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

Composiciones y métodos para tratar distrofia cristalina de bietti

Info

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

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
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    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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    • C12Y114/00—Oxidoreductases acting on paired donors, with incorporation or reduction of molecular oxygen (1.14)
    • 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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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/14011—Parvoviridae
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
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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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  • Wood Science & Technology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • Virology (AREA)
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  • Epidemiology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

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

Family

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 (15)

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)
PH (1) PH12021552038A1 (es)
SG (1) SG11202108044YA (es)
TW (1) TW202045729A (es)
WO (1) WO2020174368A1 (es)

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EP3289090B1 (en) 2015-04-30 2019-06-26 Friedrich Miescher Institute for Biomedical Research Promoter for the specific expression of genes in müller cells
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US11059871B2 (en) 2015-12-03 2021-07-13 Friedrich Miescher Institute For Biomedical Research SYNP162, a promoter for the specific expression of genes in rod photoreceptors
JP7058220B2 (ja) 2015-12-03 2022-04-21 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 ＳｙｎＰ１５９、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
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EP3579884A1 (en) 2017-02-08 2019-12-18 Friedrich Miescher Institute for Biomedical Research Synp88, a promoter for the specific expression of genes in retinal ganglion cells
WO2019097454A1 (en) 2017-11-15 2019-05-23 Friedrich Miescher Institute For Biomedical Research Primate retinal pigment epithelium cell-specific promoter
KR20200091432A (ko) 2017-11-30 2020-07-30 프리드리히 미셔 인스티튜트 포 바이오메디칼 리서치 망막 색소 상피에서의 유전자의 특이적 발현을 위한 프로모터 SynPIII
WO2023284873A1 (en) * 2021-07-15 2023-01-19 Shanghai Vitalgen Biopharma Co., Ltd. Recombinant adeno-associated viral vectors for treating bietti crystalline dystrophy
CN118028318A (zh) * 2021-12-22 2024-05-14 苏州诺洁贝生物技术有限公司 优化的cyp4v2基因及其用途
WO2025030426A1 (en) * 2023-08-09 2025-02-13 Chigenovo Co., Ltd. Pharmaceutical preparations for treating bietti's crystalline dystrophy

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