Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms

Dong-Kyu Choi; Yong-Kyu Kim; Ji HoonYu; Sang-Hyun Min; Sang-Wook Park

doi:10.1016/bs.pmbts.2021.01.020

Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms

Prog Mol Biol Transl Sci. 2021:181:271-287. doi: 10.1016/bs.pmbts.2021.01.020. Epub 2021 Feb 24.

Authors

Dong-Kyu Choi¹, Yong-Kyu Kim¹, Ji HoonYu¹, Sang-Hyun Min¹, Sang-Wook Park²

Affiliations

¹ New Drug Development Center, DGMIF, Daegu, Republic of Korea.
² Department of Oral Biochemistry, School of Dentistry, Chonnam National University, Gwangju, Republic of Korea. Electronic address: swpark@jnu.ac.kr.

PMID: 34127196
DOI: 10.1016/bs.pmbts.2021.01.020

Abstract

Generation of proper models for studying human genetic diseases has been hindered until recently by the scarcity of primary cell samples from genetic disease patients and inefficient genetic modification tools. However, recent advances in clustered, regularly interspaced short palindromic repeats (CRISPR)/Cas9 technology and human induced pluripotent stem cells (hiPSCs) have provided an opportunity to explore the function of pathogenic variants and obtain gene-corrected cells for autologous cell therapy. In this chapter, we address recent applications of CRISPR/Cas9 to hiPSCs in genetic diseases, including neurodegenerative, cardiovascular, and rare diseases.

Keywords: CRISPR/Cas9; Cardiovascular diseases; Disease modeling; Genome editing; Human pluripotent stem cells; Neurodegenerative diseases; Rare diseases.

Publication types

Research Support, Non-U.S. Gov't

MeSH terms

CRISPR-Cas Systems / genetics
Gene Editing*
Humans
Induced Pluripotent Stem Cells*