Generation of a High Number of Healthy Erythroid Cells from Gene-Edited Pyruvate Kinase Deficiency Patient-Specific Induced Pluripotent Stem Cells

Pyruvate kinase deficiency (PKD) is a rare erythroid metabolic disease caused by mutations in the PKLR gene. Erythrocytes from PKD patients show an energetic imbalance causing chronic non-spherocytic hemolytic anemia, as pyruvate kinase defects impair ATP production in erythrocytes. We generated PKD...

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Detalles Bibliográficos
Autores principales: Garate, Zita, Quintana-Bustamante, Oscar, Crane, Ana M., Olivier, Emmanuel, Poirot, Laurent, Galetto, Roman, Kosinski, Penelope, Hill, Collin, Kung, Charles, Agirre, Xabi, Orman, Israel, Cerrato, Laura, Alberquilla, Omaira, Rodriguez-Fornes, Fatima, Fusaki, Noemi, Garcia-Sanchez, Felix, Maia, Tabita M., Ribeiro, Maria L., Sevilla, Julian, Prosper, Felipe, Jin, Shengfang, Mountford, Joanne, Guenechea, Guillermo, Gouble, Agnes, Bueren, Juan A., Davis, Brian R., Segovia, Jose C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2015
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4682065/
https://www.ncbi.nlm.nih.gov/pubmed/26549847
http://dx.doi.org/10.1016/j.stemcr.2015.10.002

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