Efficient CRISPR-Cas9-based genome editing of β-globin gene on erythroid cells from homozygous β039-thalassemia patients
Lucia Carmela Cosenza, Jessica Gasparello, Nicola Romanini, Matteo Zurlo, Cristina Zuccato, Roberto Gambari, Alessia Finotti
Abstract
39 patients and for the design of combined treatments using, together with the CRISPR-Cas9 editing of the β-globin gene, other therapeutic approaches, such as, for instance, induction of HbA and/or fetal hemoglobin (HbF) using chemical inducers.
Topics & Concepts
CRISPRGenome editingCas9ThalassemiaBiologyGeneGlobinGeneticsComputational biologyCRISPR and Genetic EngineeringHemoglobinopathies and Related DisordersIron Metabolism and Disorders