JP2019500866A - 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 - Google Patents
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 Download PDFInfo
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Abstract
Description
CACACAAAACACAAATACAACAAATCTTTTAAAACTGCGATAATCCTATGTTGATTCTCTTACCTAAATTAAAAAACAAAACAAAATATGATAGCCTTGAATAAATTTTTATATGATATTTCATACTGTAGCCCAGGCTAGCCTCAAACTTGTAGCAATTCTCTTGTCTTCATCTCCTGAACACTGGAATTACAGGTGAGATCTAGAATGCTTTTCTTTTTTATGAGATACATCATCTTTAAATGAATTTAACTAACTTTATGTGTATGGATGTTTTGCCTGAATGTATGTGCAGGGCCGAGGGAGTGGGAGAGAACCCACTGGCTGTGTTAAGCCACTGAC(配列番号1)
CACACAAAACACAAATACAACAAATCTTTTAAAACTGCGATAATCCTATGTTGATTCTCTTACCTAAATTAAAAAACAAAACAAAATATGATAGCCTTGAATAAATTTTTATATGATATTTCATACTGTAGCCCAGGCTAGCCTCAAACTTGTAGCAATTCTCTTGTCTTCATCTCCTGAACACTGGAATTACAGGTGAGATCTAGAATGCTTTTCTTTTTTATGAGATACATCATCTTTAAATGAATTTAACTAACTTTATGTGTATGGATGTTTTGCCTGAATGTATGTGCAGGGCCGAGGGAGTGGGAGAGAACCCACTGGCTGTGTTAAGCCACTGAC(配列番号1)
全ゲノム高分解能DNAメチル化マップを、目的の細胞型(桿体)において作成して、候補制御領域を同定した。候補エンハンサーを、細胞型特異的DNA低メチル化の存在に基づいて選択した。そのように選択されたエレメントを、錐体および桿体において、インビボハイスループットレポーターアッセイを用いて発現についてスクリーニングした。その後、桿体特異的配列エレメントを合成し、最小プロモーター配列:ATCCTCACATGGTCCTGCTGGAGTTAGTAGAGGGTATATAATGGAAGCTCGACTTCCAGCTATCACATCCACTGTGTTGTTGTGAACTGGAATCCACTATAGGCCA(配列番号2)の前にクローニングした。ChR2−eGFPコード配列が、このプロモーターおよび最適化コザック配列(GCCACC)の直後に挿入され、その後に、ウッドチャック肝炎ウイルス転写後制御エレメント(WPRE)およびSV40ポリアデニル化部位が続いた。網膜ニューロンを、3.43E+11〜1.75E+12 GC/mLの範囲の力価を有するAAV血清型2/8を用いて、標的化した。
AAV投与について、麻酔下の動物の眼を、鋭い30ゲージ針によりレンズの近くの強膜で穿刺した。2マイクロリットルのAAV粒子懸濁液を、Hamiltonシリンジにより網膜下に注射した。3週間後、単離された網膜を、PBS中4%PFAにおいて30分間、固定し、続いて、PBS中、4℃で洗浄ステップを行った。網膜全体を、室温で1時間、PBS中10%正常ロバ血清(NDS)、1%BSA、0.5%Triton X−100で処理した。PBS中3%NDS、1%BSA、0.5%Triton X−100におけるモノクローナルのラット抗GFP抗体(Molecular Probes Inc.;1:500)およびポリクローナルのウサギ抗マウス錐体アレスチン抗体(Millipore:1:200)での処理を、室温で5日間、行った。二次ロバ抗ラットAlexa Fluor−488抗体(Molecular Probes Inc.;1:200)、抗ウサギAlexa Fluor−633およびHoechstでの処理を2時間、行った。切片を洗浄し、スライドグラス上にProLong Gold褐色防止用試薬(Molecular Probes Inc.)と共に載せ、Zeiss LSM 700 Axio Imager Z2レーザー走査型共焦点顕微鏡(Carl Zeiss Inc.)を用いて撮影した。
Claims (12)
- 配列番号1の核酸配列を含むかもしくはそれからなる、または配列番号1の前記配列と少なくとも80%同一性を有する少なくとも300bpの核酸配列からなる、単離された核酸分子であって、遺伝子をコードする核酸配列が、前記単離された核酸分子へ動作可能に連結されている場合、前記単離された核酸分子が前記遺伝子の桿体光受容器における特異的発現をもたらす、単離された核酸分子。
- 最小プロモーター、例えば、配列番号2の最小プロモーターをさらに含む、請求項1に記載の単離された核酸分子。
- 請求項1または2に記載の単離された核酸分子とストリンジェントな条件下でハイブリダイズする配列を含む単離された核酸分子。
- 特定の細胞における遺伝子発現を促進するエレメントとして請求項1または2に記載の単離された核酸を含む発現カセットであって、前記単離された核酸が、少なくとも、桿体光受容器において特異的に発現させる遺伝子をコードする核酸配列へ動作可能に連結されている、発現カセット。
- 請求項4に記載の発現カセットを含むベクター。
- ウイルスベクターである、請求項5に記載のベクター。
- 桿体光受容器における遺伝子の発現のための、請求項1もしくは2に記載の核酸の使用、または請求項4に記載の発現カセットの使用、または請求項5に記載のベクターの使用。
- 単離された細胞、細胞株、または細胞集団に、請求項4に記載の発現カセットをトランスフェクトするステップを含む、桿体光受容器において遺伝子を発現させる方法であって、前記細胞が桿体光受容器であるかまたは前記細胞が桿体光受容器を含む場合、発現させる前記遺伝子が、前記単離された細胞、細胞株、または細胞集団により特異的に発現される、方法。
- 請求項4に記載の発現カセットまたは請求項5に記載のベクターを含む、単離された細胞。
- 前記発現カセットまたはベクターが前記細胞のゲノムへ安定的に組み込まれている、請求項9に記載の細胞。
- 前記遺伝子の産物が光感受性分子、例えば、ハロロドプシンまたはチャネルロドプシンである、請求項1もしくは2に記載の単離された核酸分子、請求項4に記載の発現カセット、請求項5に記載のベクター、請求項7に記載の使用、請求項8に記載の方法、または請求項9に記載の細胞。
- 請求項1または2に記載の単離された核酸分子を含む、桿体光受容器において遺伝子を発現させるためのキット。
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EP15197902.8 | 2015-12-03 | ||
EP15197902 | 2015-12-03 | ||
PCT/IB2016/057268 WO2017093936A1 (en) | 2015-12-03 | 2016-12-01 | Synp162, a promoter for the specific expression of genes in rod photoreceptors |
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JP2019500866A true JP2019500866A (ja) | 2019-01-17 |
JP7075341B2 JP7075341B2 (ja) | 2022-05-25 |
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EP (1) | EP3383440B1 (ja) |
JP (1) | JP7075341B2 (ja) |
CN (1) | CN108472390B (ja) |
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WO2009127705A1 (en) | 2008-04-18 | 2009-10-22 | 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 |
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 |
WO2017064642A1 (en) | 2015-10-14 | 2017-04-20 | Fredrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
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 |
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 |
WO2017093935A1 (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 |
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 |
CN110072557B (zh) | 2016-11-02 | 2022-11-11 | 弗里德里克·米谢尔生物医学研究所 | 用于在方向选择性视网膜神经节细胞中特异性表达基因的启动子SynP198 |
WO2018146588A1 (en) | 2017-02-08 | 2018-08-16 | Friedrich Miescher Institute For Biomedical Research | Synp88, a promoter for the specific expression of genes in retinal ganglion cells |
CN111726985B (zh) | 2017-11-15 | 2022-06-10 | 弗里德里克·米谢尔生物医学研究所 | 灵长类动物视网膜色素上皮细胞特异性启动子 |
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 |
CN112930201A (zh) * | 2018-10-25 | 2021-06-08 | 弗里德里克·米谢尔生物医学研究所 | 用于视网膜神经节细胞中特异表达基因的启动子SynP78(ProA27) |
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EP1891976A1 (en) | 2006-08-23 | 2008-02-27 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research | Use of light sensitive genes |
EP2776459A1 (en) | 2011-11-08 | 2014-09-17 | Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute For Biomedical Research | Rod cell-specific promoter |
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WO2015121793A1 (en) | 2014-02-11 | 2015-08-20 | Friedrich Miescher Institute For Biomedical Research | Müller cell-specific promoter |
WO2015143418A2 (en) * | 2014-03-21 | 2015-09-24 | Genzyme Corporation | Gene therapy for retinitis pigmentosa |
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 |
WO2017064642A1 (en) | 2015-10-14 | 2017-04-20 | Fredrich Miescher Institute For Biomedical Research | Promoter for the specific expression of genes in retinal endothelial cells |
WO2017093935A1 (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 |
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 |
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 |
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 |
AU2016363830C1 (en) | 2015-12-04 | 2022-03-24 | Centre National De La Recherche Scientifique | Promoters and uses thereof |
WO2017199156A1 (en) | 2016-05-17 | 2017-11-23 | Friedrich Miescher Institute For Biomedical Research | Novel therapeutic tools and methods for treating blindness |
CN110072557B (zh) | 2016-11-02 | 2022-11-11 | 弗里德里克·米谢尔生物医学研究所 | 用于在方向选择性视网膜神经节细胞中特异性表达基因的启动子SynP198 |
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JP2020501538A (ja) | 2016-12-01 | 2020-01-23 | 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 | 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 |
WO2018146588A1 (en) | 2017-02-08 | 2018-08-16 | Friedrich Miescher Institute For Biomedical Research | Synp88, a promoter for the specific expression of genes in retinal ganglion cells |
CN111726985B (zh) | 2017-11-15 | 2022-06-10 | 弗里德里克·米谢尔生物医学研究所 | 灵长类动物视网膜色素上皮细胞特异性启动子 |
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US20210388387A1 (en) | 2018-10-25 | 2021-12-16 | Friedrich Miescher Institute For Biomedical Research | Synp151 (proc29), a promoter for the specific expression of genes in retinal ganglion cells |
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WO2020084537A1 (en) | 2018-10-25 | 2020-04-30 | Friedrich Miescher Institute For Biomedical Research | Synp17 (prob1), 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 |
CN112912508A (zh) | 2018-10-25 | 2021-06-04 | 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SynP194(ProB15) |
CN112930201A (zh) | 2018-10-25 | 2021-06-08 | 弗里德里克·米谢尔生物医学研究所 | 用于视网膜神经节细胞中特异表达基因的启动子SynP78(ProA27) |
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US20220119841A1 (en) | 2019-01-24 | 2022-04-21 | Friedrich Miescher Instit for Biomedical Research | Synp5 (proa9), a promoter for the specific expression of genes in retinal ganglion cells |
JP2022520875A (ja) | 2019-02-25 | 2022-04-01 | ノバルティス アーゲー | Biettiクリスタリン網膜症を治療するための組成物及び方法 |
US20220154210A1 (en) | 2019-02-25 | 2022-05-19 | Novartis Ag | Compositions and methods to treat bietti crystalline dystrophy |
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"MUS MUSCULUS BAC CLONE RP23-330N2 FROM CHROMOSOME 17, COMPLETE SEQUENCE" [09-NOV-2005] RETRIEVED FR, JPN6020043233, ISSN: 0004385239 * |
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