DK2292780T3 - Clader og sekvenser af adenoassocieret virus (AAV), vektorer, der indeholder dem, og anvendelser deraf - Google Patents
Clader og sekvenser af adenoassocieret virus (AAV), vektorer, der indeholder dem, og anvendelser deraf Download PDFInfo
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- DK2292780T3 DK2292780T3 DK10182796.2T DK10182796T DK2292780T3 DK 2292780 T3 DK2292780 T3 DK 2292780T3 DK 10182796 T DK10182796 T DK 10182796T DK 2292780 T3 DK2292780 T3 DK 2292780T3
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Claims (15)
1. Adenoassocieret virus (AAV), der omfatter mindst et AAV-capsid, som omfatter aminosyrerae i til 734 i SEQ ID NO: 186 (AAVhu.53) eller SEQ ID NO: 187 (hu.55) eller SEQ ID NO: 188 (hu.54), hvor AAV’en yderligere omfatter et minigen, som har inverterede terminale repeats fra AAV og et heterologt gen, der er operativt forbundet til regulatorsekvenser, som styrer dets ekspression i en værtscelle.
2. AAV ifølge krav 1, hvor capsidet kodes for af nukleinsyresekensen i SEQ ID NO: 176 (hu.53).
3. AAV ifølge krav 1, hvor capsidet kodes for af nukleinsyresekensen i SEQ ID NO: 177 (hu.54).
4. AAV ifølge krav 1, hvor capsidet kodes for af nukleinsyresekensen i SEQ ID NO: 178 (hu.55).
5. AAV ifølge et hvilket som helst af kravene 1 til 4, hvor de inverterede terminale repeats er fra en AAV, der er heterolog til den AAV, der leverer eapsidproteinet.
6. Isoleret capsidprotein fra adenoassocieret virus, hvilket protein er valgt, fra gruppen bestående af; vp 1 -capsidprotein, som har aminosyresekvensen 1 til 734 i SEQ ID NO: 186, SEQ ID NO: 187 eller SEQ ID NO: 188: vp2-capsidprotein, som har aminosyresekvensen 138 til 734 i SEQ ID NO: 186, SEQ ID NO: 187 eller SEQ ID NO: 188; og vp3-capsidprotein, som har aminosyresekvensen 204 til 734 i SEQ ID NO: 186, SEQ ID NO: 187 eller SEQ ID NO: 188.
7. Nukleinsyremolekyie, der omfatter en nukleinsyresekvens, som koder for et protein ifølge krav 6.
8. Nukleinsyremolekyie ifølge krav 7, hvor nukleinsyresekvensen er valgt fra gruppen bestående af: vpl, som har nukleotidsekvensen 1 til 2206 i SEQ ID NO: 176; vp2, som har nukleotidsekvensen 411 til 2206 i SEQ ID NC): 176; og vp3, som har nukleotidsekvensen 609 til 2206 i SEQ ID NO: 176.
9. Nukleinsyremolekyie ifølge krav 7 eller 8, hvor nukleinsyremolekylet yderligere omfatter en AAV-sekvens, der koder for et funktionelt AAV-rep-protein.
10. Nukleinsyremolekyie ifølge et hvilket som helst af kravene 7 til 9, hvor molekylet er et plasmid.
11. Fremgangsmåde til fembringeise af en rekombinant adenoassocieret virus (AAV), der omfatter et AAV-capsid, hvilken fremgangsmåde omfatter trinnene, der går ud på at dyrke en vantscelle, som indeholder: (a) et molekyle, der koder for et AAV-capsidprotein; (h) et funktionelt rep-gen; (c) et minigen, der omfatter inverterede terminale repeats (TTR) fra AAV og et transgen; og (d) tilstrækkelige hjælpefunktioner til at muliggøre pakningen af minigenet i AAV-capsidproteinet, hvor værtscellen omfatter et nukleinsyremolekyie ifølge et hvilket som helst af kravene 7 til 10.
12. Isoleret værtscelle, der omfatter en adenoassocieret virus ifølge et hvilket som helst af kravene 1 til 5 eller et nukleinsyremolekyie ifølge et hvilket som helst af kravene 7 til 10.
13. Sammensætning, der omfatter en AAV ifølge et hvilket som helst af kravene I til 5 eller et nukleinsyremolekyie ifølge et hvilket som helst af kravene 7 til 10 og en fysiologisk forenelig hatrer.
14. Anvendelse af en AAV ifølge et hvilket som helst af kravene 1 til 5 ved fremstillingen af et lægemiddel til at transportere et molekyle til en celle.
15. AA V ifølge et hvilket som helst af kravene I til 5 til anvendelse ved transport af et molekyle til en celle.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
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US50822603P | 2003-09-30 | 2003-09-30 | |
US56654604P | 2004-04-29 | 2004-04-29 | |
EP04788580A EP1668143B1 (en) | 2003-09-30 | 2004-09-30 | Adeno-associated virus (aav) clades, sequences, vectors containing same, and uses therefor |
Publications (1)
Publication Number | Publication Date |
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DK2292780T3 true DK2292780T3 (da) | 2017-12-04 |
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ID=34426044
Family Applications (4)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK10182587.5T DK2292779T3 (da) | 2003-09-30 | 2004-09-30 | Adenoassocierede virus (aav)-clader, sekvenser, vektorer indeholdende samme og anvendelser deraf |
DK10182098.3T DK2345731T3 (da) | 2003-09-30 | 2004-09-30 | Adenoassocieret virus (AAV)-grupperinger, sekvenser, vektorer indeholdende samme og anvendelser deraf |
DK17153156.9T DK3211085T3 (da) | 2003-09-30 | 2004-09-30 | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
DK10182796.2T DK2292780T3 (da) | 2003-09-30 | 2004-09-30 | Clader og sekvenser af adenoassocieret virus (AAV), vektorer, der indeholder dem, og anvendelser deraf |
Family Applications Before (3)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
DK10182587.5T DK2292779T3 (da) | 2003-09-30 | 2004-09-30 | Adenoassocierede virus (aav)-clader, sekvenser, vektorer indeholdende samme og anvendelser deraf |
DK10182098.3T DK2345731T3 (da) | 2003-09-30 | 2004-09-30 | Adenoassocieret virus (AAV)-grupperinger, sekvenser, vektorer indeholdende samme og anvendelser deraf |
DK17153156.9T DK3211085T3 (da) | 2003-09-30 | 2004-09-30 | Klader af adeno-associeret virus (aav), sekvenser, vektorer indeholdende disse og anvendelser deraf |
Country Status (17)
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US (13) | US7906111B2 (da) |
EP (7) | EP2298926A1 (da) |
JP (9) | JP5054975B2 (da) |
CN (4) | CN102212558B (da) |
AU (2) | AU2004278684B2 (da) |
BE (1) | BE2021C533I2 (da) |
CA (3) | CA2537793C (da) |
CY (2) | CY1119833T1 (da) |
DK (4) | DK2292779T3 (da) |
ES (5) | ES2648241T3 (da) |
FR (1) | FR21C1037I1 (da) |
HU (4) | HUE033158T2 (da) |
NZ (1) | NZ545628A (da) |
PL (2) | PL3211085T3 (da) |
PT (2) | PT3211085T (da) |
SI (2) | SI3211085T1 (da) |
WO (1) | WO2005033321A2 (da) |
Families Citing this family (519)
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ATE520707T1 (de) | 2001-11-13 | 2011-09-15 | Univ Pennsylvania | Verfahren für den nachweis und/oder die identifikation von sequenzen des adeno- assoziierten virus (aav) sowie isolation von dadurch identifizierten, neuen sequenzen |
EP2359869B1 (en) | 2001-12-17 | 2018-12-26 | The Trustees Of The University Of Pennsylvania | Adeno-associated virus (AAV) serotype 8 sequences, vectors containing same and uses therefor |
US9233131B2 (en) | 2003-06-30 | 2016-01-12 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
US9441244B2 (en) | 2003-06-30 | 2016-09-13 | The Regents Of The University Of California | Mutant adeno-associated virus virions and methods of use thereof |
EP2298926A1 (en) * | 2003-09-30 | 2011-03-23 | The Trustees of The University of Pennsylvania | Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof |
ES2442225T3 (es) | 2004-04-28 | 2014-02-10 | The Trustees Of The University Of Pennsylvania | Régimen de inmunización con cebado de adenovirus suprimido en E4 y potenciación de adenovirus suprimido en E1 |
DE602005026269D1 (de) | 2004-04-28 | 2011-03-24 | Univ Pennsylvania | Sequenzielle freisetzung immunogener moleküle über adenoviren und adeno-assoziierte viren vermittelte abgaben |
EP3409296A1 (en) | 2005-04-07 | 2018-12-05 | The Trustees of the University of Pennsylvania | Method of increasing the function of an aav vector |
WO2007120542A2 (en) | 2006-03-30 | 2007-10-25 | The Board Of Trustees Of The Leland Stanford Junior University | Aav capsid library and aav capsid proteins |
DK2009990T3 (da) * | 2006-04-07 | 2017-01-09 | Univ Texas | Fremgangsmåder og sammensætninger relateret til adenoassocierede virusfagpartikler |
US9198984B2 (en) | 2006-04-28 | 2015-12-01 | The Trustees Of The University Of Pennsylvania | Scalable production method for AAV |
US20090317417A1 (en) * | 2006-04-28 | 2009-12-24 | The Trustees Of The University Of Pennsylvania | Modified AAV Vectors Having Reduced Capsid Immunogenicity and Use Thereof |
US9725485B2 (en) | 2012-05-15 | 2017-08-08 | University Of Florida Research Foundation, Inc. | AAV vectors with high transduction efficiency and uses thereof for gene therapy |
HUE030719T2 (en) | 2007-04-09 | 2017-05-29 | Univ Florida | RAAV vector formulations and methods comprising tyrosine-modified capsid proteins for their use |
EP2019143A1 (en) * | 2007-07-23 | 2009-01-28 | Genethon | CNS gene delivery using peripheral administration of AAV vectors |
EP2058401A1 (en) * | 2007-10-05 | 2009-05-13 | Genethon | Widespread gene delivery to motor neurons using peripheral injection of AAV vectors |
WO2009108274A2 (en) * | 2008-02-26 | 2009-09-03 | The University Of North Carolina At Chapel Hill | Methods and compositions for adeno-associated virus (aav) with hi loop mutations |
US8632764B2 (en) | 2008-04-30 | 2014-01-21 | University Of North Carolina At Chapel Hill | Directed evolution and in vivo panning of virus vectors |
US9217155B2 (en) | 2008-05-28 | 2015-12-22 | University Of Massachusetts | Isolation of novel AAV'S and uses thereof |
US11219696B2 (en) | 2008-12-19 | 2022-01-11 | Nationwide Children's Hospital | Delivery of polynucleotides using recombinant AAV9 |
EP2396343B1 (en) | 2009-02-11 | 2017-05-17 | The University of North Carolina At Chapel Hill | Modified virus vectors and methods of making and using the same |
EP2425000B1 (en) | 2009-04-30 | 2019-02-20 | The Trustees Of The University Of Pennsylvania | Compositions for targeting conducting airway cells comprising adeno-associated virus constructs |
US8734809B2 (en) | 2009-05-28 | 2014-05-27 | University Of Massachusetts | AAV's and uses thereof |
WO2010141706A1 (en) * | 2009-06-03 | 2010-12-09 | Cedars-Sinai Medical Center | Effective vector platform for gene transfer and gene therapy |
US20120244127A1 (en) | 2009-10-01 | 2012-09-27 | The Trustees Of The University Of Pennsylvania | AAV Vectors Expressing SEC10 for Treating Kidney Damage |
SG10201908848RA (en) | 2010-03-29 | 2019-10-30 | Univ Pennsylvania | Pharmacologically induced transgene ablation system |
US9315825B2 (en) | 2010-03-29 | 2016-04-19 | The Trustees Of The University Of Pennsylvania | Pharmacologically induced transgene ablation system |
CA2833905C (en) | 2010-04-23 | 2019-09-10 | University Of Massachusetts | Multicistronic expression constructs |
CA3066596A1 (en) | 2010-04-23 | 2011-10-27 | University Of Massachusetts | Cns targeting aav vectors and methods of use thereof |
CA2833912C (en) | 2010-04-23 | 2021-09-21 | University Of Massachusetts | Aav-based treatment of cholesterol-related disorders |
US8663624B2 (en) | 2010-10-06 | 2014-03-04 | The Regents Of The University Of California | Adeno-associated virus virions with variant capsid and methods of use thereof |
CN107828820B (zh) * | 2010-10-27 | 2022-06-07 | 学校法人自治医科大学 | 用于向神经系统细胞导入基因的腺相关病毒粒子 |
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