Multiplex HDR for disease and correction modeling of SCID by CRISPR genome editing in human HSPCs
Ortal Iancu, Daniel Allen, Orli Knop, Yonathan Zehavi, Dor Breier, Adaya Arbiv, Atar Lev, Yu Nee Lee, Katia Beider, Arnon Nagler, Raz Somech, Ayal Hendel
Abstract
HSPCs.
Topics & Concepts
Genome editingCRISPRProgenitor cellSevere combined immunodeficiencyBiologyCas9MultiplexGenetic enhancementHaematopoiesisGeneComputational biologyStem cellGeneticsCRISPR and Genetic EngineeringVirus-based gene therapy researchCAR-T cell therapy research