TY - JOUR AU - Quintana-Bustamante, Oscar AU - Fañanas-Baquero, Sara AU - Orman, Israel AU - Torres-Ruiz Raul, Raul AU - Duchateau, Philippe AU - Poirot, Laurent AU - Gouble, Agnès AU - Bueren, Juan A AU - Segovia, Jose Carlos PY - 2019 DO - 10.1371/journal.pone.0223775 SN - 1932-6203 UR - http://hdl.handle.net/20.500.12105/9354 AB - Pyruvate Kinase Deficiency (PKD) is a rare erythroid metabolic disease caused by mutations in the PKLR gene, which encodes the erythroid specific Pyruvate Kinase enzyme. Erythrocytes from PKD patients show an energetic imbalance and are susceptible to... LA - eng PB - BioMed Central (BMC) KW - 3' Untranslated Regions KW - 5' Untranslated Regions KW - Animals KW - Cells, Cultured KW - Gene Editing KW - HEK293 Cells KW - Hematopoietic Stem Cells KW - Humans KW - Mice KW - Pyruvate Kinase KW - Transcription Activator-Like Effector Nucleases TI - Gene editing of PKLR gene in human hematopoietic progenitors through 5' and 3' UTR modified TALEN mRNA TY - journal article ER -