Raav vector compositions having tyrosine-modified capsid...

Details Raav vector compositions having tyrosine-modified capsid... Raav vector compositions having tyrosine-modified capsid...

C - Chemistry, Metallurgy

12

N

C12N 15/864 (2006.01) A61K 38/16 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2720097

Disclosed are tyrosine-modified rAAV vectors, as well as infectious virions, compositions, and pharmaceutical formulations that comprise them. Also disclosed are methods of preparing and methods for using the disclosed tyrosine-phosphorylated capsid protein mutant rAAV vectors in a variety of diagnostic and therapeutic applications including in vivo and ex vivo gene therapy, and large-scale production of rAAV vectors.

L'invention concerne des vecteurs rAAV modifiés par la tyrosine, ainsi que des virions infectieux, des compositions et des formulations pharmaceutiques qui les comprennent. L'invention concerne également des procédés de préparation et des procédés d'utilisation desdits vecteurs rAAV mutants à protéines de capside phosphorylées par la tyrosine dans une pluralité d'applications diagnostiques et thérapeutiques, y compris la thérapie génique in vivo et ex vivo, ainsi que la production à large échelle de vecteurs rAAV.

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