GENE TRANSFER INTO AIRWAY EPITHELIAL STEM CELL BY USING LENTIVIRAL VECTOR PSEUDOTYPED WITH RNA VIRUS OR DNA VIRUS SPIKE PROTEIN

摘要

The present inventors successfully introduced genes into stem cells of airway epithelial tissues using simian immunodeficiency virus vectors pseudotyped with F and HN, which are envelope glycoproteins of Sendai virus. Gene transfer into airway epithelial tissue stem cells using a vector of the present invention is useful for gene therapy of genetic respiratory diseases such as cystic fibrosis. Furthermore, it is possible to select respiratory organs such as the lungs as production tissues for providing proteins that are deficient due to genetic diseases.

主权项

1. A method of treating a genetic respiratory disease, comprising directly contacting airway cells of an individual in need thereof with a recombinant simian immunodeficiency virus (SIV) vector comprising a foreign gene operably linked to a promoter, the vector pseudotyped with spike proteins of Sendai virus envelope glycoproteins F and HN, wherein the vector integrates the foreign gene into an airway epithelial stem cell genome,
wherein the airway cells are not pretreated or pre-conditioned prior to contact with the vector, and wherein the integrated foreign gene encodes a therapeutic agent for the genetic respiratory disease and is expressed in the airway epithelial stem cell for at least 160 days.