IL274957A - SYNPIII, a promoter for specific expression of retinal pigment epithelium genes - Google Patents

SYNPIII, a promoter for specific expression of retinal pigment epithelium genes

Info

Publication number
IL274957A
IL274957A IL274957A IL27495720A IL274957A IL 274957 A IL274957 A IL 274957A IL 274957 A IL274957 A IL 274957A IL 27495720 A IL27495720 A IL 27495720A IL 274957 A IL274957 A IL 274957A
Authority
IL
Israel
Prior art keywords
synpiii
promoter
genes
specific expression
retinal pigment
Prior art date
Application number
IL274957A
Other languages
English (en)
Hebrew (he)
Original Assignee
Friedrich Miescher Institute For Biomedical Res
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Friedrich Miescher Institute For Biomedical Res filed Critical Friedrich Miescher Institute For Biomedical Res
Publication of IL274957A publication Critical patent/IL274957A/en

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    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/86Viral vectors
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Virology (AREA)
  • Epidemiology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
IL274957A 2017-11-30 2020-05-26 SYNPIII, a promoter for specific expression of retinal pigment epithelium genes IL274957A (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
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 (1)

Publication Number Publication Date
IL274957A true IL274957A (en) 2020-07-30

Family

ID=60543416

Family Applications (1)

Application Number Title Priority Date Filing Date
IL274957A IL274957A (en) 2017-11-30 2020-05-26 SYNPIII, a promoter for specific expression of retinal pigment epithelium genes

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)

* Cited by examiner, † Cited by third party
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
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
CN121013909A (zh) * 2023-04-27 2025-11-25 韩国生命工学研究院 视网膜细胞中的基因表达的启动子及包含其的载体系统

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* Cited by examiner, † Cited by third party
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
CA2346262A1 (en) * 1998-09-17 2000-03-23 University Of Florida Methods for treatment of degenerative retinal diseases
EP3437473A1 (en) 2006-05-04 2019-02-06 Wayne State University Restoration of visual responses by in vivo delivery of rhodopsin nucleic acids
EP1891976A1 (en) 2006-08-23 2008-02-27 Novartis Forschungsstiftung, Zweigniederlassung Friedrich Miescher Institute for Biomedical Research Use of light sensitive genes
WO2009112448A1 (en) 2008-03-11 2009-09-17 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
AU2012204266C1 (en) 2011-01-07 2017-12-21 Applied Genetic Technologies Corporation Promoters, expression cassettes, vectors, kits, and methods for the treatment of achromatopsia and other diseases
WO2014033095A1 (en) * 2012-08-27 2014-03-06 Friedrich Miescher Institute For Biomedical Research Retinal off circuit-specific promoter
WO2015118507A1 (en) 2014-02-10 2015-08-13 Friedrich Miescher Institute For Biomedical Research Aii retinal amacrine cell-specific promoter
ES2748282T3 (es) 2014-02-11 2020-03-16 Friedrich Miescher Institute For Biomedical Res Promotor específico de células de Müller
GB201502137D0 (en) * 2015-02-09 2015-03-25 Ucl Business Plc Treatment
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
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
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
JP6966442B2 (ja) * 2015-12-04 2021-11-17 ソルボンヌ・ユニヴェルシテSorbonne Universite プロモーター及びその使用
WO2017199156A1 (en) 2016-05-17 2017-11-23 Friedrich Miescher Institute For Biomedical Research Novel therapeutic tools and methods for treating blindness
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
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.

Also Published As

Publication number Publication date
KR20200091432A (ko) 2020-07-30
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
JP2021503934A (ja) 2021-02-15
AU2018376259A1 (en) 2020-06-11
US20210010024A1 (en) 2021-01-14
MX2020005649A (es) 2020-10-12

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