Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
Sophie Ramadier, Anne Chalumeau, Tristan Félix, Nadia Othman, Shérazade Aknoun, Antonio Casini, Giulia Maule, Cécile Masson, Anne De Cian, Giacomo Frati, Mégane Brusson, Jean‐Paul Concordet, Marina Cavazzana, Anna Cereseto, Wassim El Nemer, Mario Amendola, Benoît Wattellier, Vasco Meneghini, Annarita Miccio
Topics & Concepts
Viral vectorGenetic enhancementCRISPRFetal hemoglobinGenome editingHaematopoiesisBiologyGlobinCas9Progenitor cellGene deliveryTransfectionGeneStem cellMolecular biologyCell biologyGeneticsRecombinant DNAFetusPregnancyCRISPR and Genetic EngineeringHemoglobinopathies and Related DisordersRNA regulation and disease