Korean J Intern Med.  2017 Jan;32(1):42-61. 10.3904/kjim.2016.198.

CRISPR-Cas9: a promising tool for gene editing on induced pluripotent stem cells

• Kim EJ
• Kang KH
• Ju JH

• KMID: 2365977
• DOI: http://doi.org/10.3904/kjim.2016.198

Abstract

Recent advances in genome editing with programmable nucleases have opened up new avenues for multiple applications, from basic research to clinical therapy. The ease of use of the technology"”and particularly clustered regularly interspaced short palindromic repeats (CRISPR)"”will allow us to improve our understanding of genomic variation in disease processes via cellular and animal models. Here, we highlight the progress made in correcting gene mutations in monogenic hereditary disorders and discuss various CRISPR-associated applications, such as cancer research, synthetic biology, and gene therapy using induced pluripotent stem cells. The challenges, ethical issues, and future prospects of CRISPR-based systems for human research are also discussed.

Keyword

Clustered regularly interspaced short palindromic repeats-Cas9; Clustered regularly interspaced short palindromic repeats; Gene editing; Induced pluripotent stem cells; Genetic therapy

MeSH Terms

Clustered Regularly Interspaced Short Palindromic Repeats
Ethics
Genetic Therapy
Genome
Humans
Induced Pluripotent Stem Cells*
Models, Animal
Synthetic Biology