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Effective hematopoietic stem cell-based gene therapy in a murine model of hereditary pulmonary alveolar proteinosis
Hereditary pulmonary alveolar proteinosis due to GM-CSF receptor deficiency (herPAP) constitutes a life-threatening lung disease characterized by alveolar deposition of surfactant protein secondary to defective alveolar macrophage function. As current therapeutic options are primarily symptomatic, w...
Autores principales: | Hetzel, Miriam, Lopez-Rodriguez, Elena, Mucci, Adele, Nguyen, Ariane Hai Ha, Suzuki, Takuji, Shima, Kenjiro, Buchegger, Theresa, Dettmer, Sabine, Rodt, Thomas, Bankstahl, Jens P., Malik, Punam, Knudsen, Lars, Schambach, Axel, Hansen, Gesine, Trapnell, Bruce C., Lachmann, Nico, Moritz, Thomas |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Ferrata Storti Foundation
2020
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7109724/ https://www.ncbi.nlm.nih.gov/pubmed/31289207 http://dx.doi.org/10.3324/haematol.2018.214866 |
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