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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Publicat a:Haematologica
Autors principals: 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
Format: Artigo
Idioma:Inglês
Publicat: Ferrata Storti Foundation 2020
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Article
Accés en línia: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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