DE23193606T1 - Verfahren zur erhöhung der funktion eines aav-vektors - Google Patents

Verfahren zur erhöhung der funktion eines aav-vektors Download PDF

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DE23193606T1
DE23193606T1 DE23193606.3T DE23193606T DE23193606T1 DE 23193606 T1 DE23193606 T1 DE 23193606T1 DE 23193606 T DE23193606 T DE 23193606T DE 23193606 T1 DE23193606 T1 DE 23193606T1
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aav
seq
proteins
amino acids
capsid
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Luk Vandenberghe
Guangping Gao
James N. Wilson
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University of Pennsylvania Penn
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Abstract

Ein adeno-assoziiertes Virus (AAV), umfassend ein AAV-Kapsid und ein Minigen, das AAV-invertierte terminale Wiederholungen und ein heterologes Gen aufweist, das funktionsfähig mit regulatorischen Sequenzen verbunden ist, die die Expression des heterologen Gens in einer Wirtszelle steuern, wobei das AAV-Kapsid AAV-vp1-Proteine, AAV-vp2-Proteine und AAV-vp3-Proteine umfasst, wobei die AAV-vp1-Proteine i) die Sequenz der Aminosäuren 1 bis 738 von SEQ ID NO: 4 (AAVrh46) mit einer R478K-Modifikation oder ii) eine Sequenz mit mindestens 95 % Identität mit der vollen Länge der Aminosäuren 1 bis 738 von SEQ ID NO: 4 haben, wobei der Aminosäurerest 478 K (R478K) ist, wenn er entlang der vollen Länge der Aminosäuren 1 bis 738 von SEQ ID NO: 4 ausgerichtet ist.

Claims (9)

  1. Ein adeno-assoziiertes Virus (AAV), umfassend ein AAV-Kapsid und ein Minigen, das AAV-invertierte terminale Wiederholungen und ein heterologes Gen aufweist, das funktionsfähig mit regulatorischen Sequenzen verbunden ist, die die Expression des heterologen Gens in einer Wirtszelle steuern, wobei das AAV-Kapsid AAV-vp1-Proteine, AAV-vp2-Proteine und AAV-vp3-Proteine umfasst, wobei die AAV-vp1-Proteine i) die Sequenz der Aminosäuren 1 bis 738 von SEQ ID NO: 4 (AAVrh46) mit einer R478K-Modifikation oder ii) eine Sequenz mit mindestens 95 % Identität mit der vollen Länge der Aminosäuren 1 bis 738 von SEQ ID NO: 4 haben, wobei der Aminosäurerest 478 K (R478K) ist, wenn er entlang der vollen Länge der Aminosäuren 1 bis 738 von SEQ ID NO: 4 ausgerichtet ist.
  2. Das AAV nach Anspruch 1, wobei die invertierten terminalen AAV-Wiederholungen von einem anderen AAV stammen als das AAV, das die Capsidproteine liefert.
  3. Das AAV nach einem der Ansprüche 1 bis 2, wobei das heterologe Gen für ein Dystrophin-Genprodukt kodiert.
  4. Das AAV nach Anspruch 3, wobei das Dystrophin-Genprodukt Mikro-Dystrophin ist.
  5. Das AAV nach einem der Ansprüche 1 bis 2, wobei das heterologe Gen für Faktor IX kodiert.
  6. Eine Zusammensetzung, die das AAV nach einem der Ansprüche 1 bis 5 und einen physiologisch verträglichen Träger umfasst.
  7. Ein AAV-Vektor nach einem der Ansprüche 1 bis 5 oder eine Zusammensetzung nach Anspruch 6, zur Verwendung bei der Verabreichung eines Genprodukts an ein Subjekt.
  8. Eine Wirtszelle, die mit dem AAV nach einem der Ansprüche 1 bis 5 transduziert ist.
  9. Ein Verfahren zur Erzeugung eines rekombinanten AAV, das ein AAV-Kapsid enthält umfassend die Schritte zur Kultivierung einer Wirtszelle, die enthält: (a) eine Nukleinsäuresequenz, die für ein AAV-Kapsidprotein kodiert, ausgewählt aus SEQ ID NO: 4 mit einer R478K-Modifikation oder einer Sequenz, die mindestens 95 % Identität damit aufweist, wenn sie entlang der vollen Länge von SEQ ID NO: 4 ausgerichtet ist, wobei der Aminosäurerest 478 K (R478K) ist, wenn er entlang der gesamten Länge der Aminosäuren 1 bis 738 von SEQ ID NO: 4 ausgerichtet ist; (b) ein funktionelles rep-Gen; (c) ein Minigen, das AAV-invertierte terminale Wiederholungen (ITRs) und ein Transgen umfasst; und (d) ausreichende Hilfsfunktionen, um die Einbindung des Minigenes in das AAV-Kapsid zu ermöglichen.
DE23193606.3T 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors Pending DE23193606T1 (de)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US66908305P 2005-04-07 2005-04-07
US669083P 2005-04-07
US73349705P 2005-11-04 2005-11-04
US733497P 2005-11-04
EP23193606.3A EP4282957A3 (de) 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors

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DE23193606T1 true DE23193606T1 (de) 2024-02-15

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DE23193606.3T Pending DE23193606T1 (de) 2005-04-07 2006-04-07 Verfahren zur erhöhung der funktion eines aav-vektors

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US (13) US8999678B2 (de)
EP (12) EP4234687A2 (de)
JP (5) JP5702519B2 (de)
CN (6) CN112029737A (de)
DE (2) DE23193601T1 (de)
DK (2) DK2383346T3 (de)
ES (6) ES2426245T3 (de)
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