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DK2191001T3 - Raav-vektorsammensætninger med tyrosinmodificerede capsidproteiner og fremgangsmåder til anvendelse deraf - Google Patents

Raav-vektorsammensætninger med tyrosinmodificerede capsidproteiner og fremgangsmåder til anvendelse deraf Download PDF

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DK2191001T3
DK2191001T3 DK08733161.7T DK08733161T DK2191001T3 DK 2191001 T3 DK2191001 T3 DK 2191001T3 DK 08733161 T DK08733161 T DK 08733161T DK 2191001 T3 DK2191001 T3 DK 2191001T3
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disease
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cells
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Li Zhong
Sergei Zolotukhin
Lakshmanan Govindasamy
Mavis Agbandje-Mckenna
Arun Srivastava
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Univ Florida
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Claims (7)

1. Rekombinant serotype 2-adenoassocieret virus (rAAV2)-vektor, der omfatter mindst én muteret overfladeeksponeret tyrosinrest på dens capsidprotein, hvilken mindst ene muterede tyrosinrest er udvalgt fra gruppen, der består af Tyr252, Tyr272, Tyr444, Tyr500, Tyr700, Tyr704, Tyr730, Tyr275, Tyr281, Tyr508, Tyr576, Tyr612, Tyr673 eller Tyr720. 2. rAAV-vektor ifølge krav 1, hvor den mindst ene mutered tyrosinrest er en eller flere af Tyr252, Tyr272, Tyr444, Tyr500, Tyr700, Tyr704 eller Tyr730. 3. rAAV-vektor ifølge krav 1 eller krav 2, der yderligere omfatter mindst et første nukleinsyresegment, der koder for mindst ét terapeutisk middel. 4. rAAV-vektor ifølge et hvilket som helst af ovennævnte krav, hvor den mindst ene muterede overfladeeksponerede tyrosinrest er substitueret med phenylalanin. 5. rAAV-vektor ifølge et hvilket som helst af ovennævnte krav, der yderligere omfatter en enhancersekvens, hvor enhanceren fortrinsvis er udvalgt fra gruppen, der består af en CMV-enhancer, en syntetisk enhancer, en leverspecifik enhancer, en vaskulær specifik enhancer, en hjernespecifik enhancer, en nervecellespecifik enhancer, en lungespecifik enhancer, en muskelspecifik enhancer, en nyrespecifik enhancer, en pankreasspecifik enhancer og en øcellespecifik enhancer. 6. rAAV-vektor ifølge et hvilket som helst af kravene 3 til 5, hvor ekspressionen af det terapeutiske middel kontrolleres af en promotor, hvor promotoren er en heterolog, vævsspecifik, konstitutiv eller inducerbar promotor og fortrinsvis én, der er udvalgt fra gruppen, der består af en CMV-promotor, en a-actinpromotor, en insulinpromotor, en enolasepromotor, en BDNF-promotor, en NGF-promotor, en EGF-promotor, en vækstfaktorpromotor, en axonspecifik promotor, en dendritspecifik promotor, en hjernespecifik promotor, en hippocampusspecifik promotor, en nyrespecifik promotor, en elafinpromotor, en cytokinpromotor, en interferonpromotor, en vækstfaktorpromotor, en og-antitrypsinpromotor, en hjernespecifik promotor, en neuralcellespecifik promotor, en central nervesystemcellespecifik promotor, en perifer nervesystemcellespecifik promotor, en interleukinpromotor, en serpinpromotor, en hybrid CMV-promotor, en hybrid a-actinpromotor, en EFl-promotor, en Ula-promotor, en Ulb-promotor, en Tet-inducerbar promotor og en VP16-lexA-promotor. 7. rAAV-vektor ifølge et hvilket som helst af kravene 3 til 6, hvor det mindst ene første nukleinsyresegment er yderligere operabelt koblet til en posttranskriptionel regulatorsekvens eller et polyadenyleringssignal og fortrinsvis til et posttranskriptionelt regulatorelement fra hepatitisvirus fra skovmurmeldyr. 8. rAAV-vektor ifølge et hvilket som helst af kravene 3 til 7, hvor det terapeutiske middel er udvalgt fra gruppen, der består af et polypeptid, et peptid, et antistof, et antigenbindende fragment, et ribozym, en peptidnukleinsyre, et siRNA, et RNAi, et antisense-oligonukleotid og et antisense-polynukleotid; fortrinsvis hvor det terapeutiske middel er et protein eller polypeptid, der er udvalgt fra gruppen, der består af en adrenerg agonist, en anti-apoptosefaktor, en apoptoseinhibitor, en cytokinreceptor, et cytokin, et cytotoksin, et erytropoietisk middel, en glutaminsyredecarboxylase, et glycoprotein, en vækstfaktor, en vækstfaktorreceptor, et hormon, en hormonreceptor, en interferon, et interleukin, en interleukinreceptor, en kinase, en kinaseinhibitor, en nervevækstfaktor, et netrin, et neuroaktivt peptid, en receptor for neuroaktivt peptid, en neurogen faktor, en receptor for neurogen faktor, et neuropilin, en neurotrofisk faktor, et neurotrophin, en neurotrophinreceptor, en N-methyl-D-aspartatantagonist, et plexin, en protease, en proteaseinhibitor, en proteindecarboxylase, en proteinkinase, en proteinkinaseinhibitor, et proteolytisk protein, en inhibitor af proteolytisk protein, et semaphorin, en semaphorinreceptor, et serotonintransportprotein, en serotoninoptagelsesinhibitor, en serotoninreceptor, et serpin, en serpinreceptor og en tumorsuppressor; og mere fortrinsvis hvor det terapeutiske middel er et polypeptid, der er udvalgt fra gruppen, der består af BDNF, CNTF, CSF, EGF, FGF, G-SCF, GMCSF, gonadotropin, I FN, IFG-1, M-CSF, NGF, PDGF, PEDF, TGF, TGF-B2, TNF, VEGF, prolactin, somatotropin, XIAP1, IL-1, IL-2, IL-3, IL-4, IL-5, IL-6, IL-7, IL-8, IL-9, IL-10, IL10(I87A), viral IL-10, IL-11, IL-12, IL-13, IL-14, IL-15, IL-16, IL-17 og IL-18 . 9. rAAV-vektor ifølge et hvilket som helst af kravene 3 til 8, hvor det første nukleinsyresegment omfatter en sekvensregion, der koder for et peptid, et polypeptid, et antistof eller et antigenbindende fragment af human, murin, aviær, porcin, bovin, ovin, felin, canin, equin, epin, caprin, lupin (ulv) eller ikke-human primat oprindelse.
10. Isoleret mammaliaværtscelle, der omfatter rAAV-vektoren ifølge et hvilket som helst af ovennævnte krav, fortrinsvis en isoleret mammaliaværtscelle, der er udvalgt fra gruppen, der består af en human, ikke-human primat, murin, felin, canin, porcin, ovin, bovin, equin, epin, caprin og lupin værtscelle; og mere fortrinsvis en isoleret human endotel-, epitel-, vaskulær-, lever-, lunge-, hjerte-, pankreas-, intestinal-, nyre-, muskel-, knogle-, nerve-, blod- eller hjernecelle. 11. rAAV-vektor ifølge et hvilket som helst af kravene 1 til 9 til anvendelse til terapi. 12. rAAV-vektor ifølge et hvilket som helst af kravene 1 til 9 til anvendelse til diagnosticering, forebyggelse, behandling eller lindring af et eller flere symptomer på en sygdom, en skade, en forstyrrelse, et traume eller en dysfunktion hos et pattedyr og yderligere fortrinsvis til anvendelse til behandling, forebyggelse eller lindring af et eller flere symptomer på cancer, diabetes, autoimmunsygdom, nyresygdom, kardiovaskulær sygdom, pankreassygdom, intestinal sygdom, leversygdom, neurologisk sygdom, neuromuskulær forstyrrelse, neuromotorisk funktionsforstyrrelse, nedsat neuroskeletfunktion, nedsat neurologisk funktion, neurosensorisk dysfunktion, apopleksi, iskæmi, spiseforstyrrelse, og-antitrypsin (AAT)-mangel, Battens sygdom, Alzheimers sygdom, Huntingtons sygdom, Parkinsons sygdom, skeletsygdom, traume eller lungesygdom hos et pattedyr.
13. Anvendelse af rAAV-vektoren ifølge et hvilket som helst af kravene 1 til 9 til fremstilling af et medikament til anvendelse til diagnosticering, forebyggelse, behandling eller lindring af et eller flere symptomer på en sygdom, en skade, en forstyrrelse, et traume eller en dysfunktion hos et pattedyr og yderligere fortrinsvis til anvendelse til behandling, forebyggelse eller lindring af et eller flere symptomer på cancer, diabetes, autoimmunsygdom, nyresygdom, kardiovaskulær sygdom, pankreassygdom, intestinal sygdom, leversygdom, neurologisk sygdom, neuromuskulær forstyrrelse, neuromotorisk funktionsforstyrrelse, nedsat neuroskeletfunktion, nedsat neurologisk funktion, neurosensorisk dysfunktion, apopleksi, iskæmi, spiseforstyrrelse, og-antitrypsin (AAT)-mangel, Battens sygdom, Alzheimers sygdom, Huntingtons sygdom, Parkinsons sygdom, skeletsygdom, traume eller lungesygdom hos et pattedyr.
14. Fremgangsmåde til forøgelse af transduktionseffektiviteten af en rekombinant adenoassocieret virus (rAAV)-vektor, hvilken fremgangsmåde omfatter trinet mutering af mindst én overfladeeksponeret tyrosinrest i capsidproteinerne, hvor den mindst ene overfladeeksponerede tyrosinrest svarer til Tyr252, Tyr272, Tyr444, Tyr500, Tyr700, Tyr704, Tyr730, Tyr275, Tyr281, Tyr508, Tyr576, Tyr612, Tyr673 eller Tyr720 i vildtype-AAV2-capsidproteinet, således at transduktionseffektiviteten i en mammaliacelle af vektoren, der omfatter det muterede capsidprotein, er højere end transduktionseffektiviteten for en tilsvarende AAV, der opretholder de overfladeeksponerede tyrosinrester i capsidproteinet, hvor mammaliaværtscellen er den humane cervikalcellelinje HeLa.
15. Fremgangsmåde ifølge krav 14, hvor den mindst ene muterede tyrosinrest er en eller flere af Tyr252, Tyr272, Tyr444, Tyr500, Tyr700, Tyr704 eller Tyr730.
16. Fremgangsmåde ifølge krav 14 eller 15, hvor den mindst ene muterede overfladeeksponerede tyrosinrest er substitueret med phenylalanin.
17. Fremgangsmåde ifølge krav 14, 15 eller 16, hvor den rekombinante adenoassocierede virus (rAAV)-vektor er udvalgt fra gruppen, der består af en AAV-serotype 1, en AAV-serotype 2, en AAV-serotype 3, en AAV-serotype 4, en AAV-serotype 5 og en AAV-serotype 6.
DK08733161.7T 2007-04-09 2008-04-08 Raav-vektorsammensætninger med tyrosinmodificerede capsidproteiner og fremgangsmåder til anvendelse deraf DK2191001T3 (da)

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