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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...

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Detalles Bibliográficos
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
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Ferrata Storti Foundation 2020
Materias:	Article
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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