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Development of Inhalable Ion Channel Prosthetics for Cystic Fibrosis

Weers JG, Miller DP, Tarara TE, Lyons S, Lewandowska A, Souter C, Thornell IM, Welsh MJ, Burke MD.

Respiratory Drug Delivery 2024. Volume 1, 2024: 52-58.

Abstract:

The manuscript details critical constraints driving design of inhaled amphotericin B (AmB) formulations for use as molecular prosthetics to replace defective cystic fibrosis transmembrane conductance regulator (CFTR) ion channels for the treatment of cystic fibrosis (CF). The formulations provide ion channel activity independent of the CFTR mutation, making the formulations a potential alternative for treatment of the 10% of people with CF for whom modulators are ineffective or poorly tolerated.

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