C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/86 (2006.01) C12N 7/04 (2006.01) C12N 15/864 (2006.01)
Patent
CA 2406743
A pseudotyped rAAV is described, which contains sequences derived from AAV5. A method for producing rAAV pseudotyped with AAV5 capsid is described in which the rep proteins of the serotype or any cross-reactive serotype of the AAV ITRS are utilized. A similar method may be provided to generate a pseudotyped rAAV in which minigenes carrying AAV5 ITRs are packaged in capsids of a heterologous AAV serotype. Also provided are pharmaceutical compositions containing the pseudotyped rAAV and methods of using them for gene delivery.
L'invention concerne un rAAV pseudotypé contenant des séquences provenant de AAV5, ainsi qu'un procédé pour produire rAAV pseudotypé doté d'une capside AAV5, selon lequel on utilise les protéines Rep du sérotype ou de tout sérotype à activité croisée des AAV ITR. L'invention concerne également un procédé similaire pour générer un rAAV pseudotypé, selon lequel des minigènes portant des AAV5 ITR sont enfermés dans des capsides d'un sérotype hétérologue AAV. L'invention porte aussi sur des compositions pharmaceutiques contenant rAAV pseudotypé et sur des procédés pour les utiliser dans le transfert de gène.
Auricchio Alberto
Hildinger Markus
Wilson James M.
Gowling Lafleur Henderson Llp
The Trustees Of The University Of Pennsylvania
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