Gene therapy by secretory gland expression

Details Gene therapy by secretory gland expression Gene therapy by secretory gland expression

C - Chemistry, Metallurgy

12

N

C12N 5/10 (2006.01) A61K 35/39 (2006.01) A61K 35/55 (2006.01) A61K 38/17 (2006.01) A61K 38/18 (2006.01) A61K 38/27 (2006.01) A61K 38/28 (2006.01) A61K 38/37 (2006.01) A61K 48/00 (2006.01) C12N 15/85 (2006.01)

Patent

CA 2216149

Secretory gland cells, particularly pancreatic and salivary gland cells, are genetically altered to operatively incorporate a gene which expresses a protein which has a desired therapeutic effect on a mammalian subject. The expressed protein is secreted directly into the gastrointestinal tract and/or blood stream to obtain therapeutic blood levels of the protein thereby treating the patient in need of the protein. The transformed secretory gland cells provide long term therapeutic cure for diseases associated with a deficiency in a particular protein or which are amenable to treatment by overexpression of a protein.

Cellules sécrétrices, en particulier cellules pancréatiques et salivaires modifiées génétiquement de manière à intégrer un gène qui exprime une protéine ayant un effet thérapeutique désiré sur un mammifère. La protéine exprimée est secrétée directement dans le tractus gastro-intestinal et/ou la circulation sanguine afin d'obtenir des concentrations sanguines thérapeutiques de la protéine pour traiter les patients qui ont besoin de la protéine. Les cellules sécrétrices ainsi transformées assurent permettent une intervention thérapeutique de longue durée pour des maladies associées à une carence d'une protéine particulière ou qui sont justiciables d'un traitement caractérisé par la surexpression d'une protéine.

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