MX2021010149A - Composiciones y metodos para tratar la distrofia cristalina de bietti. - Google Patents

Composiciones y metodos para tratar la distrofia cristalina de bietti.

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Publication number
MX2021010149A
MX2021010149A MX2021010149A MX2021010149A MX2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A MX 2021010149 A MX2021010149 A MX 2021010149A
Authority
MX
Mexico
Prior art keywords
treat
compositions
methods
crystalline dystrophy
bietti crystalline
Prior art date
Application number
MX2021010149A
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English (en)
Inventor
Botond Roska
Josephine Juettner
Jacek Krol
Christie L Bell
Terri Mcgee
Original Assignee
Novartis Ag
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 filed Critical Novartis Ag
Publication of MX2021010149A publication Critical patent/MX2021010149A/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
    • A61P27/02—Ophthalmic agents
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    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0071—Oxidoreductases (1.) acting on paired donors with incorporation of molecular oxygen (1.14)
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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • 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)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • 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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    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14171—Demonstrated in vivo effect
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/42—Vector systems having a special element relevant for transcription being an intron or intervening sequence for splicing and/or stability of RNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/48—Vector systems having a special element relevant for transcription regulating transport or export of RNA, e.g. RRE, PRE, WPRE, CTE
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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

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.
MX2021010149A 2019-02-25 2020-02-24 Composiciones y metodos para tratar la distrofia cristalina de bietti. MX2021010149A (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
MX2021010149A true MX2021010149A (es) 2021-09-14

Family

ID=69780247

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2021010149A MX2021010149A (es) 2019-02-25 2020-02-24 Composiciones y metodos para tratar la 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)

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JP7042741B2 (ja) 2015-12-03 2022-03-28 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 ＳｙｎＰ１６１、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
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
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EP3931334A1 (en) 2022-01-05
CR20210444A (es) 2021-11-02
BR112021016501A2 (pt) 2021-10-26
CA3130731A1 (en) 2020-09-03
SG11202108044YA (en) 2021-09-29
KR20210132684A (ko) 2021-11-04
US20220154210A1 (en) 2022-05-19
CN113677801A (zh) 2021-11-19
JP2022521025A (ja) 2022-04-04
TW202045729A (zh) 2020-12-16
WO2020174368A1 (en) 2020-09-03
IL285447A (en) 2021-09-30
AU2020229085A1 (en) 2021-08-19

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