Recombinant adeno-associated virus-mediated gene transfer...

Details Recombinant adeno-associated virus-mediated gene transfer... Recombinant adeno-associated virus-mediated gene transfer...

A - Human Necessities

61

K

A61K 48/00 (2006.01) A61K 31/70 (2006.01) A61K 38/36 (2006.01) A61K 38/37 (2006.01) A61K 38/48 (2006.01) A61P 7/04 (2006.01) C07K 14/755 (2006.01) C12N 9/64 (2006.01) C12N 15/861 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2441454

Methods for introducing recombinant adeno-associated virus (rAAV) virions intro a cell or cells of a secretory gland are described. Recombinant AAV virions containing a heterologous gene are introduced into a duct of a secretory gland resulting in transduction of one or more secretory gland cells. Once a secretory gland cell is transduced by the rAAV virion, the heterologous gene is expressed and the expression product is then secreted. Exemplary examples of secretory glands are the liver, the submandibular gland, the parotid gland, and the sublingual gland. Using the methods of the invention, therapeutic levels of a protein are achieved. Methods for treating hemophilia are also disclosed.

l'invention concerne des procédés d'introduction de virions de virus recombiné adéno-associé (rAAV) dans une cellule ou dans des cellules d'une glande sécrétrice. Des virions AAV recombinés contenant un gène hétérologue sont introduits dans le conduit d'une glande sécrétrice résultant en une transduction d'une ou plusieurs cellules de glande sécrétrice. Lorsqu'une cellule de glande sécrétrice est transduite par le virion rAAV, le gène hétérologue est exprimé et le produit d'expression est sécrété. Des exemples de glandes sécrétrices sont le foie, la glande submandibulaire, la glande parotide, et la glande sublinguale. L'utilisation des procédés de l'invention permet d'obtenir des taux thérapeutiques de protéine. L'invention concerne aussi des procédés de traitement de l'hémophilie.

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