Innovations in programmable nucleases have expanded genetic engineering capabilities, raising the possibility of a new approach to curing monogenic hematological diseases. Feasibility studies using ex vivo targeted genome-editing, and nonintegrating viral vectors show outstanding potential for correcting genetic conditions at their root cause. This article reviews the latest technological advances in the CRISPR/Cas9 system alone and combined with engineered viruses as editing tools for human hematopoietic stem and progenitor cells (HSPCs). We discuss the early phase in human trials of genome editing-based therapies for hemoglobinopathies.
Keywords: Base editing; CRISPR/Cas9; DNA repair; Engineered viruses; Gene correction; Hematological diseases; Hematopoietic stem cells; Prime editing.
Copyright © 2022 The Authors. Published by Elsevier Inc. All rights reserved.