Cell culture platforms to study ABCA-3 deficiency in vitro

Dysfunction of type II alveolar cells due to deficiency in the lipid transporter protein, ATP-binding cassette subfamily A member3 (ABCA-3), can cause lung disease in children. The development of therapeutics to treat ABCA-3 deficiency has been hindered by the lack of appropriate cell culture models. This thesis investigates two alternative cells culture platforms, primary nasal epithelial cells and induced pluripotent stem cell-derived alveolospheres, to determine their biological relevance as type II alveolar cell surrogates. While neither of the cell culture platforms investigated perfectly recapitulate type II alveolar cell phenotype, further optimisation may generate more usable platforms in future.