DK2986635T3 - Effektiv afgivelse af store gener ved hjælp af dual-aav-vektorer - Google Patents

Effektiv afgivelse af store gener ved hjælp af dual-aav-vektorer Download PDF

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DK2986635T3
DK2986635T3 DK14721802.8T DK14721802T DK2986635T3 DK 2986635 T3 DK2986635 T3 DK 2986635T3 DK 14721802 T DK14721802 T DK 14721802T DK 2986635 T3 DK2986635 T3 DK 2986635T3
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aav
dual
vectors
sequence
plasmid
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Alberto Auricchio
Pasqualina Colella
Ivana Trapani
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Fond Telethon
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/20—Vectors comprising a special translation-regulating system translation of more than one cistron
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
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    • C12N2840/00—Vectors comprising a special translation-regulating system
    • C12N2840/44—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor
    • C12N2840/445—Vectors comprising a special translation-regulating system being a specific part of the splice mechanism, e.g. donor, acceptor for trans-splicing, e.g. polypyrimidine tract, branch point splicing

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Claims (14)

  1. EFFEKTIV AFGIVELSE AF STORE GENER VED HJÆLP AF DUAL-AAV-VEKTORER
    1. Dualkonstruktionssystem til ekspression af den kodende sekvens for et gen af interesse i en værtscelle, idet den kodende sekvens består af en 5'-endedel og af en 3'-endedel, hvilket dualkonstruktionssystem omfatter: a) et første plasmid, der i en 5'-3'-retning omfatter: - en AAV 5'-inverteret terminal gentagelses-(5'-ITR)-sekvens; - en promotorsekvens; - 5'-endedelen af den kodende sekvens, idet 5'-endedelen er operabelt forbundet med og under styring af promotoren; - en nukleinsyresekvens for et splejsningsdonorsignal; og - en AAV 3'-inverteret terminal gentagelses-(3'-ITR)-sekvens; og b) et andet plasmid, der i en 5'-3'-retning omfatter: - en AAV 5'-inverteret terminal gentagelses-(5'-ITR)-sekvens; - en nukleinsyresekvens for et splejsningsacceptorsignal; - 3'-enden af den kodende sekvens; - en polyadenyleringssignalnukleinsyresekvens; og - en AAV 3'-inverteret terminal gentagelses-(3'-ITR)-sekvens, hvor det første plasmid endvidere omfatter en nukleinsyresekvens for en rekombinogen region i 5'-positionen i AAV 3'-ITR'en i det første plasmid og i 3'-positionen i nukleinsyresekvensen for splejsningsdonorsignalet, og hvor det andet plasmid endvidere omfatternukleinsyresekvensen forden rekombinogene region i 3'-positionen i AAV5'-ITR'en i det andet plasmid og i 5'-positionen i nukleinsyresekvensen forsplejsningsacceptorsignalet, hvor den rekombinogene region er en rekombinogen region i en F1-fag, der består af sekvensen: GGGATTTTGCCGATTTCGGCCTATTGGTTAAAAAATGAGCTGATTTAACAAAAATTTAA CGC GAATTTTAACAAAAT (SEQ ID No. 3) eller et fragment deraf, som bibeholder den rekombinogene egenskab for SEQ ID No. 3, hvor den kodende sekvens efter indføring af det første plasmid og det andet plasmid i værtscellen rekonstituerer ved hjælp af splejsningsdonor- og splejsningsacceptorsignalerne.
  2. 2. Dualkonstruktionssystem ifølge krav 1, hvor nukleotidsekvensen for ITR'erne stammer fra den samme AAV-serotype eller fra forskellige AAV-serotyper, fortrinsvis hvor 3'-ITR'en i det første plasmid og 5'-ITR'en i det andet plasmid er fra den samme AAV-serotype, fortrinsvis hvor henholdsvis 5'-ITR'en og 3'-ITR'en i det første plasmid og 5'-ITR'en og 3'-ITR'en i det andet plasmid er fra forskellige AAV-serotyper, fortrinsvis hvor 5'-ITR'en i det første plasmid og 3'-ITR'en i det andet plasmid er fra forskellige AAV-serotyper.
  3. 3. Dualkonstruktionssystem ifølge et hvilket som helst af de foregående krav, hvor nukleinsyresekvensen for splejsningsdonorsignalet består af sekvensen: GTAAGTATCAAGGTTACAAGACAGGTTTAAGGAGACCAATAGAAACTGGGCTTGTCGA GACA GAGAAGACTCTTGCGTTTCT (SEQ ID No. 1), og/eller hvor nukleinsyresekvensen for splejsningsacceptorsignalet består af sekvensen GATAGGCACCTATTGGTCTTACTGACATCCACTTTGCCTTTCTCTCCACAG (SEQ ID No. 2).
  4. 4. Dualkonstruktionssystem ifølge et hvilket som helst af de foregående krav, hvor den kodende sekvens er en nukleotidsekvens, der koder for et protein, som kan korrigere en nedarvet retinal degeneration.
  5. 5. Dualkonstruktionssystem ifølge krav 4, hvor den kodende sekvens er valgt fra gruppen bestående af: ABCA4, MY07A, CEP290, CDH23, EYS, USH2a, GPR98 eller ALMS1.
  6. 6. Viralt dual-adeno-associeret virus-(AAV)-vektorsystem, der omfatter: a) en første viral AAV-vektor, der indeholder det første plasmid som defineret i et hvilket som helst af kravene 1 til 5, og b) en anden viral AAV-vektor, der indeholder det andet plasmid som defineret i et hvilket som helst af kravene 1 til 5.
  7. 7. Viralt dual-AAV-vektorsystem ifølge krav 6, hvor adeno-associeret virus-(AAV)-vektorerne er valgt fra den samme eller forskellige AAV-serotyper, fortrinsvis hvor det adeno-associerede virus er valgt fra serotypen 2, serotypen 8, serotypen 5, serotypen 7 eller serotypen 9.
  8. 8. Værtscelle, der omfatter det virale dualvektorsystem ifølge et hvilket som helst af kravene 6 til 7.
  9. 9. Dualkonstruktionssystem ifølge et hvilket som helst af kravene 1 til 5, viralt dualvektorsystem ifølge et hvilket som helst af kravene 6 til 7 eller værtscelle ifølge krav 8 til medicinsk anvendelse, fortrinsvis til anvendelse ved genterapi.
  10. 10. Dualkonstruktionssystem, viralt dual vektorsystem, værtscelle ifølge krav 9 til anvendelse ved behandling og/eller forebyggelse af en patologi eller sygdom, der er kendetegnet ved en retinal degeneration, fortrinsvis hvor den retinale degeneration er nedarvet, fortrinsvis hvor patologien eller sygdommen er valgt fra gruppen bestående af: retinitis pigmentosa, Lebers kongenitale amaurose (LCA), Stargardt-sygdom, Usher-sygdom, Alstrom-syndrom, en sygdom forårsaget af en mutation i ABCA4-ger\et.
  11. 11. Farmaceutisk sammensætning, der omfatter dualkonstruktionssystemet ifølge et hvilket som helst af kravene 1 til 5, det virale dualvektorsystem ifølge et hvilket som helst af kravene 6 til 7 eller værtscellen ifølge krav 8 og et farmaceutisk acceptabelt vehikel.
  12. 12. Nukleinsyre bestående af SEQ ID No. 3 eller et fragment deraf, som bibeholder den rekombinogene egenskab for SEQ ID No. 3, til anvendelse som en rekombinogen region.
  13. 13. In v/'fro-fremgangsmåde til induktion af genetisk rekombination, hvilken fremgangsmåde omfatter anvendelse af sekvensen bestående af SEQ ID No. 3 eller et fragment deraf, som bibeholder den rekombinogene egenskab for SEQ ID No. 3.
  14. 14. Sekvens bestående af SEQ ID No. 3 eller et fragment deraf, som bibeholder den rekombinogene egenskab for SEQ ID No. 3, til anvendelse ved en behandlingsfremgangsmåde, fortrinsvis ved hjælp af genterapi, hvor fremgangsmåden inducerer genetisk rekombination.
DK14721802.8T 2013-04-18 2014-04-18 Effektiv afgivelse af store gener ved hjælp af dual-aav-vektorer DK2986635T3 (da)

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