Improved raav expression systems and methods for enhancing transduction of mammalian neural cells

作者: William W. Hauswirth , Nicholas Muzyczka , Corinna Burger , Ronald J. Mandel , Sergei Zolotukhin

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摘要: Disclosed are serotype-specific recombinant adeno-associated viral (rAAV) vectors, as well particles and compositions comprising them, useful in the expression of neurotherapeutic agents (including peptides polypeptides) selected mammalian neural cells, tissues organ systems them. In particular embodiments, rAAV serotype 1 5 vectors disclosed for delivery therapeutic to cells affected mammals.

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