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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Bibliographic Details
Main Author: Garate, Z (author)
Other Authors: Quintana-Bustamante, O (author), Crane, AM (author), Olivier, E (author), Poirot, L (author), Galetto, R (author), Kosinski, P (author), Hill, C (author), Kung, C (author), Agirre, X (author), Orman, I (author), Cerrato, L (author), Alberquilla, O (author), Rodriguez-Fornes, F (author), Fusaki, N (author), Garcia-Sanchez, F (author), Maia, TM (author), Ribeiro, ML (author), Sevilla, J (author), Prosper, F (author), Jin, S (author), Mountford, J (author), Guenechea, G (author), Gouble, A (author), Bueren, JA (author), Davis, BR (author), Segovia, JC (author)
Format: article
Language:eng
Published: 2017
Subjects:
Anemia Hemolítica Congénita não Esferocítica
Células Eritroides
Células-Tronco Pluripotentes Induzidas
Piruvato Quinase
Erros Inatos do Metabolismo dos Piruvatos
Online Access:http://hdl.handle.net/10400.4/2083
Country:Portugal
Oai:oai:rihuc.huc.min-saude.pt:10400.4/2083

Internet

http://hdl.handle.net/10400.4/2083

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