132T patent/ES2284545T3/es not_active Expired - Lifetime
- 2000-11-15 DE DE60034478T patent/DE60034478T2/de not_active Expired - Lifetime
- 2000-11-15 EP EP00982132A patent/EP1232276B8/de not_active Expired - Lifetime
- 2000-11-15 WO PCT/US2000/031444 patent/WO2001036620A2/en not_active Ceased
- 2000-11-15 AU AU19202/01A patent/AU1920201A/en not_active Abandoned
- 2000-11-15 EP EP07007344A patent/EP1818407A3/de not_active Withdrawn
-
2002
- 2002-05-06 US US10/139,763 patent/US7312324B2/en not_active Expired - Lifetime
-
2007
- 2007-11-13 US US11/938,906 patent/US20080070297A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| EP1232276B8 (de) | 2007-06-27 |
| WO2001036620A2 (en) | 2001-05-25 |
| EP1232276B1 (de) | 2007-04-18 |
| DE60034478D1 (de) | 2007-05-31 |
| EP1818407A3 (de) | 2007-08-29 |
| WO2001036620A3 (en) | 2002-02-14 |
| EP1818407A2 (de) | 2007-08-15 |
| DE60034478T2 (de) | 2008-01-10 |
| US7312324B2 (en) | 2007-12-25 |
| US20030017139A1 (en) | 2003-01-23 |
| US20080070297A1 (en) | 2008-03-20 |
| AU1920201A (en) | 2001-05-30 |
| EP1232276A2 (de) | 2002-08-21 |
| ES2284545T3 (es) | 2007-11-16 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| UEP | Publication of translation of european patent specification |
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