Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Titel:
Clinically relevant gene editing in hematopoietic stem cells for the treatment of pyruvate kinase deficiency
Auteur:
FaƱanas-Baquero, Sara Quintana-Bustamante, Oscar Dever, Daniel P. Alberquilla, Omaira Sanchez-Dominguez, Rebeca Camarena, Joab Ojeda-Perez, Isabel Dessy-Rodriguez, Mercedes Turk, Rolf Schubert, Mollie S. Lattanzi, Annalisa Xu, Liwen Lopez-Lorenzo, Jose L. Bianchi, Paola Bueren, Juan A. Behlke, Mark A. Porteus, Matthew Segovia, Jose-Carlos