JP2021503934A5 - - Google Patents
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- JP2021503934A5 JP2021503934A5 JP2020529489A JP2020529489A JP2021503934A5 JP 2021503934 A5 JP2021503934 A5 JP 2021503934A5 JP 2020529489 A JP2020529489 A JP 2020529489A JP 2020529489 A JP2020529489 A JP 2020529489A JP 2021503934 A5 JP2021503934 A5 JP 2021503934A5
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- 150000007523 nucleic acids Chemical class 0.000 claims description 86
- 108020004707 nucleic acids Proteins 0.000 claims description 44
- 102000039446 nucleic acids Human genes 0.000 claims description 44
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 42
- 108090000623 proteins and genes Proteins 0.000 claims description 29
- 230000014509 gene expression Effects 0.000 claims description 27
- 238000000034 method Methods 0.000 claims description 19
- 239000013598 vector Substances 0.000 claims description 16
- 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 6
- 108010035848 Channelrhodopsins Proteins 0.000 claims description 6
- 108010050754 Halorhodopsins Proteins 0.000 claims description 6
- 239000013603 viral vector Substances 0.000 claims description 3
- 210000003583 retinal pigment epithelium Anatomy 0.000 claims 15
- 102100021921 ATP synthase subunit a Human genes 0.000 claims 5
- 102100040958 Aconitate hydratase, mitochondrial Human genes 0.000 claims 5
- 102400000068 Angiostatin Human genes 0.000 claims 5
- 108010079709 Angiostatins Proteins 0.000 claims 5
- 102100021569 Apoptosis regulator Bcl-2 Human genes 0.000 claims 5
- 108091012583 BCL2 Proteins 0.000 claims 5
- 102100026596 Bcl-2-like protein 1 Human genes 0.000 claims 5
- 101150008012 Bcl2l1 gene Proteins 0.000 claims 5
- 102000004219 Brain-derived neurotrophic factor Human genes 0.000 claims 5
- 108090000715 Brain-derived neurotrophic factor Proteins 0.000 claims 5
- 108010005939 Ciliary Neurotrophic Factor Proteins 0.000 claims 5
- 102100031614 Ciliary neurotrophic factor Human genes 0.000 claims 5
- 102100031162 Collagen alpha-1(XVIII) chain Human genes 0.000 claims 5
- 102100025287 Cytochrome b Human genes 0.000 claims 5
- 102100028203 Cytochrome c oxidase subunit 3 Human genes 0.000 claims 5
- 108010079505 Endostatins Proteins 0.000 claims 5
- 108010092408 Eosinophil Peroxidase Proteins 0.000 claims 5
- 102100028471 Eosinophil peroxidase Human genes 0.000 claims 5
- 108090000379 Fibroblast growth factor 2 Proteins 0.000 claims 5
- 102000003974 Fibroblast growth factor 2 Human genes 0.000 claims 5
- 102000024452 GDNF Human genes 0.000 claims 5
- 108091010837 Glial cell line-derived neurotrophic factor Proteins 0.000 claims 5
- 101000753741 Homo sapiens ATP synthase subunit a Proteins 0.000 claims 5
- 101000965314 Homo sapiens Aconitate hydratase, mitochondrial Proteins 0.000 claims 5
- 101000858267 Homo sapiens Cytochrome b Proteins 0.000 claims 5
- 101000861034 Homo sapiens Cytochrome c oxidase subunit 3 Proteins 0.000 claims 5
- 101000722054 Homo sapiens Dynamin-like 120 kDa protein, mitochondrial Proteins 0.000 claims 5
- 101001076418 Homo sapiens Interleukin-1 receptor type 1 Proteins 0.000 claims 5
- 101000614988 Homo sapiens Mediator of RNA polymerase II transcription subunit 12 Proteins 0.000 claims 5
- 101000604411 Homo sapiens NADH-ubiquinone oxidoreductase chain 1 Proteins 0.000 claims 5
- 101001109052 Homo sapiens NADH-ubiquinone oxidoreductase chain 4 Proteins 0.000 claims 5
- 101001109060 Homo sapiens NADH-ubiquinone oxidoreductase chain 4L Proteins 0.000 claims 5
- 101000598279 Homo sapiens NADH-ubiquinone oxidoreductase chain 5 Proteins 0.000 claims 5
- 101000632623 Homo sapiens NADH-ubiquinone oxidoreductase chain 6 Proteins 0.000 claims 5
- 101000722063 Homo sapiens Optic atrophy 3 protein Proteins 0.000 claims 5
- 101000894525 Homo sapiens Transforming growth factor-beta-induced protein ig-h3 Proteins 0.000 claims 5
- 101000640713 Homo sapiens Transmembrane protein 126A Proteins 0.000 claims 5
- 102000026659 IL10 Human genes 0.000 claims 5
- 102100026016 Interleukin-1 receptor type 1 Human genes 0.000 claims 5
- 108090000174 Interleukin-10 Proteins 0.000 claims 5
- 102100021070 Mediator of RNA polymerase II transcription subunit 12 Human genes 0.000 claims 5
- 101000597433 Mus musculus Nucleoredoxin-like protein 1 Proteins 0.000 claims 5
- 102100038625 NADH-ubiquinone oxidoreductase chain 1 Human genes 0.000 claims 5
- 102100021506 NADH-ubiquinone oxidoreductase chain 4 Human genes 0.000 claims 5
- 102100021452 NADH-ubiquinone oxidoreductase chain 4L Human genes 0.000 claims 5
- 102100036971 NADH-ubiquinone oxidoreductase chain 5 Human genes 0.000 claims 5
- 102100028386 NADH-ubiquinone oxidoreductase chain 6 Human genes 0.000 claims 5
- 102100025325 Optic atrophy 3 protein Human genes 0.000 claims 5
- 102100021398 Transforming growth factor-beta-induced protein ig-h3 Human genes 0.000 claims 5
- 102100033846 Transmembrane protein 126A Human genes 0.000 claims 5
- FZCSTZYAHCUGEM-UHFFFAOYSA-N aspergillomarasmine B Natural products OC(=O)CNC(C(O)=O)CNC(C(O)=O)CC(O)=O FZCSTZYAHCUGEM-UHFFFAOYSA-N 0.000 claims 5
- 108700000711 bcl-X Proteins 0.000 claims 5
- 208000009606 optic atrophy 7 Diseases 0.000 claims 5
- 230000004083 survival effect Effects 0.000 claims 5
- 241000288906 Primates Species 0.000 claims 3
- 241000702421 Dependoparvovirus Species 0.000 claims 2
- 210000004027 cell Anatomy 0.000 description 19
- 210000000844 retinal pigment epithelial cell Anatomy 0.000 description 7
- 239000003068 molecular probe Substances 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
- 210000001525 retina Anatomy 0.000 description 2
- 101100298998 Caenorhabditis elegans pbs-3 gene Proteins 0.000 description 1
- 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
- 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
- 238000002360 preparation method Methods 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 230000002123 temporal effect Effects 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 |
|---|---|---|---|
| EP17204750.8 | 2017-11-30 | ||
| 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 (2)
| Publication Number | Publication Date |
|---|---|
| JP2021503934A JP2021503934A (ja) | 2021-02-15 |
| JP2021503934A5 true JP2021503934A5 (enExample) | 2022-01-06 |
Family
ID=60543416
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020529489A Ceased JP2021503934A (ja) | 2017-11-30 | 2018-11-28 | 網膜色素上皮中の遺伝子の特異的発現のためのプロモーターＳｙｎＰＩＩＩ |
Country Status (15)
| Country | Link |
|---|---|
| US (1) | US11591615B2 (enExample) |
| EP (1) | EP3717651A1 (enExample) |
| JP (1) | JP2021503934A (enExample) |
| KR (1) | KR20200091432A (enExample) |
| CN (1) | CN111542611A (enExample) |
| AU (1) | AU2018376259A1 (enExample) |
| BR (1) | BR112020010928A2 (enExample) |
| CA (1) | CA3083682A1 (enExample) |
| CR (1) | CR20200236A (enExample) |
| IL (1) | IL274957A (enExample) |
| MX (1) | MX2020005649A (enExample) |
| PH (1) | PH12020550748A1 (enExample) |
| RU (1) | RU2020121408A (enExample) |
| SG (1) | SG11202005024QA (enExample) |
| WO (1) | WO2019106035A1 (enExample) |
Families Citing this family (14)
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| WO2024225830A1 (ko) * | 2023-04-27 | 2024-10-31 | 한국생명공학연구원 | 유전자의 망막 세포 내 발현을 위한 프로모터 및 이를 포함하는 벡터 시스템 |
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 |
| AU775245B2 (en) | 1998-09-17 | 2004-07-22 | Catholic University Nijmegen | Methods for treatment of degenerative retinal diseases |
| EP2019588A4 (en) | 2006-05-04 | 2010-11-24 | Univ Wayne State | RESTORATION OF VISUAL RESPONSES BY IN VIVO ADMINISTRATION OF NUCLEIC ACIDS OF RHODOPSIN |
| EP1891976A1 (en) | 2006-08-23 | 2008-02-27 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research | Use of light sensitive genes |
| EP2254992A1 (en) | 2008-03-11 | 2010-12-01 | Novartis Forschungsstiftung, Zweigniederlassung | Novel tool for the analysis of neural circuits |
| PT2271369E (pt) | 2008-04-18 | 2014-07-10 | Novartis Forschungsstiftung | Novas ferramentas terapêuticas e métodos para tratar a cegueira |
| EP2661494B1 (en) | 2011-01-07 | 2019-06-12 | Applied Genetic Technologies Corporation | Promoters, expression cassettes and vectors for use treating achromatopsia and other diseases |
| EP2888368A1 (en) * | 2012-08-27 | 2015-07-01 | Friedrich Miescher Institute for Biomedical Research | Retinal off circuit-specific promoter |
| US9999685B2 (en) | 2014-02-10 | 2018-06-19 | Friedrich Miescher Institute For Biomedical Research | AII retinal amacrine cell-specific promoter |
| EP3105334B1 (en) | 2014-02-11 | 2019-07-03 | Friedrich Miescher Institute for Biomedical Research | Müller cell-specific promoter |
| GB201502137D0 (en) * | 2015-02-09 | 2015-03-25 | Ucl Business Plc | Treatment |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| 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 |
| CN110072558A (zh) | 2016-12-01 | 2019-07-30 | 弗里德里克·米谢尔生物医学研究所 | 用于使基因在中间神经元中特异性表达的启动子SynPI |
| 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 |
| WO2019106027A1 (en) | 2017-11-30 | 2019-06-06 | Friedrich Miescher Institute For Biomedical Research | SynP61, A 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 |
| 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） |
| 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 |
| WO2020084538A1 (en) | 2018-10-25 | 2020-04-30 | Friedrich Miescher Institute For Biomedical Research | Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes |
| WO2020084540A1 (en) | 2018-10-25 | 2020-04-30 | Friedrich Miescher Institute For Biomedical Research | Synp78 (proa27), a promoter for the specific expression of genes in retinal ganglion cells |
| 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 |
| 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） |
| 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 |
| EP3914300A1 (en) | 2019-01-24 | 2021-12-01 | 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 |
| US20220154210A1 (en) | 2019-02-25 | 2022-05-19 | 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 BR BR112020010928-2A patent/BR112020010928A2/pt not_active IP Right Cessation
- 2018-11-28 MX MX2020005649A patent/MX2020005649A/es unknown
- 2018-11-28 CA CA3083682A patent/CA3083682A1/en active Pending
- 2018-11-28 WO PCT/EP2018/082880 patent/WO2019106035A1/en not_active Ceased
- 2018-11-28 AU AU2018376259A patent/AU2018376259A1/en not_active Abandoned
- 2018-11-28 EP EP18833621.8A patent/EP3717651A1/en not_active Withdrawn
- 2018-11-28 KR KR1020207018116A patent/KR20200091432A/ko not_active Ceased
- 2018-11-28 CN CN201880084876.5A patent/CN111542611A/zh active Pending
- 2018-11-28 RU RU2020121408A patent/RU2020121408A/ru unknown
- 2018-11-28 SG SG11202005024QA patent/SG11202005024QA/en unknown
- 2018-11-28 JP JP2020529489A patent/JP2021503934A/ja not_active Ceased
- 2018-11-28 CR CR20200236A patent/CR20200236A/es unknown
- 2018-11-28 US US16/768,057 patent/US11591615B2/en active Active
-
2020
- 2020-05-26 IL IL274957A patent/IL274957A/en unknown
- 2020-05-28 PH PH12020550748A patent/PH12020550748A1/en unknown
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