CR20200236A - Synpiii, un promotor para la expresión específica de genes en el epitelio pigmentario retiniano - Google Patents
Synpiii, un promotor para la expresión específica de genes en el epitelio pigmentario retinianoInfo
- Publication number
- CR20200236A CR20200236A CR20200236A CR20200236A CR20200236A CR 20200236 A CR20200236 A CR 20200236A CR 20200236 A CR20200236 A CR 20200236A CR 20200236 A CR20200236 A CR 20200236A CR 20200236 A CR20200236 A CR 20200236A
- Authority
- CR
- Costa Rica
- Prior art keywords
- nucleic acid
- retinal pigment
- pigment epithelium
- synpiii
- promoter
- Prior art date
Links
- 108090000623 proteins and genes Proteins 0.000 title abstract 3
- 210000003583 retinal pigment epithelium Anatomy 0.000 title abstract 2
- 150000007523 nucleic acids Chemical class 0.000 abstract 5
- 108091028043 Nucleic acid sequence Proteins 0.000 abstract 3
- 108020004707 nucleic acids Proteins 0.000 abstract 2
- 102000039446 nucleic acids Human genes 0.000 abstract 2
Classifications
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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
-
- 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
-
- 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
- A61K48/0075—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 delivery route, e.g. oral, subcutaneous
-
- 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
- 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
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)
- General Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Organic Chemistry (AREA)
- Wood Science & Technology (AREA)
- General Health & Medical Sciences (AREA)
- Molecular Biology (AREA)
- Biochemistry (AREA)
- Biophysics (AREA)
- Microbiology (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Epidemiology (AREA)
- Pharmacology & Pharmacy (AREA)
- Medicinal Chemistry (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (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)
Abstract
<p>La presente invención aporta una molécula de ácido nucleico aislada que comprende o consiste en la secuencia de ácido nucleico de SEQ ID NO: 1 o una secuencia de ácido nucleico de, al menos, 1000 pb; que tiene al menos un 80% de identidad con la secuencia SEQ ID NO: 1 antes indicada, en el que dicha molécula de ácido nucleico aislada conduce específicamente a la expresión en células del epitelio pigmentario de la retina de un gen cuando se une operativamente a una secuencia de ácido nucleico que codifica el gen antes dicho.</p>
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17204750 | 2017-11-30 | ||
| PCT/EP2018/082880 WO2019106035A1 (en) | 2017-11-30 | 2018-11-28 | Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CR20200236A true CR20200236A (es) | 2020-10-19 |
Family
ID=60543416
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CR20200236A CR20200236A (es) | 2017-11-30 | 2018-11-28 | Synpiii, un promotor para la expresión específica de genes en el epitelio pigmentario retiniano |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US11591615B2 (es) |
| EP (1) | EP3717651A1 (es) |
| JP (1) | JP2021503934A (es) |
| KR (1) | KR20200091432A (es) |
| CN (1) | CN111542611A (es) |
| AU (1) | AU2018376259A1 (es) |
| BR (1) | BR112020010928A2 (es) |
| CA (1) | CA3083682A1 (es) |
| CR (1) | CR20200236A (es) |
| IL (1) | IL274957A (es) |
| MX (1) | MX2020005649A (es) |
| PH (1) | PH12020550748A1 (es) |
| RU (1) | RU2020121408A (es) |
| SG (1) | SG11202005024QA (es) |
| WO (1) | WO2019106035A1 (es) |
Families Citing this family (14)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN101970013B (zh) | 2008-04-18 | 2014-11-05 | 诺瓦提斯研究基金会弗里德里克·米谢尔生物医学研究所 | 治疗失明的新型治疗工具和方法 |
| WO2016174624A1 (en) | 2015-04-30 | 2016-11-03 | Friedrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in müller cells |
| JP6898920B2 (ja) | 2015-09-15 | 2021-07-07 | フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ | 光受容器のターゲッティングにより失明を治療するための新規な治療用ツールおよび方法 |
| EP3362101B1 (en) | 2015-10-14 | 2021-05-19 | Friedrich Miescher Institute for Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
| ES2909444T3 (es) | 2015-12-03 | 2022-05-06 | Friedrich Miescher Institute For Biomedical Res | Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones |
| CA3006952A1 (en) | 2015-12-03 | 2017-06-08 | Friedrich Miescher Institute For Biomedical Research | Synp161, a promoter for the specific expression of genes in rod photoreceptors |
| EP3383440B1 (en) | 2015-12-03 | 2021-06-30 | Friedrich Miescher Institute for Biomedical Research | Synp162, a promoter for the specific expression of genes in rod photoreceptors |
| CN108474001B (zh) | 2015-12-03 | 2022-04-08 | 弗里德里克·米谢尔生物医学研究所 | SynP160，用于基因在视杆光感受器中特异性表达的启动子 |
| AU2017354086C1 (en) | 2016-11-02 | 2021-05-06 | Friedrich Miescher Institute For Biomedical Research | SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
| CN110392582A (zh) | 2017-02-08 | 2019-10-29 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP88 |
| AU2018369975B2 (en) | 2017-11-15 | 2022-05-19 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
| KR20200091432A (ko) | 2017-11-30 | 2020-07-30 | 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 | 망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII |
| WO2024110770A1 (en) * | 2022-11-22 | 2024-05-30 | INSERM (Institut National de la Santé et de la Recherche Médicale) | A new promoter for retinal pigment epithelium (rpe) targeted gene therapy |
| AU2024260900A1 (en) * | 2023-04-27 | 2025-11-13 | Korea Research Institute Of Bioscience And Biotechnology | Promoter for gene expression in retinal cells, and vector system comprising same |
Family Cites Families (39)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB9206874D0 (en) | 1992-03-30 | 1992-05-13 | Connaught Lab | Generation of improved inducible mammalian expression vectors |
| NZ511037A (en) | 1998-09-17 | 2005-02-25 | Univ Florida | Methods for treatment of degenerative retinal diseases |
| WO2007131180A2 (en) | 2006-05-04 | 2007-11-15 | Wayne State University | Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids |
| EP1891976A1 (en) | 2006-08-23 | 2008-02-27 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research | Use of light sensitive genes |
| WO2009112448A1 (en) | 2008-03-11 | 2009-09-17 | Novartis Forschungsstiftung, Zweigniederlassung | Novel tool for the analysis of neural circuits |
| CN101970013B (zh) | 2008-04-18 | 2014-11-05 | 诺瓦提斯研究基金会弗里德里克·米谢尔生物医学研究所 | 治疗失明的新型治疗工具和方法 |
| WO2012094560A2 (en) | 2011-01-07 | 2012-07-12 | Applied Genetic Technologies Corporation | Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases |
| EP2888368A1 (en) * | 2012-08-27 | 2015-07-01 | Friedrich Miescher Institute for Biomedical Research | Retinal off circuit-specific promoter |
| WO2015118507A1 (en) | 2014-02-10 | 2015-08-13 | Friedrich Miescher Institute For Biomedical Research | Aii retinal amacrine cell-specific promoter |
| ES2748282T3 (es) | 2014-02-11 | 2020-03-16 | Friedrich Miescher Institute For Biomedical Res | Promotor específico de células de Müller |
| GB201502137D0 (en) * | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
| WO2016174624A1 (en) | 2015-04-30 | 2016-11-03 | Friedrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in müller cells |
| JP6898920B2 (ja) | 2015-09-15 | 2021-07-07 | フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ | 光受容器のターゲッティングにより失明を治療するための新規な治療用ツールおよび方法 |
| EP3362101B1 (en) | 2015-10-14 | 2021-05-19 | Friedrich Miescher Institute for Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
| CA3006952A1 (en) | 2015-12-03 | 2017-06-08 | Friedrich Miescher Institute For Biomedical Research | Synp161, a promoter for the specific expression of genes in rod photoreceptors |
| EP3383440B1 (en) | 2015-12-03 | 2021-06-30 | Friedrich Miescher Institute for Biomedical Research | Synp162, a promoter for the specific expression of genes in rod photoreceptors |
| ES2909444T3 (es) | 2015-12-03 | 2022-05-06 | Friedrich Miescher Institute For Biomedical Res | Synp159, un promotor para la expresión específica de genes en los fotorreceptores de los bastones |
| CN108474001B (zh) | 2015-12-03 | 2022-04-08 | 弗里德里克·米谢尔生物医学研究所 | SynP160，用于基因在视杆光感受器中特异性表达的启动子 |
| EP3384036B1 (en) | 2015-12-04 | 2021-02-24 | Sorbonne Université | Promoters and uses thereof |
| US20190209708A1 (en) | 2016-05-17 | 2019-07-11 | Friedrich Miescher Institute For Biomedical Research | Novel therapeutic tools and methods for treating blindness |
| AU2017354086C1 (en) | 2016-11-02 | 2021-05-06 | Friedrich Miescher Institute For Biomedical Research | SynP198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
| WO2018099975A1 (en) | 2016-12-01 | 2018-06-07 | Friedrich Miescher Institute For Biomedical Research | Synpi, a promoter for the specific expression of genes in interneurons |
| WO2018099974A1 (en) | 2016-12-01 | 2018-06-07 | Friedrich Miescher Institute For Biomedical Research | Synp107, a promoter for the specific expression of genes in interneurons |
| CN110392582A (zh) | 2017-02-08 | 2019-10-29 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在视网膜神经节细胞中特异性表达的启动子SynP88 |
| AU2018369975B2 (en) | 2017-11-15 | 2022-05-19 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
| KR20200091432A (ko) | 2017-11-30 | 2020-07-30 | 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 | 망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII |
| CR20200235A (es) | 2017-11-30 | 2020-10-19 | Friedrich Miescher Institute For Biomedical Res | Synp61, un promotor específico de células del epitelio pigmentario retiniano de primates |
| JP2022505516A (ja) | 2018-10-25 | 2022-01-14 | フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ | 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターＳｙｎＰ１７（ＰｒｏＢ１） |
| JP2022512780A (ja) | 2018-10-25 | 2022-02-07 | フリードリッヒ ミーシェー インスティトゥート フォー バイオメディカル リサーチ | 網膜神経節細胞中の遺伝子の特異的発現のためのプロモーターＳｙｎＰ１９４（ＰｒｏＢ１５） |
| US20210388386A1 (en) | 2018-10-25 | 2021-12-16 | Friedrich Miescher Institute For Biomedical Research | Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors |
| EP3870242A1 (en) | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells |
| EP3870709A1 (en) | 2018-10-25 | 2021-09-01 | Friedrich Miescher Institute for Biomedical Research | Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes |
| US20210355505A1 (en) | 2018-10-25 | 2021-11-18 | Friedrich Miescher Institute For Biomedical Research | Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells |
| WO2020152626A1 (en) | 2019-01-24 | 2020-07-30 | Friedrich Miescher Institute For Biomedical Research | Synp166 (proa36), a promoter for the specific expression of genes in photoreceptors |
| EP3914299A1 (en) | 2019-01-24 | 2021-12-01 | Friedrich Miescher Institute for Biomedical Research | Synp5 (proa9), a promoter for the specific expression of genes in retinal ganglion cells |
| US20220090062A1 (en) | 2019-01-24 | 2022-03-24 | Friedrich Miescher Institute For Biomedical Research | Synp66 (proa21), a promoter for the specific expression of genes in retinal ganglion cells |
| US20220119807A1 (en) | 2019-01-24 | 2022-04-21 | Friedrich Miescher Institute For Biomedical Research | Synp35 (proc8), a promoter for the specific expression of genes in retinal ganglion cells |
| SG11202108044YA (en) | 2019-02-25 | 2021-09-29 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
| WO2020174369A2 (en) | 2019-02-25 | 2020-09-03 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
-
2018
- 2018-11-28 KR KR1020207018116A patent/KR20200091432A/ko not_active Ceased
- 2018-11-28 CR CR20200236A patent/CR20200236A/es unknown
- 2018-11-28 US US16/768,057 patent/US11591615B2/en active Active
- 2018-11-28 EP EP18833621.8A patent/EP3717651A1/en not_active Withdrawn
- 2018-11-28 CA CA3083682A patent/CA3083682A1/en active Pending
- 2018-11-28 JP JP2020529489A patent/JP2021503934A/ja not_active Ceased
- 2018-11-28 CN CN201880084876.5A patent/CN111542611A/zh active Pending
- 2018-11-28 AU AU2018376259A patent/AU2018376259A1/en not_active Abandoned
- 2018-11-28 BR BR112020010928-2A patent/BR112020010928A2/pt not_active IP Right Cessation
- 2018-11-28 WO PCT/EP2018/082880 patent/WO2019106035A1/en not_active Ceased
- 2018-11-28 RU RU2020121408A patent/RU2020121408A/ru unknown
- 2018-11-28 MX MX2020005649A patent/MX2020005649A/es unknown
- 2018-11-28 SG SG11202005024QA patent/SG11202005024QA/en unknown
-
2020
- 2020-05-26 IL IL274957A patent/IL274957A/en unknown
- 2020-05-28 PH PH12020550748A patent/PH12020550748A1/en unknown
Also Published As
| Publication number | Publication date |
|---|---|
| US11591615B2 (en) | 2023-02-28 |
| WO2019106035A1 (en) | 2019-06-06 |
| KR20200091432A (ko) | 2020-07-30 |
| AU2018376259A1 (en) | 2020-06-11 |
| EP3717651A1 (en) | 2020-10-07 |
| SG11202005024QA (en) | 2020-06-29 |
| IL274957A (en) | 2020-07-30 |
| CN111542611A (zh) | 2020-08-14 |
| US20210010024A1 (en) | 2021-01-14 |
| RU2020121408A (ru) | 2021-12-30 |
| JP2021503934A (ja) | 2021-02-15 |
| PH12020550748A1 (en) | 2021-04-26 |
| RU2020121408A3 (es) | 2022-02-04 |
| CA3083682A1 (en) | 2019-06-06 |
| BR112020010928A2 (pt) | 2020-11-24 |
| MX2020005649A (es) | 2020-10-12 |
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