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TALEN-mediated functional correction of human iPSC-derived macrophages in context of hereditary pulmonary alveolar proteinosis
Hereditary pulmonary alveolar proteinosis (herPAP) constitutes a rare, life threatening lung disease characterized by the inability of alveolar macrophages to clear the alveolar airspaces from surfactant phospholipids. On a molecular level, the disorder is defined by a defect in the CSF2RA gene codi...
Autores principales: | Kuhn, Alexandra, Ackermann, Mania, Mussolino, Claudio, Cathomen, Toni, Lachmann, Nico, Moritz, Thomas |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5680188/ https://www.ncbi.nlm.nih.gov/pubmed/29123113 http://dx.doi.org/10.1038/s41598-017-14566-8 |
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