Gene editing of PKLR gene in human hematopoietic progenitors through 5’ and 3’ UTR modified TALEN mRNA

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 hemolysis. Gene editing of hematopoietic stem ce...

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Detalles Bibliográficos
Autores principales: Quintana-Bustamante, Oscar, Fañanas-Baquero, Sara, Orman, Israel, Torres, Raul, Duchateau, Philippe, Poirot, Laurent, Gouble, Agnès, Bueren, Juan A., Segovia, Jose C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6795450/
https://www.ncbi.nlm.nih.gov/pubmed/31618280
http://dx.doi.org/10.1371/journal.pone.0223775

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