The Application of CRISPR/Cas9 Technologies and Therapies in Stem Cells

Abstract

The ability to precisely modify genomic sequences has always been a powerful tool for determining the relationship between genotype and phenotype, and for targeted editing of disease-causing genetic lesions. Until recently, it has been difficult to routinely achieve this goal. The advent of CRISPR/Cas9 has resulted in the widespread adoption of genome editing as a standard laboratory procedure and has accelerated the options available for researchers and clinicians working on accurate in vitro cell models of disease, or for the development of future ex vivo and in vivo gene therapies. In this review, we provide an update on CRISPR/Cas9 technologies and delve into how this tool can be used for stem cell engineering, focusing on the most pressing and feasible applications. Lastly, we briefly discuss the ethical concerns that have caused worldwide alarm in the stem cell field. Despite the technical challenges that still lie ahead, CRISPR-based genome editing offers unprecedented opportunities for advancing stem cell therapies to the clinic.