Gene transfer into airway epithelial stem cell by using...

Details Gene transfer into airway epithelial stem cell by using... Gene transfer into airway epithelial stem cell by using...

C - Chemistry, Metallurgy

12

N

C12N 15/867 (2006.01) A61K 35/76 (2006.01) A61K 38/00 (2006.01) A61K 48/00 (2006.01) A61P 11/00 (2006.01) C12N 5/10 (2006.01)

Patent

CA 2627485

A gene is successfully transferred into airway epithelial stem cells by using a monkey immunodeficiency virus vector having been pseudotyped with Sendai virus envelope glycoproteins F and HN. This gene transfer into airway epithelial stem cells with the use of the vector as described above is useful in gene therapy for a hereditary respiratory disease such as cystic fibrosis. Moreover, it becomes possible to select a respiratory organ such as the lung as a tissue that is capable of producing and supplying a protein deficient in a hereditary disease.

La présente invention divulgue qu~il est possible de transférer un gène dans des cellules souches épithéliales des voies respiratoires au moyen d~un vecteur viral d~immunodéficience simien que l'on a pseudotypé avec des glycoprotéines F et HN de l~enveloppe du virus Sendai. Le transfert de gène ainsi effectué de la manière décrite ci-dessus s~avère utile pour le traitement par thérapie génique d'une maladie respiratoire héréditaire telle que la mucoviscidose. Il devient en outre possible de sélectionner un organe respiratoire tel le poumon en tant que tissu capable de produire et de fournir une protéine qui est manquante dans une maladie héréditaire.

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