Lentiviral-mediated gene complementation rescues pathogenic ABCA3 variants
Ashley L. Cooney, Shakayla Lamer, Ping Yang, Daniel J. Wegner, Frances V. White, F. Sessions Cole, Christine Wohlford-Lenane, Erin Hennessey, Pushpinder Bawa, Darrell N. Kotton, Patrick L. Sinn, Jennifer Wambach, 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.
American Journal of Respiratory Cell and Molecular Biology · Mar 4, 2026Peer-reviewedMolecular Biology