CR20210444A - Composiciones y métodos para tratar distrofia cristalina de bietti - Google Patents

Composiciones y métodos para tratar distrofia cristalina de bietti

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Publication number
CR20210444A
CR20210444A CR20210444A CR20210444A CR20210444A CR 20210444 A CR20210444 A CR 20210444A CR 20210444 A CR20210444 A CR 20210444A CR 20210444 A CR20210444 A CR 20210444A CR 20210444 A CR20210444 A CR 20210444A
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CR
Costa Rica
Prior art keywords
treat
compositions
methods
crystalline dystrophy
bietti crystalline
Prior art date
Application number
CR20210444A
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English (en)
Inventor
Botond Roska
Josephine Juettner
Jacek Krol
Terri Mcgee
Christie L Bell
Original Assignee
Novartis Ag
Friedrich Miescher Institute For Biomedical Res
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Publication date
Application filed by Novartis Ag, Friedrich Miescher Institute For Biomedical Res filed Critical Novartis Ag
Publication of CR20210444A publication Critical patent/CR20210444A/es

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    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/005—Medicinal 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0075—Medicinal 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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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • C12Y114/14—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14) with reduced flavin or flavoprotein as one donor, and incorporation of one atom of oxygen (1.14.14)
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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Abstract

En la presente se proveen vectores virales para insertar un gen del CYP4V2 heterólogo en la retina, por ej., las células del EPR de la retina para tratar sujetos con distrofia cristalina de Bietti.
CR20210444A 2019-02-25 2020-02-24 Composiciones y métodos para tratar distrofia cristalina de bietti CR20210444A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962810250P 2019-02-25 2019-02-25
PCT/IB2020/051557 WO2020174368A1 (en) 2019-02-25 2020-02-24 Compositions and methods to treat bietti crystalline dystrophy

Publications (1)

Publication Number Publication Date
CR20210444A true CR20210444A (es) 2021-11-02

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ID=69780247

Family Applications (1)

Application Number Title Priority Date Filing Date
CR20210444A CR20210444A (es) 2019-02-25 2020-02-24 Composiciones y métodos para tratar distrofia cristalina de bietti

Country Status (14)

Country Link
US (1) US20220154210A1 (es)
EP (1) EP3931334A1 (es)
JP (1) JP2022521025A (es)
KR (1) KR20210132684A (es)
CN (1) CN113677801A (es)
AU (1) AU2020229085A1 (es)
BR (1) BR112021016501A2 (es)
CA (1) CA3130731A1 (es)
CR (1) CR20210444A (es)
IL (1) IL285447A (es)
MX (1) MX2021010149A (es)
SG (1) SG11202108044YA (es)
TW (1) TW202045729A (es)
WO (1) WO2020174368A1 (es)

Families Citing this family (12)

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US10941417B2 (en) 2015-04-30 2021-03-09 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in Müller cells
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US11254934B2 (en) 2015-10-14 2022-02-22 Friedrich Miescher Institute For Biomedical Research Promoter for the specific expression of genes in retinal endothelial cells
CN108472390B (zh) 2015-12-03 2022-04-15 弗里德里克·米谢尔生物医学研究所 SynP162,用于基因在视杆光感受器中特异性表达的启动子
CN108472389B (zh) 2015-12-03 2022-04-08 弗里德里克·米谢尔生物医学研究所 SynP161，用于基因在视杆光感受器中特异性表达的启动子
US10995344B2 (en) 2015-12-03 2021-05-04 Friedrich Miescher Institute For Biomedical Research SYNP159, a promoter for the specific expression of genes in rod photoreceptors
KR20190077471A (ko) 2016-11-02 2019-07-03 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 방향 선택적인 망막 신경절 세포에서의 유전자의 특이적 발현을 위한 프로모터, synp198
CN110392582A (zh) 2017-02-08 2019-10-29 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SynP88
CR20200210A (es) 2017-11-15 2020-09-23 Friedrich Miescher Institute For Biomedical Res Promotor especìfico de las cèlulas del epitelio pigmentario retinal en primates
CR20200236A (es) 2017-11-30 2020-10-19 Friedrich Miescher Institute For Biomedical Res Synpiii, un promotor para la expresión específica de genes en el epitelio pigmentario retiniano
EP4370663A1 (en) * 2021-07-15 2024-05-22 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating bietti crystalline dystrophy
CN114381465B (zh) * 2021-12-22 2024-01-16 苏州诺洁贝生物技术有限公司 优化的cyp4v2基因及其用途

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TW202045729A (zh) 2020-12-16
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