Targeting gene therapy to airway epithelia

Gene therapy approaches for treating cystic fibrosis have been stymied by the low efficiency of gene transfer to the airway epithelia. On page 635, Kreda et al. overcome this problem by retargeting a viral vector to a G protein-coupled receptor (GPCR) that is abundantly expressed in airway epithelial cells. They show that conjugation of a receptor's natural small-molecule agonist UTP will direct an adenovirus vector to a specific GPCR. Binding of the agonist to the receptor mediates internalization of the vector and gene transfer to the human airway epithelial cells. This approach potentially could be extended to other tissues by designing ligand–viral vector conjugates directed toward other tissue-specific GPCRs.

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Sinclair, M. Targeting gene therapy to airway epithelia. Nat Biotechnol 18, 586 (2000). https://doi.org/10.1038/76388

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