AR092317A1 - Vectores virales para el tratamiento de distrofia retiniana - Google Patents

Vectores virales para el tratamiento de distrofia retiniana

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Publication number
AR092317A1
AR092317A1 ARP130101507A ARP130101507A AR092317A1 AR 092317 A1 AR092317 A1 AR 092317A1 AR P130101507 A ARP130101507 A AR P130101507A AR P130101507 A ARP130101507 A AR P130101507A AR 092317 A1 AR092317 A1 AR 092317A1
Authority
AR
Argentina
Prior art keywords
viral vectors
treatment
retina
retinian
dystrophy
Prior art date
Application number
ARP130101507A
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English (en)
Inventor
Peter Dryja Thaddeus
Reddy Police Seshidhar
Choi Vivian
Eric Bigelow Chad
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 AR092317A1 publication Critical patent/AR092317A1/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
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    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
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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
    • 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
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    • 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
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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    • C12N15/09—Recombinant DNA-technology
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    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
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    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
    • C12N2750/14341—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14343—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14311—Parvovirus, e.g. minute virus of mice
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    • C12N2750/14345—Special targeting system for viral vectors
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
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    • C12N2750/14371—Demonstrated in vivo effect
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/60—Vectors comprising as targeting moiety peptide derived from defined protein from viruses
    • C12N2810/6027—Vectors comprising as targeting moiety peptide derived from defined protein from viruses ssDNA viruses
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    • C12N2810/00—Vectors comprising a targeting moiety
    • C12N2810/50—Vectors comprising as targeting moiety peptide derived from defined protein
    • C12N2810/80—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates
    • C12N2810/85—Vectors comprising as targeting moiety peptide derived from defined protein from vertebrates mammalian

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Abstract

La presente se relaciona con vectores virales capaces de entregar un gen heterólogo en la retina y en particular el de entregar genes RLBP1 en células RPE y células de Müller de la retina. La presente también se relaciona con ácidos nucleicos útiles en la producción de vectores virales, composiciones que comprenden los vectores virales y usos de las composiciones y vectores virales. La presente también se relaciona con métodos para entregar y/o expresar un gen heterólogo en la retina, mejorar la tasa de adaptación a la oscuridad en un sujeto y el tratamiento de la distrofia retiniana asociada con el RLBP1.
ARP130101507A 2012-05-04 2013-05-03 Vectores virales para el tratamiento de distrofia retiniana AR092317A1 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201261642630P 2012-05-04 2012-05-04
US201361776167P 2013-03-11 2013-03-11

Publications (1)

Publication Number Publication Date
AR092317A1 true AR092317A1 (es) 2015-04-15

Family

ID=48670626

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Application Number Title Priority Date Filing Date
ARP130101507A AR092317A1 (es) 2012-05-04 2013-05-03 Vectores virales para el tratamiento de distrofia retiniana

Country Status (10)

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US (5) US9163259B2 (es)
EP (2) EP3326655A1 (es)
JP (4) JP6290185B2 (es)
CN (2) CN108753824B (es)
AR (1) AR092317A1 (es)
CA (2) CA2872447C (es)
ES (1) ES2947159T3 (es)
TW (1) TW201350581A (es)
UY (1) UY34780A (es)
WO (1) WO2013164793A2 (es)

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CA2872447A1 (en) 2013-11-07
JP7100232B2 (ja) 2022-07-13
JP2020054358A (ja) 2020-04-09
US10550404B2 (en) 2020-02-04
TW201350581A (zh) 2013-12-16
US20210047656A1 (en) 2021-02-18
EP3326655A1 (en) 2018-05-30
JP2022088372A (ja) 2022-06-14
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US20180080046A1 (en) 2018-03-22
EP2844302B1 (en) 2023-03-15
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CA3182080A1 (en) 2013-11-07
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CN108753824A (zh) 2018-11-06
CN104470545A (zh) 2015-03-25
WO2013164793A3 (en) 2014-01-16
UY34780A (es) 2013-11-29
JP2018108083A (ja) 2018-07-12
US9163259B2 (en) 2015-10-20
CN104470545B (zh) 2018-07-06
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