KR20200091432A - 망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII - Google Patents

망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII Download PDF

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KR20200091432A
KR20200091432A KR1020207018116A KR20207018116A KR20200091432A KR 20200091432 A KR20200091432 A KR 20200091432A KR 1020207018116 A KR1020207018116 A KR 1020207018116A KR 20207018116 A KR20207018116 A KR 20207018116A KR 20200091432 A KR20200091432 A KR 20200091432A
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nucleic acid
gene
sequence
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cells
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요제피네 위트너
야체크 크롤
보톤트 로스카
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프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
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    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005Medicinal 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
    • A61K48/0058Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075Medicinal 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
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    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/008Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination

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KR1020207018116A 2017-11-30 2018-11-28 망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII Ceased KR20200091432A (ko)

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Application Number Priority Date Filing Date Title
EP17204750 2017-11-30
EP17204750.8 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

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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)
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Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024225830A1 (ko) * 2023-04-27 2024-10-31 한국생명공학연구원 유전자의 망막 세포 내 발현을 위한 프로모터 및 이를 포함하는 벡터 시스템

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PT2271369E (pt) 2008-04-18 2014-07-10 Novartis Forschungsstiftung Novas ferramentas terapêuticas e métodos para tratar a cegueira
US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
ES2926677T3 (es) 2015-09-15 2022-10-27 Friedrich Miescher Institute For Biomedical Res Herramientas y métodos terapéuticos novedosos para tratar la ceguera mediante el direccionamiento a los fotorreceptores
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
WO2017093931A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp159, a promoter for the specific expression of genes in rod photoreceptors
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
ES2886664T3 (es) 2015-12-03 2021-12-20 Friedrich Miescher Institute For Biomedical Res SynP162, un promotor para la expresión específica de genes en fotorreceptores de bastón
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
RU2766353C2 (ru) 2016-11-02 2022-03-15 Фридрих Мишер Инститьют Фор Байомедикал Рисерч Synp198, промотор для специфической экспрессии генов в ганглионарных клетках сетчатки, избирательных в отношении направления
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
CA3082080A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
CN111542611A (zh) 2017-11-30 2020-08-14 弗里德里克·米谢尔生物医学研究所 用于使基因在视网膜色素上皮中特异性表达的启动子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

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ES2926677T3 (es) 2015-09-15 2022-10-27 Friedrich Miescher Institute For Biomedical Res Herramientas y métodos terapéuticos novedosos para tratar la ceguera mediante el direccionamiento a los fotorreceptores
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
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
WO2017093931A1 (en) 2015-12-03 2017-06-08 Friedrich Miescher Institute For Biomedical Research Synp159, a promoter for the specific expression of genes in rod photoreceptors
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EP3548094A1 (en) 2016-12-01 2019-10-09 Friedrich Miescher Institute for Biomedical Research Synpi, a promoter for the specific expression of genes in interneurons
CN110072559A (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SynP107
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
CA3082080A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
JP7390290B2 (ja) 2017-11-30 2023-12-01 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 霊長類網膜色素上皮細胞特異的プロモーターSynP61
CN111542611A (zh) 2017-11-30 2020-08-14 弗里德里克·米谢尔生物医学研究所 用于使基因在视网膜色素上皮中特异性表达的启动子SynPIII
JP2022505517A (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SynP151(ProC29)
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)
JP2022512784A (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SynP78(ProA27)
US20210388385A1 (en) 2018-10-25 2021-12-16 Friedrich Miescher Institute For Biomedical Research Synp27 (prob12), a promoter for the specific expression of genes in protoplasmic astrocytes
WO2020084542A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp194 (prob15), a promoter for the specific expression of genes in retinal ganglion cells
WO2020084539A1 (en) 2018-10-25 2020-04-30 Friedrich Miescher Institute For Biomedical Research Synp57 (proa14), a promoter for the specific expression of genes in photoreceptors
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
EP3914721A1 (en) 2019-01-24 2021-12-01 Friedrich Miescher Institute for Biomedical Research Synp35 (proc8), a promoter for the specific expression of genes in retinal ganglion cells
US20220154211A1 (en) 2019-02-25 2022-05-19 Novartis Ag Compositions and methods to treat bietti crystalline dystrophy
MX2021010149A (es) 2019-02-25 2021-09-14 Novartis Ag Composiciones y metodos para tratar la distrofia cristalina de bietti.

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024225830A1 (ko) * 2023-04-27 2024-10-31 한국생명공학연구원 유전자의 망막 세포 내 발현을 위한 프로모터 및 이를 포함하는 벡터 시스템

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US11591615B2 (en) 2023-02-28
PH12020550748A1 (en) 2021-04-26
SG11202005024QA (en) 2020-06-29
CA3083682A1 (en) 2019-06-06
BR112020010928A2 (pt) 2020-11-24
EP3717651A1 (en) 2020-10-07
WO2019106035A1 (en) 2019-06-06
CN111542611A (zh) 2020-08-14
RU2020121408A (ru) 2021-12-30
CR20200236A (es) 2020-10-19
RU2020121408A3 (enExample) 2022-02-04
IL274957A (en) 2020-07-30
JP2021503934A (ja) 2021-02-15
AU2018376259A1 (en) 2020-06-11
US20210010024A1 (en) 2021-01-14
MX2020005649A (es) 2020-10-12

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