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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...

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Detalles Bibliográficos
Autores principales: Kuhn, Alexandra, Ackermann, Mania, Mussolino, Claudio, Cathomen, Toni, Lachmann, Nico, Moritz, Thomas
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2017
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