Genetic modification of the lung as a portal for gene delivery

Details Genetic modification of the lung as a portal for gene delivery Genetic modification of the lung as a portal for gene delivery

A - Human Necessities

61

K

A61K 48/00 (2006.01) A61K 9/00 (2006.01) A61K 31/66 (2006.01) A61K 31/663 (2006.01) A61K 38/37 (2006.01) A61K 38/46 (2006.01) A61K 38/47 (2006.01) A61K 38/48 (2006.01) A61P 3/00 (2006.01) A61P 7/04 (2006.01) A61P 37/04 (2006.01) C12N 9/18 (2006.01) C12N 9/24 (2006.01) C12N 9/40 (2006.01)

Patent

CA 2400634

The present invention relates to methods for treatment of systemic disorders using the lung as a depot organ for transgene delivery. Transfection of the pulmonary epithelium, particularly the deep alveolar cells, or pulmonary endothelial cells, is achieved via local administration of a transgene delivery vector to the lung. The transfected cells express the transgene, and the protein thereby expressed is communicated into the circulatory system. Once entering into the circulatory system, the protein is able to achieve a systemic therapeutic effect.

La présente invention concerne des méthodes de traitement des troubles systémiques dans lesquelles le poumon est utilisé comme organe de stockage pour l'administration de transgènes. La transfection de l'épithélium pulmonaire, en particulier des cellules alvéolaires profondes, ou des cellules endothéliales pulmonaires, s'effectue via l'administration locale d'un vecteur d'administration de transgène au poumon. Les cellules transfectées expriment le transgène, et la protéine ainsi exprimée pénètre dans le système circulatoire. Une fois entrée dans le système circulatoire, la protéine peut avoir un effet thérapeutique systémique.

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