1.
Hetzel M, Lopez-Rodriguez E, Mucci A, Nguyen AHH, Suzuki T, Shima K, Buchegger T, Dettmer S, Rodt T, Bankstahl JP, Malik P, Knudsen L, Schambach A, Hansen G, Trapnell BC, Lachmann N, Moritz T. Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis. Haematologica 2020; 105(4):1147-1157; https://doi.org/10.3324/haematol.2018.214866.