Molecular BiologyAmerican Journal of Respiratory Cell and Molecular BiologyPeer-reviewed
Lentiviral-mediated gene complementation rescues pathogenic ABCA3 variants
Ashley L. Cooney, Shakayla Lamer, Ping Yang…·Paul B. McCray
This study tests lentiviral-mediated ABCA3 gene complementation in cell lines with specific pathogenic variants, showing partial rescue of cellular functions and differential effects based on variant type.
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