CA2942776C - Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter - Google Patents
Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter Download PDFInfo
- Publication number
- CA2942776C CA2942776C CA2942776A CA2942776A CA2942776C CA 2942776 C CA2942776 C CA 2942776C CA 2942776 A CA2942776 A CA 2942776A CA 2942776 A CA2942776 A CA 2942776A CA 2942776 C CA2942776 C CA 2942776C
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- seq
- sequence
- cone
- polynucleotide
- expression
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
- A61K35/66—Microorganisms or materials therefrom
- A61K35/76—Viruses; Subviral particles; Bacteriophages
- A61K35/761—Adenovirus
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- 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
-
- 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
- C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P27/00—Drugs for disorders of the senses
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
-
- 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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—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
- C12N2800/00—Nucleic acids vectors
- C12N2800/24—Vectors characterised by the absence of particular element, e.g. selectable marker, viral origin of replication
-
- 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
- C12N2830/30—Vector systems having a special element relevant for transcription being an enhancer not forming part of the promoter region
-
- 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
- C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Genetics & Genomics (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Virology (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- Biotechnology (AREA)
- Medicinal Chemistry (AREA)
- Biomedical Technology (AREA)
- General Engineering & Computer Science (AREA)
- Microbiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Public Health (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- Immunology (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Mycology (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Chemical Kinetics & Catalysis (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Cell Biology (AREA)
- Toxicology (AREA)
- Gastroenterology & Hepatology (AREA)
- Peptides Or Proteins (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201461954330P | 2014-03-17 | 2014-03-17 | |
| US61/954,330 | 2014-03-17 | ||
| US201562127185P | 2015-03-02 | 2015-03-02 | |
| US62/127,185 | 2015-03-02 | ||
| PCT/US2015/021087 WO2015142941A1 (en) | 2014-03-17 | 2015-03-17 | Compositions and methods for enhanced gene expression in cone cells |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| CA2942776A1 CA2942776A1 (en) | 2015-09-24 |
| CA2942776C true CA2942776C (en) | 2023-01-24 |
Family
ID=54068212
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CA2942776A Active CA2942776C (en) | 2014-03-17 | 2015-03-17 | Polyneucleotide cassette and expression vector for expression of a gene in cone cells using truncated m-opsin promoter |
Country Status (11)
| Country | Link |
|---|---|
| US (3) | US10000741B2 (https=) |
| EP (2) | EP3119798B1 (https=) |
| JP (4) | JP6689249B2 (https=) |
| KR (2) | KR102537394B1 (https=) |
| CN (2) | CN106414474B (https=) |
| AU (3) | AU2015231439B2 (https=) |
| CA (1) | CA2942776C (https=) |
| ES (2) | ES3042252T3 (https=) |
| IL (2) | IL259321B2 (https=) |
| SG (2) | SG11201607738WA (https=) |
| WO (1) | WO2015142941A1 (https=) |
Families Citing this family (71)
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|---|---|---|---|---|
| WO2011034947A2 (en) | 2009-09-15 | 2011-03-24 | University Of Washington | Reagents and methods for modulating cone photoreceptor activity |
| HRP20171334T1 (hr) | 2011-04-22 | 2017-11-17 | The Regents Of The University Of California | Virioni adeno-povezanog virusa s kapsid varijantom i postupci njihove upotrebe |
| TWI775096B (zh) | 2012-05-15 | 2022-08-21 | 澳大利亞商艾佛蘭屈澳洲私營有限公司 | 使用腺相關病毒(aav)sflt-1治療老年性黃斑部退化(amd) |
| US11136557B2 (en) | 2013-05-31 | 2021-10-05 | The Regents Of The University Of California | Adeno-associated virus variants and methods of use thereof |
| KR102537394B1 (ko) | 2014-03-17 | 2023-05-30 | 애드베룸 바이오테크놀로지스, 인코포레이티드 | 원추세포에서 증강된 유전자 발현을 위한 조성물 및 방법 |
| MY187898A (en) * | 2015-03-02 | 2021-10-27 | Adverum Biotechnologies Inc | Compositions and methods for intravitreal delivery of polynucleotides to retinal cones |
| BR112018011059A2 (pt) | 2015-12-03 | 2018-11-21 | Friedrich Miescher Institute For Biomedical Research | synp161, um promotor para a expressão específica de genes em fotoreceptores de haste |
| WO2017093934A1 (en) * | 2015-12-03 | 2017-06-08 | Friedrich Miescher Institute For Biomedical Research | Synp160, a promoter for the specific expression of genes in rod photoreceptors |
| US11090392B2 (en) | 2015-12-14 | 2021-08-17 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| GB2545763A (en) | 2015-12-23 | 2017-06-28 | Adverum Biotechnologies Inc | Mutant viral capsid libraries and related systems and methods |
| CN107287239B (zh) * | 2016-04-11 | 2020-09-22 | 厦门继景生物技术有限责任公司 | 一种用于视网膜色素变性的基因治疗载体及药物 |
| ES2908467T3 (es) * | 2016-04-29 | 2022-04-29 | Adverum Biotechnologies Inc | Evasión de anticuerpos neutralizantes por un virus adenoasociado recombinante |
| US12173305B2 (en) | 2016-05-26 | 2024-12-24 | University Of Iowa Research Foundation | cis and trans requirements for terminal resolution of human bocavirus 1 |
| ES2916344T3 (es) * | 2016-06-16 | 2022-06-30 | Adverum Biotechnologies Inc | Composiciones y métodos para reducir la neovascularización ocular |
| US11197936B2 (en) * | 2016-07-08 | 2021-12-14 | The Trustees Of The University Of Pennsylvania | Methods and compositions for treatment of disorders and diseases involving RDH12 |
| EP3827812B1 (en) | 2016-07-29 | 2025-10-29 | The Regents of the University of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
| CA3040179A1 (en) | 2016-10-19 | 2018-04-26 | Adverum Biotechnologies, Inc. | Modified aav capsids and uses thereof |
| WO2018134168A1 (en) * | 2017-01-17 | 2018-07-26 | INSERM (Institut National de la Santé et de la Recherche Médicale) | Methods of expressing a polynucleotide of interest in the cone photoreceptors |
| BR112019017327A2 (pt) * | 2017-03-01 | 2020-04-14 | Univ Pennsylvania | terapia gênica para distúrbios oculares |
| CN110546257B (zh) | 2017-03-17 | 2024-03-01 | 阿德夫拉姆生物技术股份有限公司 | 用于增强基因表达的组合物和方法 |
| WO2018208703A1 (en) * | 2017-05-08 | 2018-11-15 | Lambdavision Incorporated | A method for stimulating retinal cells and treating vision loss |
| US11827898B2 (en) | 2017-06-14 | 2023-11-28 | The Trustees Of The University Of Pennsylvania | Gene therapy for ocular disorders |
| WO2019006182A1 (en) | 2017-06-30 | 2019-01-03 | The Regents Of The University Of California | VIRIONS OF ADENO-ASSOCIATED VIRUSES OF CAPSID VARIANTS AND METHODS OF USE THEREOF |
| JP2020534788A (ja) | 2017-08-28 | 2020-12-03 | ザ リージェンツ オブ ザ ユニバーシティ オブ カリフォルニア | アデノ随伴ウイルスカプシド変異体及びその使用方法 |
| SG11202002276VA (en) * | 2017-09-20 | 2020-04-29 | 4D Molecular Therapeutics Inc | Adeno-associated virus variant capsids and methods of use thereof |
| JP7420710B2 (ja) * | 2017-10-20 | 2024-01-23 | アンセルム（アンスティチュート・ナシオナル・ドゥ・ラ・サンテ・エ・ドゥ・ラ・ルシェルシュ・メディカル） | 組換えａａｖ９由来ベクターの治療有効量の網膜下送達を含む、対象の錐体視細胞において目的のポリヌクレオチドを発現させる方法 |
| SG11202003778QA (en) | 2017-11-13 | 2020-05-28 | Univ California | Compositions and methods for enhancing visual function |
| SG11202004527YA (en) | 2017-11-15 | 2020-06-29 | Univ Michigan Regents | Viral vectors comprising rdh12 coding regions and methods of treating retinal dystrophies |
| WO2019113225A1 (en) * | 2017-12-05 | 2019-06-13 | University Of Washington | Compositions and methods for enhancing functional expression of therapeutic genes in photoreceptors |
| GB201800546D0 (en) * | 2018-01-12 | 2018-02-28 | Ucl Business Plc | Treatment |
| WO2019169291A1 (en) | 2018-03-02 | 2019-09-06 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Use of il-34 to treat retinal inflammation and neurodegeneration |
| CN109097364B (zh) * | 2018-09-03 | 2022-02-11 | 深圳广三系农业科技有限公司 | 一种植物胚乳特异性表达启动子pOsEnS100的鉴定和应用 |
| EP3893718A4 (en) | 2018-12-12 | 2022-11-09 | Tesseract Health, Inc. | BIOMETRIC IDENTIFICATION SYSTEMS AND RELATED DEVICES |
| WO2020167770A1 (en) * | 2019-02-11 | 2020-08-20 | President And Fellows Of Harvard College | Txnip and ldhb compositions and methods for the treatment of degenerative ocular diseases |
| US12270033B2 (en) | 2019-02-15 | 2025-04-08 | Exhaura, Ltd. | Dual leucine zipper kinase inhibitors for gene therapy |
| EP3955970A1 (en) * | 2019-04-15 | 2022-02-23 | University Of Iowa Research Foundation | Methods and compositions for transgene expression |
| CA3137135A1 (en) * | 2019-04-19 | 2020-10-22 | University Of Massachusetts | Gene therapies for stargardt disease (abca4) |
| US11737665B2 (en) | 2019-06-21 | 2023-08-29 | Tesseract Health, Inc. | Multi-modal eye imaging with shared optical path |
| WO2021062012A1 (en) | 2019-09-25 | 2021-04-01 | Emory University | Use of klk10 and engineered derivatizations thereof |
| US20240108756A1 (en) * | 2019-10-17 | 2024-04-04 | The Johns Hopkins University | Compositions and methods for using alternative splicing to control specificity of gene therapy |
| JP7703543B2 (ja) | 2020-01-17 | 2025-07-07 | ザ ユナイテッド ステイツ オブ アメリカ アズ リプリゼンテッド バイ ザ セクレタリー、デパートメント オブ ヘルス アンド ヒューマン サービシーズ | Ｃｒｘ常染色体優性網膜症の処置のための遺伝子治療 |
| US12257320B2 (en) | 2020-03-11 | 2025-03-25 | Massachusetts Eye And Ear Infirmary | Gene therapy for NMNAT1-associated retinal degeneration |
| WO2021204407A1 (en) | 2020-04-10 | 2021-10-14 | Sorbonne Université | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| AU2021263563B2 (en) | 2020-04-27 | 2023-02-02 | 4D Molecular Therapeutics Inc. | Codon optimized GLA genes and uses thereof |
| CN111429547B (zh) * | 2020-04-27 | 2023-05-30 | 中国人民解放军空军工程大学 | 一种基于假同色搜索的异常色觉测试图合成方法 |
| WO2022020548A2 (en) * | 2020-07-23 | 2022-01-27 | President And Fellows Of Harvard College | Variant txnip compositions and methods of use thereof for the treatment of degenerative ocular diseases |
| WO2022026632A2 (en) * | 2020-07-29 | 2022-02-03 | University Of Florida Research Foundation, Incorporated | Improved aav-mediated x-linked retinoschisis therapies |
| CN112062827B (zh) * | 2020-09-16 | 2022-10-21 | 中国人民解放军军事科学院军事医学研究院 | Cep55蛋白在调控纤毛去组装和制备纤毛相关疾病模型中的应用 |
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| US12385064B2 (en) * | 2021-03-26 | 2025-08-12 | Adverum Biotechnologies, Inc. | Intravitreal dosing for delivery of polynucleotides to retinal cones |
| GB202104611D0 (en) * | 2021-03-31 | 2021-05-12 | Univ Oxford Innovation Ltd | Gene therapy for retinal disease |
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| CN113408798B (zh) * | 2021-06-14 | 2022-03-29 | 华中师范大学 | 一种面向色觉异常人群的无障碍vr教学资源色彩优化方法 |
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| KR102876040B1 (ko) | 2021-07-30 | 2025-10-23 | 주식회사 엘지에너지솔루션 | 이차 전지용 전극, 이의 제조방법, 및 상기 전극을 포함하는 이차 전지 |
| AU2022340595A1 (en) * | 2021-09-06 | 2024-04-18 | Huidagene Therapeutics (Singapore) Pte. Ltd. | Treatment of rpe65-associated eye diseases and disorders |
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| WO2023122804A1 (en) * | 2021-12-24 | 2023-06-29 | The Trustees Of The University Of Pennsylvania | Compositions and methods comprising a cardiac-specific promoter |
| WO2023205758A2 (en) * | 2022-04-22 | 2023-10-26 | West Virginia University Board of Governors on behalf of West Virginia University | Raav-cone opsin compositions and methods for treating blue cone monochromacy and color blindness |
| CN114848850B (zh) * | 2022-04-28 | 2023-12-15 | 武汉中眸生物科技有限责任公司 | Rs1基因在制备xlrs治疗剂中的应用及治疗剂 |
| WO2024011109A2 (en) * | 2022-07-06 | 2024-01-11 | Adverum Biotechnologies, Inc. | Compositions and methods for treatment of achromotopsia |
| TW202421773A (zh) * | 2022-09-30 | 2024-06-01 | 美商聖堤生物科技股份有限公司 | 用於光受體之細胞型特異性調控元件 |
| EP4357359A1 (en) | 2022-10-20 | 2024-04-24 | Sparingvision | G-protein-gated-k+ channel-mediated enhancements in light sensitivity in rod-cone dystrophy (rcd) |
| CN118791624B (zh) * | 2023-04-11 | 2026-04-03 | 苏州星明优健生物技术有限公司 | 用于治疗视网膜神经退行性疾病的新型gpcr嵌合视蛋白 |
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| WO2025261326A1 (zh) * | 2024-06-20 | 2025-12-26 | 云舟生物科技(广州)股份有限公司 | 视锥细胞特异性启动子以及其用途 |
| CN118562953A (zh) * | 2024-06-21 | 2024-08-30 | 复旦大学附属眼耳鼻喉科医院 | 检测opn1lw与opn1mw基因单倍体型的试剂在近视诊断试剂盒中的应用及产品 |
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