CN112912111A - 用于视网膜神经节细胞中特异表达基因的启动子SynP17(ProB1) - Google Patents

用于视网膜神经节细胞中特异表达基因的启动子SynP17(ProB1) Download PDF

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CN112912111A
CN112912111A CN201980069409.XA CN201980069409A CN112912111A CN 112912111 A CN112912111 A CN 112912111A CN 201980069409 A CN201980069409 A CN 201980069409A CN 112912111 A CN112912111 A CN 112912111A
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nucleic acid
gene
cell
acid molecule
isolated nucleic
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J·于特纳
J·科洛尔
B·罗斯卡
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Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research
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Novartis Forschungsstiftung Zweigniederlassung Friedrich Miescher Institute for Biomedical Research
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    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
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    • 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
    • C12N15/86Viral vectors
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    • C12N5/00Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/062Sensory transducers, e.g. photoreceptors; Sensory neurons, e.g. for hearing, taste, smell, pH, touch, temperature, pain
    • 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
    • A61K48/005Medicinal 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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    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic 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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  • Engineering & Computer Science (AREA)
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  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CN201980069409.XA 2018-10-25 2019-10-24 用于视网膜神经节细胞中特异表达基因的启动子SynP17(ProB1) Pending CN112912111A (zh)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP18202519.7 2018-10-25
EP18202519 2018-10-25
PCT/IB2019/059088 WO2020084537A1 (en) 2018-10-25 2019-10-24 Synp17 (prob1), a promoter for the specific expression of genes in retinal ganglion cells

Publications (1)

Publication Number Publication Date
CN112912111A true CN112912111A (zh) 2021-06-04

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CN201980069409.XA Pending CN112912111A (zh) 2018-10-25 2019-10-24 用于视网膜神经节细胞中特异表达基因的启动子SynP17(ProB1)

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US (1) US20210395750A1 (https=)
EP (1) EP3870238A1 (https=)
JP (1) JP2022505516A (https=)
CN (1) CN112912111A (https=)
WO (1) WO2020084537A1 (https=)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120290554A (zh) * 2024-09-10 2025-07-11 广州市未来基因递送研究院 感光细胞特异性双向启动子及其用途

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MX2010011467A (es) 2008-04-18 2011-02-15 Novartis Forschungsstiftung Novedosas herramientas y metodos terapeuticos para tratar la ceguera.
US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
US10857241B2 (en) 2015-09-15 2020-12-08 Friedrich Miescher Institute For Biomedical Research Therapeutical tools and methods for treating blindness by targeting photoreceptors
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
EP3383440B1 (en) 2015-12-03 2021-06-30 Friedrich Miescher Institute for Biomedical Research Synp162, a promoter for the specific expression of genes in rod photoreceptors
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
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
ES2909444T3 (es) 2015-12-03 2022-05-06 Friedrich Miescher Institute For Biomedical Res Synp159, 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
MX2020005033A (es) 2017-11-15 2020-10-28 Friedrich Miescher Institute For Biomedical Res Promotor especifico de las celulas del epitelio pigmentario retinal en primates.
SG11202005024QA (en) 2017-11-30 2020-06-29 Friedrich Miescher Institute For Biomedical Res Synpiii, a promoter for the specific expression of genes in retinal pigment epithelium

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108474001A (zh) * 2015-12-03 2018-08-31 弗里德里克·米谢尔生物医学研究所 SynP160,用于基因在视杆光感受器中特异性表达的启动子

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101342522B1 (ko) * 2011-02-22 2013-12-17 숙명여자대학교산학협력단 EphA7 유전자의 인핸서 인자 및 그것의 용도
EP3176177A1 (en) * 2015-12-03 2017-06-07 Friedrich Miescher Institute for Biomedical Research Synp157, a promoter for the specific expression of genes in rod photoreceptors

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN108474001A (zh) * 2015-12-03 2018-08-31 弗里德里克·米谢尔生物医学研究所 SynP160,用于基因在视杆光感受器中特异性表达的启动子

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
JOSEPHINE JÜTTNER等: "Targeting neuronal and glial cell types with synthetic promoter AAVs in mice, non-human primates, and humans", NATURE NEUROSCIENCE, pages 1 - 73 *
SANDRA SIEGERT等: "Transcriptional code and disease map for adult retinal cell types", NATURE NEUROSCIENCE, vol. 15, no. 3, pages 487 - 497 *

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN120290554A (zh) * 2024-09-10 2025-07-11 广州市未来基因递送研究院 感光细胞特异性双向启动子及其用途

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EP3870238A1 (en) 2021-09-01
JP2022505516A (ja) 2022-01-14
US20210395750A1 (en) 2021-12-23
WO2020084537A1 (en) 2020-04-30

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Application publication date: 20210604