DE60231975D1 - Muskelspezifische expressionsvektoren - Google Patents
Muskelspezifische expressionsvektorenInfo
- Publication number
- DE60231975D1 DE60231975D1 DE60231975T DE60231975T DE60231975D1 DE 60231975 D1 DE60231975 D1 DE 60231975D1 DE 60231975 T DE60231975 T DE 60231975T DE 60231975 T DE60231975 T DE 60231975T DE 60231975 D1 DE60231975 D1 DE 60231975D1
- Authority
- DE
- Germany
- Prior art keywords
- muscle
- promoter
- elements
- expression vectors
- specific expression
- 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.)
- Expired - Lifetime
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)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Organic Chemistry (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Chemical & Material Sciences (AREA)
- General Health & Medical Sciences (AREA)
- Diabetes (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Biotechnology (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Hematology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Molecular Biology (AREA)
- Microbiology (AREA)
- Obesity (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 |
|---|---|
| DE60231975D1 true DE60231975D1 (de) | 2009-05-28 |
Family
ID=23128549
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| DE60231975T Expired - Lifetime DE60231975D1 (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 (66)
| 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 |
| US8557969B2 (en) | 2004-12-01 | 2013-10-15 | The Regents Of The University Of Michigan | Compositions and methods for regulating cardiac performance |
| WO2008054713A2 (en) * | 2006-10-30 | 2008-05-08 | 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 |
| US20140031417A1 (en) | 2011-04-06 | 2014-01-30 | Universite Pierre Et Marie Curie (Paris 6) | Skeletal muscle-specific enhancer |
| EP4124346A3 (en) * | 2012-06-19 | 2023-04-19 | University of Florida Research Foundation, Inc. | Compositions and methods for treating diseases |
| US9845465B2 (en) | 2012-08-15 | 2017-12-19 | University Of Virginia Patent Foundation | Compositions and methods for treating peripheral arterial disease |
| WO2014100714A1 (en) | 2012-12-20 | 2014-06-26 | Sarepta Therapeutics, Inc. | Improved exon skipping compositions for treating muscular dystrophy |
| NZ775701A (en) | 2013-03-14 | 2022-08-26 | Sarepta Therapeutics Inc | Exon skipping compositions for treating muscular dystrophy |
| HUE042218T2 (hu) | 2013-03-14 | 2019-06-28 | Sarepta Therapeutics Inc | Exonátugrást elõsegítõ készítmények izomdisztrófia kezelésére |
| 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 |
| CA2936726C (en) | 2014-01-21 | 2024-04-30 | Vrije Universiteit Brussel | Muscle-specific nucleic acid regulatory elements and methods and use thereof |
| EP3265480B1 (de) | 2015-03-05 | 2021-03-24 | Peter und Traudl Engelhorn-Stiftung zur Förderung der Lebenswissenschaften | System zur präsentation von peptiden auf der zelloberfläche |
| 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 |
| IL258005B2 (en) * | 2015-09-17 | 2025-01-01 | Res Inst Nationwide Childrens Hospital | Methods and materials for gene therapy for beta-4,1-N-acetylgalactosaminyltransferase 2 |
| KR102353847B1 (ko) | 2016-04-14 | 2022-01-21 | 베니텍 바이오파마 리미티드 | 안구인두 근이영양증(opmd)의 치료용 시약 및 이의 용도 |
| EP4011206B1 (en) | 2016-06-21 | 2023-11-01 | The Curators of the University of Missouri | Modified dystrophin proteins |
| EP3662060A2 (en) | 2017-08-03 | 2020-06-10 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| 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 |
| MX2020012077A (es) | 2018-05-15 | 2021-03-09 | Voyager Therapeutics Inc | Composiciones y metodos para el tratamiento de la enfermedad de parkinson. |
| TW202015742A (zh) | 2018-05-15 | 2020-05-01 | 美商航海家醫療公司 | 投遞腺相關病毒（ａａｖ）之組成物和方法 |
| WO2019241486A1 (en) | 2018-06-13 | 2019-12-19 | Voyager Therapeutics, Inc. | Engineered 5' untranslated regions (5' utr) for aav production |
| AU2019299861A1 (en) | 2018-07-02 | 2021-01-14 | Voyager Therapeutics, Inc. | Treatment of amyotrophic lateral sclerosis and disorders associated with the spinal cord |
| EP3861113A1 (en) | 2018-10-04 | 2021-08-11 | Voyager Therapeutics, Inc. | Methods for measuring the titer and potency of viral vector particles |
| EP3861107A1 (en) | 2018-10-05 | 2021-08-11 | Voyager Therapeutics, Inc. | Engineered nucleic acid constructs encoding aav production proteins |
| WO2020077165A1 (en) | 2018-10-12 | 2020-04-16 | Voyager Therapeutics, Inc. | Compositions and methods for delivery of aav |
| JP2022505106A (ja) | 2018-10-15 | 2022-01-14 | ボイジャー セラピューティクス インコーポレイテッド | バキュロウイルス／Ｓｆ９システムにおけるｒＡＡＶの大規模産生のための発現ベクター |
| BR112021009481A2 (pt) | 2018-11-16 | 2021-08-17 | Astellas Pharma Inc. | método para tratamento da distrofia muscular direcionando gene de utrofina. |
| CN113631225A (zh) | 2019-01-18 | 2021-11-09 | 沃雅戈治疗公司 | 用于生产aav颗粒的方法和系统 |
| EP3931317A4 (en) | 2019-02-28 | 2023-01-04 | Benitec IP Holdings Inc. | COMPOSITIONS AND METHODS FOR TREATMENT OF OCULOPHARYNGEAL MUSCULAR DYSTROPHY (OPMD) |
| CN113747926A (zh) * | 2019-04-08 | 2021-12-03 | 吉尼松公司 | 用于肌肉表达的杂合启动子 |
| EP3966227A1 (en) | 2019-05-07 | 2022-03-16 | Voyager Therapeutics, Inc. | Compositions and methods for the vectored augmentation of protein destruction, expression and/or regulation |
| CA3135271A1 (en) | 2019-05-28 | 2020-12-03 | Astellas Pharma Inc. | Method for treating muscular dystrophy by targeting dmpk gene |
| TW202112797A (zh) | 2019-08-16 | 2021-04-01 | 日商摩大力斯醫療公司 | 經由靶向lama1基因治療肌肉萎縮症的方法 |
| 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 |
| MX2022012752A (es) | 2020-04-15 | 2023-01-16 | Voyager Therapeutics Inc | Compuestos de union a tau. |
| US20230203102A1 (en) | 2020-05-13 | 2023-06-29 | 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 |
| WO2022073041A2 (en) | 2020-09-30 | 2022-04-07 | NGGT, Inc. | Dual functional expression vectors and methods of use thereof |
| JP2023548746A (ja) | 2020-10-13 | 2023-11-21 | クリヤ セラピューティクス インコーポレイテッド | サイトカインをコードする核酸を送達するためのウイルスベクターコンストラクトおよびがんを処置するためのその使用 |
| 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 |
| CN117280030A (zh) | 2021-02-12 | 2023-12-22 | 梅兰德制药公司 | 用于治疗缺氧和缺血相关病症的药剂、组合物和方法 |
| 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 |
| EP4402121A1 (en) | 2021-09-14 | 2024-07-24 | Renagade Therapeutics Management Inc. | Acyclic lipids and methods of use thereof |
| EP4402123A1 (en) | 2021-09-14 | 2024-07-24 | Renagade Therapeutics Management Inc. | Cyclic 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. |
| CN118829729A (zh) * | 2022-03-16 | 2024-10-22 | 罗格斯新泽西州立大学 | 受控的肌肉特异性基因递送 |
| CA3255619A1 (en) | 2022-04-07 | 2023-10-12 | Renagade Therapeutics Man Inc | CYCLIC LIPIDS AND LIPID NANOPARTICLES (LPNs) FOR THE SUPPLY OF NUCLEIC ACIDS OR PEPTIDES INTENDED FOR USE IN VACCINATION AGAINST INFECTIOUS AGENTS |
| EP4522757A2 (en) | 2022-05-13 | 2025-03-19 | Voyager Therapeutics, Inc. | Compositions and methods for the treatment of her2 positive cancer |
| EP4543473A1 (en) | 2022-06-22 | 2025-04-30 | Voyager Therapeutics, Inc. | Tau binding compounds |
| WO2024015329A1 (en) * | 2022-07-11 | 2024-01-18 | Vita Therapeutics, Inc. | Manufacturing of therapeutic satellite cells for treating muscular dystrophy |
| KR20250069606A (ko) | 2022-09-15 | 2025-05-19 | 보이저 테라퓨틱스, 인크. | 타우 결합 화합물 |
| WO2025014832A1 (en) | 2023-07-07 | 2025-01-16 | Kriya Therapeutics, Inc. | Periocular delivery of aav vectors for treating ophthalmic pathologies |
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 |
| CA2326327C (en) * | 1998-04-24 | 2009-06-16 | University Of Florida | Recombinant adeno-associated virus vector encoding alpha-1-antitrypsin serum protein |
| CN100422320C (zh) * | 2000-04-28 | 2008-10-01 | 肖啸 | 编码抗肌萎缩蛋白小基因的dna序列及其使用方法 |
| US6593110B2 (en) * | 2000-05-04 | 2003-07-15 | California Institute Of Technology | Checkpoint-activating oligonucleotides |
-
2002
- 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 EP EP08103273A patent/EP2017338A1/en not_active Withdrawn
- 2002-05-22 CA CA002448120A patent/CA2448120A1/en not_active Abandoned
- 2002-05-22 JP JP2002592469A patent/JP2004535801A/ja active Pending
- 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 |
|---|---|
| CA2448120A1 (en) | 2002-11-28 |
| EP1390490A2 (en) | 2004-02-25 |
| WO2002095006A2 (en) | 2002-11-28 |
| JP2004535801A (ja) | 2004-12-02 |
| EP1390490A4 (en) | 2005-05-11 |
| US20110212529A1 (en) | 2011-09-01 |
| EP2017338A1 (en) | 2009-01-21 |
| WO2002095006A3 (en) | 2003-03-13 |
| US20030100526A1 (en) | 2003-05-29 |
| EP1390490B1 (en) | 2009-04-15 |
| ATE428777T1 (de) | 2009-05-15 |
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Legal Events
| Date | Code | Title | Description |
|---|---|---|---|
| 8364 | No opposition during term of opposition |