Background: Induced pluripotent stem cells hold tremendous potential for biological and
therapeutic applications. The development of efficient technologies for targeted genome alteration of
stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. The revolutionary
technology for genome editing known as the clustered regularly interspaced short palindromic
repeat (CRISPR)-associated protein 9 (Cas9) system is recently recognized as a powerful tool for editing
DNA at specific loci.
Objective: The ease of use of the CRISPR-Cas9 technology will allow us to improve our understanding
of genomic variation in disease processes via cellular and animal models. More recently, this system
was modified to repress (CRISPR interference, CRISPRi) or activate (CRISPR activation, CRISPRa)
gene expression without alterations in the DNA, which amplified the scope of applications of CRISPR
systems for stem cell biology.
Results and Conclusion: Here, we highlight latest advances of CRISPR-associated applications in
human pluripotent stem cells. The challenges and future prospects of CRISPR-based systems for human
research are also discussed.