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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Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Cyhoeddwyd yn:Haematologica
Prif Awduron: 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
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: Ferrata Storti Foundation 2020
Pynciau:
Article
Mynediad Ar-lein: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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