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AR123838A1 - Vector mejorado de virus adenoasociado (aav) y usos de este - Google Patents

Vector mejorado de virus adenoasociado (aav) y usos de este

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Publication number
AR123838A1
AR123838A1 ARP210102870A ARP210102870A AR123838A1 AR 123838 A1 AR123838 A1 AR 123838A1 AR P210102870 A ARP210102870 A AR P210102870A AR P210102870 A ARP210102870 A AR P210102870A AR 123838 A1 AR123838 A1 AR 123838A1
Authority
AR
Argentina
Prior art keywords
nucleic acid
aavhu68
aav
host cell
capsid
Prior art date
Application number
ARP210102870A
Other languages
English (en)
Inventor
Qiang Wang
Original Assignee
Univ Pennsylvania
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 Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of AR123838A1 publication Critical patent/AR123838A1/es

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    • C12N5/00—Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
    • C12N5/10—Cells modified by introduction of foreign genetic material
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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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
    • C12N2750/14142—Use of virus, viral particle or viral elements as a vector virus or viral particle as vehicle, e.g. encapsulating small organic molecule
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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
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14151—Methods of production or purification of viral material
    • C12N2750/14152—Methods of production or purification of viral material relating to complementing cells and packaging systems for producing virus or viral particles

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  • Health & Medical Sciences (AREA)
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  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
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  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Cell Biology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicinal Chemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)

Abstract

Se indican en el presente documento secuencia de ácidos nucleicos modificadas que codifican AAVhu68 que proporciona rendimientos mejorados de partículas virales AAVhu68 empaquetadas. También se indica una célula hospedadora de empaquetamiento que comprende la secuencia de nucleótidos modificada y métodos de uso de esta para producir vectores de AAVhu68 recombinantes, útiles para el suministro de genes. También se indican líquidos sobrenadantes celulares de producción que comprenden rendimientos mejorados de vectores de AAV recombinante con cápsides de AAVhu68 producidas utilizando las secuencias de nucleótidos que se indican en el presente documento. Reivindicación 1: Una célula hospedadora de producción para generar un virus adenoasociado recombinante (rAAV) que tiene una cápside de AAVhu68 caracterizada porque comprende: (a) una secuencia de ácidos nucleicos de la SEQ ID nº 1 o una secuencia de ácidos nucleicos al menos 99% idéntica a esta, que codifica la proteína de la cápside de AAVhu68 vp1 ligada operativamente a secuencias de control de la expresión que dirigen la expresión de la proteína de la cápside de AAVhu68 vp1 en la célula hospedadora; (b) una molécula de ácido nucleico para su empaquetamiento en la cápside de AAVhu68; dicha molécula de ácido nucleico comprende al menos una repetición terminal invertida (ITR) de AAV y una secuencia de ácidos nucleicos que no corresponde a un AAV y que codifica un producto génico ligado operativamente a secuencias que dirigen la expresión del producto en una célula hospedadora, y (c) suficientes funciones de AAV rep y funciones auxiliares para permitir el empaquetamiento de la molécula de ácido nucleico en la cápside de AAVhu68. Reivindicación 12: Una suspensión que comprende células hospedadoras de producción según cualquiera de las reivindicaciones 1 a 11. Reivindicación 15: Un cultivo celular que comprende la célula hospedadora de producción según cualquiera de las reivindicaciones 1 a 11. Reivindicación 17: Un sustrato caracterizado porque comprende una línea celular adherida que comprende la célula hospedadora de producción según cualquiera de las reivindicaciones 1 a 11. Reivindicación 18: Un cultivo celular caracterizado porque comprende la célula hospedadora de producción según cualquiera de las reivindicaciones 1 a 11. Reivindicación 20: Una composición que comprende una población combinada de virus adenoasociados recombinantes hu68 (rAAVhu68) producidos mediante la suspensión, el cultivo celular o el sustrato según una cualquiera de las reivindicaciones 12 a 19.
ARP210102870A 2020-10-18 2021-10-18 Vector mejorado de virus adenoasociado (aav) y usos de este AR123838A1 (es)

Applications Claiming Priority (1)

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US202063093275P 2020-10-18 2020-10-18

Publications (1)

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AR123838A1 true AR123838A1 (es) 2023-01-18

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ARP210102870A AR123838A1 (es) 2020-10-18 2021-10-18 Vector mejorado de virus adenoasociado (aav) y usos de este

Country Status (8)

Country Link
US (1) US20230383313A1 (es)
EP (1) EP4229186A1 (es)
AR (1) AR123838A1 (es)
AU (1) AU2021359874A1 (es)
CA (1) CA3195553A1 (es)
IL (1) IL302127A (es)
TW (1) TW202229315A (es)
WO (1) WO2022082109A1 (es)

Families Citing this family (5)

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TW202338086A (zh) 2022-01-10 2023-10-01 賓州大學委員會 有用於治療異染性白質失養症之組成物
AR128239A1 (es) 2022-01-10 2024-04-10 Univ Pennsylvania Composiciones y métodos útiles para el tratamiento de trastornos mediados por c9orf72
WO2024258961A1 (en) 2023-06-12 2024-12-19 The Trustees Of The University Of Pennsylvania Aav gene therapy for mucopolysaccharidosis iiib
AR133098A1 (es) 2023-06-29 2025-08-27 Univ Pennsylvania Aav mutante con motivos dirigidos al sistema nervioso central y composiciones que lo contienen
WO2025106661A1 (en) 2023-11-14 2025-05-22 The Trustees Of The University Of Pennsylvania Compositions with cardiac and skeletal musclespecific targeting motifs and uses thereof

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Also Published As

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TW202229315A (zh) 2022-08-01
CA3195553A1 (en) 2022-04-21
IL302127A (en) 2023-06-01
AU2021359874A1 (en) 2023-05-25
US20230383313A1 (en) 2023-11-30
EP4229186A1 (en) 2023-08-23
AU2021359874A9 (en) 2024-02-08
WO2022082109A1 (en) 2022-04-21

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