Barbara R Grubb

UNCCH, School of Medicine, Medicine

Scopus Publication Detail

The publication detail shows the title, authors (with indicators showing other profiled authors), information on the publishing organization, abstract and a link to the article in Scopus. This abstract is what is used to create the fingerprint of the publication.

Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans

Barbara R. Grubb; Raymond J. Pickles; Hong Ye; James R. Yankaskas; Ralph N. Vick; John F. Engelhardt; James M. Wilson; Larry G. Johnson; Richard C. Boucher (Profiled Authors: Raymond J Pickles; James R Yankaskas; Barbara R Grubb; Richard C Boucher; Ralph N Vick)

Nature. 1994;371(6500):802-806.

The success of adenoviral vectors for gene therapy of lung disease in cystic fibrosis (CF) depends on efficient transfer of the complementary DNA encoding the correct version of the cystic fibrosis transmembrane regulator (CFTR) to the affected columnar epithelial cells lining the airways of the lung. Pre-clinical studies in vitro suggest that low doses of adenovirus vectors carrying this CFTR cDNA can correct defective Cl^- transport in cultured human CF airway epithelia. Here we use mice carrying the disrupted CF gene to test the efficacy of this transfer system in vivo. We find that even repeated high doses can only partially (50%) correct the CF defect in Cl^- transport in vivo and do not correct the Na⁺ transport defect at all. We investigated this discrepancy between the in vivo and in vitro transfer efficiency using CF mouse and human samples, and found that it reflects a difference in the susceptibility to adenovirus-5 transduction of the epithelial cell types dosed in vivo (columnar) and in vitro (basal-cell-like). These studies indicate that more efficient adenoviral gene-transfer vectors and/or refinement of dosing strategies are needed for therapy of CF lung disease.

PMID: 7523956    

Scientific Context

This section shows information related to the publication - computed using the fingerprint of the publication - including related publications, related experts with fingerprints representing significant amounts of overlap between their fingerprint and this publication. The red dots indicate whether those experts or terms appear within the publication, thereby showing potential and actual connections.