JP2020501538A5 - - Google Patents
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- JP2020501538A5 JP2020501538A5 JP2019529228A JP2019529228A JP2020501538A5 JP 2020501538 A5 JP2020501538 A5 JP 2020501538A5 JP 2019529228 A JP2019529228 A JP 2019529228A JP 2019529228 A JP2019529228 A JP 2019529228A JP 2020501538 A5 JP2020501538 A5 JP 2020501538A5
- Authority
- JP
- Japan
- Prior art keywords
- nucleic acid
- expression cassette
- acid sequence
- vector
- seq
- 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.)
- Pending
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- 150000007523 nucleic acids Chemical class 0.000 claims description 32
- 210000004027 cell Anatomy 0.000 claims description 27
- 230000014509 gene expression Effects 0.000 claims description 25
- 108020004707 nucleic acids Proteins 0.000 claims description 18
- 102000039446 nucleic acids Human genes 0.000 claims description 18
- 108091028043 Nucleic acid sequence Proteins 0.000 claims description 14
- 239000013598 vector Substances 0.000 claims description 14
- 108090000623 proteins and genes Proteins 0.000 claims description 13
- 238000000034 method Methods 0.000 claims description 8
- 210000001153 interneuron Anatomy 0.000 claims description 7
- 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 3
- 108010035848 Channelrhodopsins Proteins 0.000 claims description 3
- 108010050754 Halorhodopsins Proteins 0.000 claims description 3
- 239000013603 viral vector Substances 0.000 claims description 2
- 241000702421 Dependoparvovirus Species 0.000 claims 1
- 108700026220 vif Genes Proteins 0.000 claims 1
- 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
- 238000002347 injection Methods 0.000 description 2
- 239000007924 injection Substances 0.000 description 2
- 229960003793 midazolam Drugs 0.000 description 2
- 239000012109 Alexa Fluor 568 Substances 0.000 description 1
- 206010002091 Anaesthesia Diseases 0.000 description 1
- XQFRJNBWHJMXHO-RRKCRQDMSA-N IDUR Chemical compound C1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 XQFRJNBWHJMXHO-RRKCRQDMSA-N 0.000 description 1
- 241000699670 Mus sp. Species 0.000 description 1
- 241000283973 Oryctolagus cuniculus Species 0.000 description 1
- CZMRCDWAGMRECN-UGDNZRGBSA-N Sucrose Chemical compound O[C@H]1[C@H](O)[C@@H](CO)O[C@@]1(CO)O[C@@H]1[C@H](O)[C@@H](O)[C@H](O)[C@@H](CO)O1 CZMRCDWAGMRECN-UGDNZRGBSA-N 0.000 description 1
- 229930006000 Sucrose Natural products 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 230000037005 anaesthesia Effects 0.000 description 1
- HSWPZIDYAHLZDD-UHFFFAOYSA-N atipamezole Chemical compound C1C2=CC=CC=C2CC1(CC)C1=CN=CN1 HSWPZIDYAHLZDD-UHFFFAOYSA-N 0.000 description 1
- 229960003002 atipamezole Drugs 0.000 description 1
- 239000005388 borosilicate glass Substances 0.000 description 1
- 210000004556 brain Anatomy 0.000 description 1
- 210000005252 bulbus oculi Anatomy 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- 230000018044 dehydration Effects 0.000 description 1
- 238000006297 dehydration reaction Methods 0.000 description 1
- 238000005516 engineering process Methods 0.000 description 1
- PJMPHNIQZUBGLI-UHFFFAOYSA-N fentanyl Chemical compound C=1C=CC=CC=1N(C(=O)CC)C(CC1)CCN1CCC1=CC=CC=C1 PJMPHNIQZUBGLI-UHFFFAOYSA-N 0.000 description 1
- 229960002428 fentanyl Drugs 0.000 description 1
- 229960004381 flumazenil Drugs 0.000 description 1
- OFBIFZUFASYYRE-UHFFFAOYSA-N flumazenil Chemical compound C1N(C)C(=O)C2=CC(F)=CC=C2N2C=NC(C(=O)OCC)=C21 OFBIFZUFASYYRE-UHFFFAOYSA-N 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
- 229960004716 idoxuridine Drugs 0.000 description 1
- HRLIOXLXPOHXTA-UHFFFAOYSA-N medetomidine Chemical compound C=1C=CC(C)=C(C)C=1C(C)C1=CN=C[N]1 HRLIOXLXPOHXTA-UHFFFAOYSA-N 0.000 description 1
- 229960002140 medetomidine Drugs 0.000 description 1
- DDLIGBOFAVUZHB-UHFFFAOYSA-N midazolam Chemical compound C12=CC(Cl)=CC=C2N2C(C)=NC=C2CN=C1C1=CC=CC=C1F DDLIGBOFAVUZHB-UHFFFAOYSA-N 0.000 description 1
- 239000000203 mixture Substances 0.000 description 1
- 229960004127 naloxone Drugs 0.000 description 1
- UZHSEJADLWPNLE-GRGSLBFTSA-N naloxone Chemical compound O=C([C@@H]1O2)CC[C@@]3(O)[C@H]4CC5=CC=C(O)C2=C5[C@@]13CCN4CC=C UZHSEJADLWPNLE-GRGSLBFTSA-N 0.000 description 1
- 210000002569 neuron Anatomy 0.000 description 1
- 238000002360 preparation method Methods 0.000 description 1
- 210000000880 retinal rod photoreceptor cell Anatomy 0.000 description 1
- 210000002966 serum Anatomy 0.000 description 1
- 239000005720 sucrose Substances 0.000 description 1
- 238000001890 transfection Methods 0.000 description 1
- 230000000007 visual effect Effects 0.000 description 1
- 238000005406 washing Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2022002979A JP2022050590A (ja) | 2016-12-01 | 2022-01-12 | インターニューロン中の遺伝子の特異的発現のためのプロモーターｓｙｎｐｉ |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP16201749.5 | 2016-12-01 | ||
| EP16201749 | 2016-12-01 | ||
| PCT/EP2017/080829 WO2018099975A1 (en) | 2016-12-01 | 2017-11-29 | Synpi, a promoter for the specific expression of genes in interneurons |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002979A Division JP2022050590A (ja) | 2016-12-01 | 2022-01-12 | インターニューロン中の遺伝子の特異的発現のためのプロモーターｓｙｎｐｉ |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2020501538A JP2020501538A (ja) | 2020-01-23 |
| JP2020501538A5 true JP2020501538A5 (enExample) | 2021-01-14 |
Family
ID=57460429
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2019529228A Pending JP2020501538A (ja) | 2016-12-01 | 2017-11-29 | インターニューロン中の遺伝子の特異的発現のためのプロモーターｓｙｎｐｉ |
| JP2022002979A Withdrawn JP2022050590A (ja) | 2016-12-01 | 2022-01-12 | インターニューロン中の遺伝子の特異的発現のためのプロモーターｓｙｎｐｉ |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022002979A Withdrawn JP2022050590A (ja) | 2016-12-01 | 2022-01-12 | インターニューロン中の遺伝子の特異的発現のためのプロモーターｓｙｎｐｉ |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20190376083A1 (enExample) |
| EP (1) | EP3548094A1 (enExample) |
| JP (2) | JP2020501538A (enExample) |
| CN (1) | CN110072558A (enExample) |
| WO (1) | WO2018099975A1 (enExample) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2679246A3 (en) | 2008-04-18 | 2014-02-26 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Novel therapeutical tools and methods for treating blindness |
| WO2016174624A1 (en) | 2015-04-30 | 2016-11-03 | Friedrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in müller cells |
| 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 |
| ES2881782T3 (es) | 2015-10-14 | 2021-11-30 | Friedrich Miescher Institute For Biomedical Res | Promotor para la expresión específica de genes en células endoteliales retinianas. |
| US10898586B2 (en) | 2015-12-03 | 2021-01-26 | Friedrich Miescher Institute For Biomedical Research | SynP161, a promoter for the specific expression of genes in rod photoreceptors |
| JP7058220B2 (ja) | 2015-12-03 | 2022-04-21 | 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 | ＳｙｎＰ１５９、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 |
| US10994026B2 (en) | 2015-12-03 | 2021-05-04 | Friedrich Miescher Institute For Biomedical Research | SynP160, a promoter for the specific expression of genes in rod photoreceptors |
| US11059871B2 (en) | 2015-12-03 | 2021-07-13 | Friedrich Miescher Institute For Biomedical Research | SYNP162, a promoter for the specific expression of genes in rod photoreceptors |
| WO2018083607A1 (en) | 2016-11-02 | 2018-05-11 | Friedrich Miescher Institute For Biomedical Research | Synp198, a promoter for the specific expression of genes in direction selective retinal ganglion cells |
| JP7112414B2 (ja) | 2017-02-08 | 2022-08-03 | 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ｓｙｎｐ８８ |
| WO2019097454A1 (en) | 2017-11-15 | 2019-05-23 | Friedrich Miescher Institute For Biomedical Research | Primate retinal pigment epithelium cell-specific promoter |
| US11591615B2 (en) | 2017-11-30 | 2023-02-28 | Friedrich Miescher Institute For Biomedical Research | SynPIII, a promoter for the specific expression of genes in retinal pigment epithelium |
| CN116529381A (zh) * | 2020-12-03 | 2023-08-01 | 弗里德里克·米谢尔生物医学研究所 | 利用孤儿基序和CpG密度的组合来控制异源转基因的表达 |
Family Cites Families (5)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2357832T3 (es) * | 2002-10-31 | 2011-05-03 | Janssen Pharmaceutica N.V. | Genes sensibles a crh en el cns. |
| EP2679246A3 (en) * | 2008-04-18 | 2014-02-26 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Novel therapeutical tools and methods for treating blindness |
| US20110098346A1 (en) * | 2008-05-19 | 2011-04-28 | Agency For Science, Technology And Research | Nucleic acid molecule and method of targeting gene expression to gliomas |
| WO2013068413A1 (en) * | 2011-11-08 | 2013-05-16 | Novartis Forschungsstiftung, Zweigniederlassung, Friedrich Miescher Institute For Biomedical Research | Rod cell-specific promoter |
| US20130195763A1 (en) * | 2012-01-27 | 2013-08-01 | Charles F. Landry | Methods and compositions for improving sleep and memory |
-
2017
- 2017-11-29 JP JP2019529228A patent/JP2020501538A/ja active Pending
- 2017-11-29 EP EP17816564.3A patent/EP3548094A1/en not_active Withdrawn
- 2017-11-29 WO PCT/EP2017/080829 patent/WO2018099975A1/en not_active Ceased
- 2017-11-29 CN CN201780072399.6A patent/CN110072558A/zh not_active Withdrawn
- 2017-11-29 US US16/464,478 patent/US20190376083A1/en not_active Abandoned
-
2022
- 2022-01-12 JP JP2022002979A patent/JP2022050590A/ja not_active Withdrawn
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