Genetic modification of the lung as a portal for gene delivery
AssigneeGenzyme Corporation
InventorsChester Li, Robin Ziegler, Seng Cheng, Nelson Yew
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Abstract
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.
Claims15
A method for treatment of a patient suffering from a systemic disorder or disease, comprising administering to the lung a transgene delivery vector, said transgene delivery vector comprising a nucleotide sequence which encodes for a therapeutic protein, such that the transgene delivery vector transfects lung cells, expresses the therapeutic protein, and the therapeutic protein enters into the patient's circulatory system.
Dependencies are inferred from the claim text.