JP2021503933A5 - - Google Patents
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- JP2021503933A5 JP2021503933A5 JP2020529473A JP2020529473A JP2021503933A5 JP 2021503933 A5 JP2021503933 A5 JP 2021503933A5 JP 2020529473 A JP2020529473 A JP 2020529473A JP 2020529473 A JP2020529473 A JP 2020529473A JP 2021503933 A5 JP2021503933 A5 JP 2021503933A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- isolated nucleic
- acid molecule
- acid sequence
- pharmaceutical composition
- 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.)
- Granted
Links
- 150000007523 nucleic acids Chemical group 0.000 claims description 75
- 108020004707 nucleic acids Proteins 0.000 claims description 46
- 102000039446 nucleic acids Human genes 0.000 claims description 46
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 29
- 108090000623 proteins and genes Proteins 0.000 claims description 17
- 241000288906 Primates Species 0.000 claims description 13
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims description 12
- 239000013598 vector Substances 0.000 claims description 12
- 201000010099 disease Diseases 0.000 claims description 5
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims description 5
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- 206010064930 age-related macular degeneration Diseases 0.000 claims description 3
- 208000002780 macular degeneration Diseases 0.000 claims description 3
- 206010012689 Diabetic retinopathy Diseases 0.000 claims description 2
- 206010020880 Hypertrophy Diseases 0.000 claims description 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 2
- 239000000790 retinal pigment Substances 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims 14
- 108010035848 Channelrhodopsins Proteins 0.000 claims 2
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 108010050754 Halorhodopsins Proteins 0.000 claims 2
- 238000000034 method Methods 0.000 description 6
- 239000003068 molecular probe Substances 0.000 description 3
- 210000001525 retina Anatomy 0.000 description 3
- 241000283074 Equus asinus Species 0.000 description 2
- 229920004890 Triton X-100 Polymers 0.000 description 2
- 239000013504 Triton X-100 Substances 0.000 description 2
- YQEZLKZALYSWHR-UHFFFAOYSA-N Ketamine Chemical compound C=1C=CC=C(Cl)C=1C1(NC)CCCCC1=O YQEZLKZALYSWHR-UHFFFAOYSA-N 0.000 description 1
- 241000282560 Macaca mulatta Species 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 210000000981 epithelium Anatomy 0.000 description 1
- 238000005562 fading Methods 0.000 description 1
- 239000000835 fiber Substances 0.000 description 1
- 239000011521 glass Substances 0.000 description 1
- PCHJSUWPFVWCPO-UHFFFAOYSA-N gold Chemical compound [Au] PCHJSUWPFVWCPO-UHFFFAOYSA-N 0.000 description 1
- 239000010931 gold Substances 0.000 description 1
- 229910052737 gold Inorganic materials 0.000 description 1
- 238000005286 illumination Methods 0.000 description 1
- 229960003299 ketamine Drugs 0.000 description 1
- 210000003928 nasal cavity Anatomy 0.000 description 1
- 229960002511 phenobarbital sodium Drugs 0.000 description 1
- WRLGYAWRGXKSKG-UHFFFAOYSA-M phenobarbital sodium Chemical compound [Na+].C=1C=CC=CC=1C1(CC)C(=O)NC([O-])=NC1=O WRLGYAWRGXKSKG-UHFFFAOYSA-M 0.000 description 1
- 239000000049 pigment Substances 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 230000002123 temporal effect Effects 0.000 description 1
- 210000001519 tissue Anatomy 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- 238000005406 washing Methods 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP17204746.6 | 2017-11-30 | ||
| EP17204746 | 2017-11-30 | ||
| PCT/EP2018/082872 WO2019106027A1 (en) | 2017-11-30 | 2018-11-28 | SynP61, A PRIMATE RETINAL PIGMENT EPITHELIUM CELL-SPECIFIC PROMOTER |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021503933A JP2021503933A (ja) | 2021-02-15 |
| JP2021503933A5 true JP2021503933A5 (enExample) | 2022-01-06 |
| JP7390290B2 JP7390290B2 (ja) | 2023-12-01 |
Family
ID=60543414
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020529473A Active JP7390290B2 (ja) | 2017-11-30 | 2018-11-28 | 霊長類網膜色素上皮細胞特異的プロモーターＳｙｎＰ６１ |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US20230193314A1 (enExample) |
| EP (1) | EP3717509A1 (enExample) |
| JP (1) | JP7390290B2 (enExample) |
| KR (1) | KR20200091435A (enExample) |
| CN (1) | CN111527102A (enExample) |
| AU (1) | AU2018376544B2 (enExample) |
| BR (1) | BR112020010775A2 (enExample) |
| CA (1) | CA3083404A1 (enExample) |
| CR (1) | CR20200235A (enExample) |
| IL (1) | IL274885A (enExample) |
| MX (1) | MX2020005648A (enExample) |
| PH (1) | PH12020550766A1 (enExample) |
| RU (1) | RU2020121390A (enExample) |
| SG (1) | SG11202005002QA (enExample) |
| WO (1) | WO2019106027A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PT2271369E (pt) | 2008-04-18 | 2014-07-10 | Novartis Forschungsstiftung | Novas ferramentas terapêuticas e métodos para tratar a cegueira |
| ES2747433T3 (es) | 2015-04-30 | 2020-03-10 | Friedrich Miescher Institute For Biomedical Res | Promotor para la expresión específica de genes en células de Müller |
| WO2017046084A1 (en) | 2015-09-15 | 2017-03-23 | Friedrich Miescher Institute For Biomedical Research | Novel therapeutical tools and methods for treating blindness by targeting photoreceptors |
| 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 |
| EP3383439B1 (en) | 2015-12-03 | 2019-09-25 | Friedrich Miescher Institute for Biomedical Research | Synp161, a promoter for the specific expression of genes in rod photoreceptors |
| ES2900486T3 (es) | 2015-12-03 | 2022-03-17 | Friedrich Miescher Institute For Biomedical Res | SynP160, un promotor para la expresión específica de genes en los fotorreceptores de los bastones |
| CN108430519B (zh) | 2015-12-03 | 2022-04-15 | 弗里德里克·米谢尔生物医学研究所 | SynP159，用于基因在视杆光感受器中特异性表达的启动子 |
| JP7075341B2 (ja) | 2015-12-03 | 2022-05-25 | 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 | ＳｙｎＰ１６２、桿体光受容器における遺伝子の特異的発現のためのプロモーター |
| KR20190077471A (ko) | 2016-11-02 | 2019-07-03 | 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 | 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198 |
| 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 |
| BR112020010928A2 (pt) | 2017-11-30 | 2020-11-24 | Friedrich Miescher Institute For Biomedical Research | synpiii, um promotor para a expressão de gene específica em epitélio de pigmento retinal |
| WO2020174369A2 (en) * | 2019-02-25 | 2020-09-03 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US7309487B2 (en) * | 2004-02-09 | 2007-12-18 | George Inana | Methods and compositions for detecting and treating retinal diseases |
| EP3105334B1 (en) * | 2014-02-11 | 2019-07-03 | Friedrich Miescher Institute for Biomedical Research | Müller cell-specific promoter |
| AU2018369975B2 (en) * | 2017-11-15 | 2022-05-19 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
-
2018
- 2018-11-28 WO PCT/EP2018/082872 patent/WO2019106027A1/en not_active Ceased
- 2018-11-28 KR KR1020207018368A patent/KR20200091435A/ko not_active Ceased
- 2018-11-28 AU AU2018376544A patent/AU2018376544B2/en not_active Ceased
- 2018-11-28 CN CN201880084798.9A patent/CN111527102A/zh active Pending
- 2018-11-28 MX MX2020005648A patent/MX2020005648A/es unknown
- 2018-11-28 JP JP2020529473A patent/JP7390290B2/ja active Active
- 2018-11-28 CA CA3083404A patent/CA3083404A1/en active Pending
- 2018-11-28 RU RU2020121390A patent/RU2020121390A/ru unknown
- 2018-11-28 SG SG11202005002QA patent/SG11202005002QA/en unknown
- 2018-11-28 CR CR20200235A patent/CR20200235A/es unknown
- 2018-11-28 US US16/768,055 patent/US20230193314A1/en not_active Abandoned
- 2018-11-28 EP EP18833620.0A patent/EP3717509A1/en active Pending
- 2018-11-28 BR BR112020010775-1A patent/BR112020010775A2/pt not_active IP Right Cessation
-
2020
- 2020-05-24 IL IL274885A patent/IL274885A/en unknown
- 2020-05-28 PH PH12020550766A patent/PH12020550766A1/en unknown
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