Gene therapy-emulating small molecule treatments in cystic fibrosis airway epithelial cells and patients.

ABSTRACT:

Background

Several small molecule corrector and potentiator drugs have recently been licensed for Cystic Fibrosis (CF) therapy. However, other aspects of the disease, especially inflammation, are less effectively treated by these drugs. We hypothesized that small molecule drugs could function either alone or as an adjuvant to licensed therapies to treat these aspects of the disease, perhaps emulating the effects of gene therapy in CF cells. The cardiac glycoside digitoxin, which has been shown to inhibit TNFα/NFκB signaling in CF lung epithelial cells, may serve as such a therapy.

Methods

IB3-1 CF lung epithelial cells were treated with different Vertex (VX) drugs, digitoxin, and various drug mixtures, and ELISA assays were used to assess suppression of baseline and TNFα-act

SUBMITTER: Yang Q 

PROVIDER: S-EPMC6925517 | biostudies-literature | 2019 Dec

REPOSITORIES: biostudies-literature