Editing human hematopoietic stem cells: advances and challenges

Genome editing of hematopoietic stem and progenitor cells is being developed for the treatment of several inherited disorders of the hematopoietic system. The adaptation of CRISPR-Cas9-based technologies to make precise CHANGES TO THE GENOME, and developments in altering the specificity and efficiency, and improving the delivery of nucleases to target cells have led to several breakthroughs. Many clinical trials are ongoing, and several pre-clinical models have been reported that would allow these genetic therapies to one day offer a potential cure to patients with diseases where limited options currently exist. However, there remain several challenges with respect to establishing safety, expanding accessibility and improving the manufacturing pro -cesses of these therapeutic products. This review focuses on some of the recent advances in the field of genome editing of hematopoietic stem and progenitor cells and illustrates the ongoing challenges.(c) 2022 International Society for Cell & Gene Therapy. Published by Elsevier Inc. All rights reserved.

Automated summary

Genome editing technologies, specifically CRISPR-Cas9, have made significant progress in precise genome alterations for the treatment of inherited hematopoietic disorders. Despite ongoing clinical trials and promising pre-clinical models, challenges still remain in ensuring safety, accessibility, and improving the manufacturing processes of these therapies. This review highlights recent advances and ongoing challenges in genome editing of hematopoietic stem and progenitor cells.