Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency

Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency

Pyruvate kinase deficiency (PKD), an autosomal-recessive disorder, is the main cause of chronic non-spherocytic hemolytic anemia. PKD is caused by mutations in the pyruvate kinase, liver and red blood cell (PKLR) gene, which encodes for the erythroid pyruvate kinase protein (RPK). RPK is implicated...

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Bibliographic Details
Main Authors: Sara Fañanas-Baquero, Oscar Quintana-Bustamante, Daniel P. Dever, Omaira Alberquilla, Rebeca Sanchez-Dominguez, Joab Camarena, Isabel Ojeda-Perez, Mercedes Dessy-Rodriguez, Rolf Turk, Mollie S. Schubert, Annalisa Lattanzi, Liwen Xu, Jose L. Lopez-Lorenzo, Paola Bianchi, Juan A. Bueren, Mark A. Behlke, Matthew Porteus, Jose-Carlos Segovia
Format: Article
Language:English
Published: Elsevier 2021-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hematopoietic stem cell
gene editing
hemolytic anemia
pyruvate kinase deficiency
gene therapy
CRISPR-Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000851

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http://www.sciencedirect.com/science/article/pii/S2329050121000851

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