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

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

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

1 Scopus citations

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.

Original language	English
Title of host publication	Reprogramming the Genome
Subtitle of host publication	CRISPR-Cas-based Human Disease Therapy
Editors	Vijai Singh
Publisher	Elsevier B.V.
Pages	271-287
Number of pages	17
ISBN (Print)	9780323853231
DOIs	https://doi.org/10.1016/bs.pmbts.2021.01.020
State	Published - Jan 2021

Publication series

Name	Progress in Molecular Biology and Translational Science
Volume	181
ISSN (Print)	1877-1173
ISSN (Electronic)	1878-0814

UN SDGs

This output contributes to the following UN Sustainable Development Goals (SDGs)

SDG 3 Good Health and Well-being

Keywords

CRISPR/Cas9
Cardiovascular diseases
Disease modeling
Genome editing
Human pluripotent stem cells
Neurodegenerative diseases
Rare diseases

Access to Document

10.1016/bs.pmbts.2021.01.020

Cite this

Choi, D. K., Kim, Y. K., HoonYu, J., Min, S. H., & Park, S. W. (2021). Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms. In V. Singh (Ed.), Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy (pp. 271-287). (Progress in Molecular Biology and Translational Science; Vol. 181). Elsevier B.V.. https://doi.org/10.1016/bs.pmbts.2021.01.020

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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.",

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author = "Choi, \{Dong Kyu\} and Kim, \{Yong Kyu\} and Ji HoonYu and Min, \{Sang Hyun\} and Park, \{Sang Wook\}",

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year = "2021",

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doi = "10.1016/bs.pmbts.2021.01.020",

language = "English",

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Choi, DK, Kim, YK, HoonYu, J, Min, SH & Park, SW 2021, Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms. in V Singh (ed.), Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy. Progress in Molecular Biology and Translational Science, vol. 181, Elsevier B.V., pp. 271-287. https://doi.org/10.1016/bs.pmbts.2021.01.020

Genome editing of hPSCs: Recent progress in hPSC-based disease modeling for understanding disease mechanisms. / Choi, Dong Kyu; Kim, Yong Kyu; HoonYu, Ji et al.
Reprogramming the Genome: CRISPR-Cas-based Human Disease Therapy. ed. / Vijai Singh. Elsevier B.V., 2021. p. 271-287 (Progress in Molecular Biology and Translational Science; Vol. 181).

Research output: Chapter in Book/Report/Conference proceeding › Chapter › peer-review

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T1 - Genome editing of hPSCs

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AU - Choi, Dong Kyu

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AU - Min, Sang Hyun

AU - Park, Sang Wook

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AB - 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.

KW - CRISPR/Cas9

KW - Cardiovascular diseases

KW - Disease modeling

KW - Genome editing

KW - Human pluripotent stem cells

KW - Neurodegenerative diseases

KW - Rare diseases

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DO - 10.1016/bs.pmbts.2021.01.020

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SN - 9780323853231

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BT - Reprogramming the Genome

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PB - Elsevier B.V.

ER -

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

Abstract

Publication series

UN SDGs

Keywords

Access to Document

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