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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Publicado no:Haematologica
Main Authors: 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: Artigo
Idioma:Inglês
Publicado em: Ferrata Storti Foundation 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7109724/
https://ncbi.nlm.nih.gov/pubmed/31289207
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3324/haematol.2018.214866
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