Editing the Genome Ex Vivo Stem Cell Therapy

This review is focused on the recent development in the field of genomic editing of stem cells, in particular, the use of CRISPR/Cas9 systems to edit hemopoietic stem cells for beta-globinopathies and immunodeficiency syndromes. Additionally, the use of optimized genome-editing technologies in induced pluripotent stem cells will allow all affected tissue systems to be addressed. The convergence of stem cell technologies and efficient...