Clinically Relevant Gene Editing in Hematopoietic Stem Cells for the Treatment of Pyruvate Kinase Deficiency Hemolytic Anemia
Sara Fañanas-Baquero, Oscar Quintana Bustamante, Daniel P. Dever, Omaira Alberquilla, Rebeca Sánchez‐Domínguez, Joab Camarena, Isabel Ojeda-Pérez, Mercedes Dessy-Rodríguez, Rolf Turk, Mollie S. Schubert, Annalisa Lattanzi, Liwen Xu, José Luis López Lorenzo, Paola Ilaria Bianchi, Juan Antonio Bueren, , Jose Carlos Segovia
This study uses gene editing in hematopoietic stem cells to treat Pyruvate Kinase Deficiency Hemolytic Anemia, restoring RPK function and ATP levels in erythroid cells.
Molecular Therapy — Methods & Clinical Development · May 13, 2021Peer-reviewedGenetics