ATE428777T1 - Muskelspezifische expressionsvektoren - Google Patents
Muskelspezifische expressionsvektorenInfo
- Publication number
- ATE428777T1 ATE428777T1 AT02739449T AT02739449T ATE428777T1 AT E428777 T1 ATE428777 T1 AT E428777T1 AT 02739449 T AT02739449 T AT 02739449T AT 02739449 T AT02739449 T AT 02739449T AT E428777 T1 ATE428777 T1 AT E428777T1
- Authority
- AT
- Austria
- Prior art keywords
- muscle
- promoter
- elements
- expression vectors
- specific expression
- Prior art date
Links
- 239000013604 expression vector Substances 0.000 title 1
- 210000003205 muscle Anatomy 0.000 abstract 4
- 239000003623 enhancer Substances 0.000 abstract 3
- 102100036912 Desmin Human genes 0.000 abstract 2
- 108010044052 Desmin Proteins 0.000 abstract 2
- 108010059343 MM Form Creatine Kinase Proteins 0.000 abstract 2
- 102000013394 Troponin I Human genes 0.000 abstract 2
- 108010065729 Troponin I Proteins 0.000 abstract 2
- 210000005045 desmin Anatomy 0.000 abstract 2
- 102000007469 Actins Human genes 0.000 abstract 1
- 108010085238 Actins Proteins 0.000 abstract 1
- 108700028146 Genetic Enhancer Elements Proteins 0.000 abstract 1
- 210000000107 myocyte Anatomy 0.000 abstract 1
- 230000002085 persistent effect Effects 0.000 abstract 1
- 230000001105 regulatory 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
-
- 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
- A61P3/06—Antihyperlipidemics
-
- 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
- A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
- A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P7/00—Drugs for disorders of the blood or the extracellular fluid
-
- 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
- C12N2830/80—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)
- Engineering & Computer Science (AREA)
- Life Sciences & Earth Sciences (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- Diabetes (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Biotechnology (AREA)
- General Engineering & Computer Science (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Hematology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Molecular Biology (AREA)
- Obesity (AREA)
- Microbiology (AREA)
- Biochemistry (AREA)
- Endocrinology (AREA)
- Emergency Medicine (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US29330401P | 2001-05-24 | 2001-05-24 | |
| PCT/US2002/016748 WO2002095006A2 (en) | 2001-05-24 | 2002-05-22 | Muscle-specific expression vectors |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| ATE428777T1 true ATE428777T1 (de) | 2009-05-15 |
Family
ID=23128549
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| AT02739449T ATE428777T1 (de) | 2001-05-24 | 2002-05-22 | Muskelspezifische expressionsvektoren |
Country Status (7)
| Country | Link |
|---|---|
| US (2) | US20030100526A1 (enExample) |
| EP (2) | EP1390490B1 (enExample) |
| JP (1) | JP2004535801A (enExample) |
| AT (1) | ATE428777T1 (enExample) |
| CA (1) | CA2448120A1 (enExample) |
| DE (1) | DE60231975D1 (enExample) |
| WO (1) | WO2002095006A2 (enExample) |
Families Citing this family (67)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US20030157064A1 (en) * | 2001-11-09 | 2003-08-21 | Pascal Neuville | Chimeric promoters for controlling expression in muscle cells |
| US20080044393A1 (en) * | 2004-07-16 | 2008-02-21 | White Robert L | Retinal dystrophin transgene and methods of use thereof |
| WO2007046826A2 (en) * | 2004-12-01 | 2007-04-26 | The Regents Of The University Of Michigan | Compositions and methods for regulating cardiac performance |
| US8383601B2 (en) * | 2006-10-30 | 2013-02-26 | Thomas Jefferson University | Tissue specific gene therapy treatment |
| US20120322861A1 (en) * | 2007-02-23 | 2012-12-20 | Barry John Byrne | Compositions and Methods for Treating Diseases |
| CA2834729A1 (en) | 2011-04-06 | 2012-10-11 | Universite Pierre Et Marie Curie (Paris 6) | A skeletal muscle-specific enhancer |
| SMT202000445T1 (it) * | 2012-06-19 | 2020-09-10 | Univ Florida | Composizioni e metodi per trattare patologie |
| WO2014028762A1 (en) | 2012-08-15 | 2014-02-20 | University Of Virginia Patent Foundation | Compositions and methods for treating peripheral arterial disease |
| JP2016502858A (ja) | 2012-12-20 | 2016-02-01 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィを処置するための改善されたエキソンスキッピング組成物 |
| EA202090946A3 (ru) | 2013-03-14 | 2021-07-30 | Сарепта Терапьютикс, Инк. | Композиции, обеспечивающие пропускание экзонов, для лечения мышечной дистрофии |
| HRP20190382T1 (hr) | 2013-03-14 | 2019-04-19 | Sarepta Therapeutics, Inc. | Pripravci koji preskaču ekson za liječenje mišićne distrofije |
| WO2015035364A1 (en) * | 2013-09-09 | 2015-03-12 | University Of Washington Through Its Center For Commercialization | Proteins for targeting neuronal nitric oxide synthase to muscle sarcolemma and related methods of use |
| SG11201605906UA (en) | 2014-01-21 | 2016-08-30 | Univ Bruxelles | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| CA2980819C (en) | 2015-03-05 | 2024-03-05 | Peter Und Traudl Engelhorn-Stiftung Zur Forderung Der Lebenswissenschaften | System for the presentation of peptides on the cell surface |
| GB201508025D0 (en) | 2015-05-11 | 2015-06-24 | Ucl Business Plc | Fabry disease gene therapy |
| CN104894651B (zh) * | 2015-06-29 | 2017-04-12 | 天津诺禾医学检验所有限公司 | 微量起始dna的高通量测序文库构建方法及其所构建的高通量测序文库 |
| AU2016323575B2 (en) * | 2015-09-17 | 2022-10-27 | Research Institute At Nationwide Children's Hospital | Methods and materials for GALGT2 gene therapy |
| CN109219659B (zh) | 2016-04-14 | 2022-09-09 | 贝尼泰克生物制药有限公司 | 用于治疗眼咽型肌营养不良(opmd)的试剂及其用途 |
| US11202840B2 (en) | 2016-06-21 | 2021-12-21 | The Curators Of The University Of Missouri | Modified dystrophin proteins |
| TWI832036B (zh) | 2017-08-03 | 2024-02-11 | 美商航海家醫療公司 | 用於aav之遞送之組合物及方法 |
| EP4454654A3 (en) | 2017-10-16 | 2025-02-19 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (als) |
| US11434502B2 (en) | 2017-10-16 | 2022-09-06 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis (ALS) |
| MA51753A (fr) | 2018-02-05 | 2020-12-16 | Audentes Therapeutics Inc | Éléments de régulation de la transcription et utilisations associées |
| CN120665951A (zh) | 2018-02-07 | 2025-09-19 | 吉尼松公司 | 杂合调控元件 |
| US12129476B2 (en) | 2018-04-27 | 2024-10-29 | Voyager Therapeutics, Inc. | Methods for measuring the potency of AADC viral vectors |
| EP3794126A1 (en) | 2018-05-15 | 2021-03-24 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of parkinson's disease |
| WO2019222329A1 (en) | 2018-05-15 | 2019-11-21 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| CA3103963A1 (en) | 2018-07-02 | 2020-01-09 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| TW202035689A (zh) | 2018-10-04 | 2020-10-01 | 美商航海家醫療公司 | 測量病毒載體粒子的效價及強度之方法 |
| JP2022512621A (ja) | 2018-10-05 | 2022-02-07 | ボイジャー セラピューティクス インコーポレイテッド | Ａａｖ産生タンパク質をコードする操作された核酸コンストラクト |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| EP3867389A1 (en) | 2018-10-15 | 2021-08-25 | Voyager Therapeutics, Inc. | Expression vectors for large-scale production of raav in the baculovirus/sf9 system |
| CA3119618A1 (en) | 2018-11-16 | 2020-05-22 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| MX2021008542A (es) | 2019-01-18 | 2021-11-12 | Voyager Therapeutics Inc | Métodos y sistemas para la producción de partículas de aav. |
| US20220098614A1 (en) | 2019-02-28 | 2022-03-31 | Benitec IP Holdings Inc. | Compositions and Methods for Treating Oculopharyngeal Muscular Dystrophy (OPMD) |
| US20220162640A1 (en) * | 2019-04-08 | 2022-05-26 | Genethon | Hybrid promoters for muscle expression |
| WO2020227515A1 (en) | 2019-05-07 | 2020-11-12 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| SG11202111427RA (en) | 2019-05-28 | 2021-11-29 | Astellas Pharma Inc | Method for treating muscular dystrophy by targeting dmpk gene |
| BR112022002889A2 (pt) | 2019-08-16 | 2022-05-17 | Modalis Therapeutics Corp | Método para tratar distrofia muscular tendo como alvo o gene lama1 |
| WO2021046155A1 (en) | 2019-09-03 | 2021-03-11 | Voyager Therapeutics, Inc. | Vectorized editing of nucleic acids to correct overt mutations |
| WO2021202651A1 (en) | 2020-04-01 | 2021-10-07 | Voyager Therapeutics, Inc. | Redirection of tropism of aav capsids |
| BR112022020753A2 (pt) | 2020-04-15 | 2022-12-20 | Voyager Therapeutics Inc | Compostos de ligação a tau |
| AU2021273447B2 (en) | 2020-05-13 | 2026-03-05 | Voyager Therapeutics, Inc. | Redirection of tropism of AAV capsids |
| WO2021230385A1 (en) | 2020-05-15 | 2021-11-18 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting utrophin gene |
| WO2021247995A2 (en) | 2020-06-04 | 2021-12-09 | Voyager Therapeutics, Inc. | Compositions and methods of treating neuropathic pain |
| CA3192120A1 (en) * | 2020-09-11 | 2022-03-17 | James A. Williams | Muscle-specific hybrid promoter |
| US20220098615A1 (en) | 2020-09-30 | 2022-03-31 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
| EP4228610A1 (en) | 2020-10-13 | 2023-08-23 | Kriya Therapeutics, Inc. | Viral vector constructs for delivery of nucleic acids encoding cytokines and uses thereof for treating cancer |
| AR124143A1 (es) | 2020-11-25 | 2023-02-15 | Astellas Pharma Inc | Método para tratar la distrofia muscular mediante el direccionamiento al gen dmpk |
| WO2022164923A1 (en) | 2021-01-26 | 2022-08-04 | Kriya Therapeutics, Inc. | Vector constructs for delivery of nucleic acids encoding therapeutic anti-tnf antibodies and methods of using the same |
| EP4036238A1 (en) * | 2021-01-27 | 2022-08-03 | The University Court of the University of Edinburgh | Functional nucleic acid molecule and method |
| US20240294909A1 (en) | 2021-02-12 | 2024-09-05 | Merand Pharmaceuticals, Inc. | Agents, compositions, and methods for the treatment of hypoxia and ischemia-related disorders |
| EP4301860A1 (en) | 2021-03-04 | 2024-01-10 | Kriya Therapeutics, Inc. | Viral vector constructs incorporating dna for inhibiting toll like receptors and methods of using the same |
| CN117897492A (zh) * | 2021-08-04 | 2024-04-16 | 吉尼松公司 | 用于肌肉和cns中的基因表达的杂合启动子 |
| WO2023023055A1 (en) | 2021-08-16 | 2023-02-23 | Renagade Therapeutics Management Inc. | Compositions and methods for optimizing tropism of delivery systems for rna |
| TW202328067A (zh) | 2021-09-14 | 2023-07-16 | 美商雷納嘉德醫療管理公司 | 環狀脂質及其使用方法 |
| EP4402121A1 (en) | 2021-09-14 | 2024-07-24 | Renagade Therapeutics Management Inc. | Acyclic lipids and methods of use thereof |
| EP4405396A2 (en) | 2021-09-20 | 2024-07-31 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| MX2024008509A (es) | 2022-01-09 | 2024-07-19 | Kriya Therapeutics Inc | Construcciones de vectores para la distribucion de acidos nucleicos que codifican anticuerpos terapeuticos anti-igf-1r y metodos para usarlos. |
| JP2025508177A (ja) * | 2022-03-16 | 2025-03-21 | ラトガース ザ ステイト ユニバーシティー オブ ニュージャージー | 制御された筋肉特異的遺伝子送達 |
| WO2023196931A1 (en) | 2022-04-07 | 2023-10-12 | Renagade Therapeutics Management Inc. | Cyclic lipids and lipid nanoparticles (lnp) for the delivery of nucleic acids or peptides for use in vaccinating against infectious agents |
| EP4522757A2 (en) | 2022-05-13 | 2025-03-19 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| WO2023250388A1 (en) | 2022-06-22 | 2023-12-28 | Voyager Therapeutics, Inc. | Tau binding compounds |
| KR20250057779A (ko) * | 2022-07-11 | 2025-04-29 | 비타 테라퓨틱스 인코포레이티드 | 근이영양증을 치료하기 위한 치료적 위성 세포의 제조 |
| CN120019071A (zh) | 2022-09-15 | 2025-05-16 | 沃雅戈治疗公司 | Tau结合化合物 |
| KR20260051103A (ko) | 2023-07-07 | 2026-04-15 | 크리야 테라퓨틱스, 인크. | 안과 병리상태를 치료하기 위한 aav 벡터의 안구주위 전달 |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5266488A (en) * | 1988-04-06 | 1993-11-30 | The Regents Of The University Of California | Troponin T gene promoter and derivatives thereof |
| US5795872A (en) * | 1995-09-19 | 1998-08-18 | Pharmadigm, Inc. | DNA construct for immunization |
| US5846528A (en) * | 1996-01-18 | 1998-12-08 | Avigen, Inc. | Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence |
| US5858351A (en) * | 1996-01-18 | 1999-01-12 | Avigen, Inc. | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
| PT1071806E (pt) * | 1998-04-24 | 2004-09-30 | Univ Florida | Vector viral adeno-associado recombinante que codifica a alfa-1-antitripsina para terapia genetica |
| EP1287125B1 (en) * | 2000-04-28 | 2009-07-29 | Asklepios Biopharmaceutical, Inc. | Dna sequences encoding dystrophin minigenes and methods of use thereof |
| WO2001083703A2 (en) * | 2000-05-04 | 2001-11-08 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
-
2002
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-22 EP EP02739449A patent/EP1390490B1/en not_active Expired - Lifetime
- 2002-05-22 AT AT02739449T patent/ATE428777T1/de not_active IP Right Cessation
- 2002-05-22 WO PCT/US2002/016748 patent/WO2002095006A2/en not_active Ceased
- 2002-05-22 DE DE60231975T patent/DE60231975D1/de not_active Expired - Lifetime
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 2002-05-22 EP EP08103273A patent/EP2017338A1/en not_active Withdrawn
- 2002-05-24 US US10/156,604 patent/US20030100526A1/en not_active Abandoned
-
2007
- 2007-03-12 US US11/716,899 patent/US20110212529A1/en not_active Abandoned
Also Published As
| Publication number | Publication date |
|---|---|
| JP2004535801A (ja) | 2004-12-02 |
| WO2002095006A2 (en) | 2002-11-28 |
| EP1390490A4 (en) | 2005-05-11 |
| DE60231975D1 (de) | 2009-05-28 |
| EP1390490B1 (en) | 2009-04-15 |
| CA2448120A1 (en) | 2002-11-28 |
| WO2002095006A3 (en) | 2003-03-13 |
| US20030100526A1 (en) | 2003-05-29 |
| EP2017338A1 (en) | 2009-01-21 |
| US20110212529A1 (en) | 2011-09-01 |
| EP1390490A2 (en) | 2004-02-25 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| RER | Ceased as to paragraph 5 lit. 3 law introducing patent treaties |