GeneticsMolecular Therapy — Methods & Clinical DevelopmentPeer-reviewed
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…·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.
22 citations