May I cut in? Gene editing approaches in human induced pluripotent stem cells

Nicholas Brookhouser, Sreedevi Raman, Christopher Potts, David Brafman

Research output: Contribution to journalReview article

Abstract

In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods.

Original languageEnglish (US)
Article number5
JournalCells
Volume6
Issue number1
DOIs
StatePublished - Mar 1 2017

Fingerprint

Induced Pluripotent Stem Cells
Stem cells
Genes
Technology
Clustered Regularly Interspaced Short Palindromic Repeats
Cell engineering
Regenerative Medicine
Zinc Fingers
Human Development
Drug Discovery
Transcription
Cell- and Tissue-Based Therapy
Zinc
Gene Editing
Genome
Molecules

Keywords

  • CRISPR/Cas9
  • Genome editing
  • Homology-directed repair
  • Human induced pluripotent stem cells (hiPSCs)
  • TALEN
  • ZFN

ASJC Scopus subject areas

  • Biochemistry, Genetics and Molecular Biology(all)

Cite this

May I cut in? Gene editing approaches in human induced pluripotent stem cells. / Brookhouser, Nicholas; Raman, Sreedevi; Potts, Christopher; Brafman, David.

In: Cells, Vol. 6, No. 1, 5, 01.03.2017.

Research output: Contribution to journalReview article

Brookhouser, Nicholas ; Raman, Sreedevi ; Potts, Christopher ; Brafman, David. / May I cut in? Gene editing approaches in human induced pluripotent stem cells. In: Cells. 2017 ; Vol. 6, No. 1.
@article{1b093fc7e3da46939ea62589fe925370,
title = "May I cut in? Gene editing approaches in human induced pluripotent stem cells",
abstract = "In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods.",
keywords = "CRISPR/Cas9, Genome editing, Homology-directed repair, Human induced pluripotent stem cells (hiPSCs), TALEN, ZFN",
author = "Nicholas Brookhouser and Sreedevi Raman and Christopher Potts and David Brafman",
year = "2017",
month = "3",
day = "1",
doi = "10.3390/cells6010005",
language = "English (US)",
volume = "6",
journal = "Cells",
issn = "2073-4409",
publisher = "Multidisciplinary Digital Publishing Institute (MDPI)",
number = "1",

}

TY - JOUR

T1 - May I cut in? Gene editing approaches in human induced pluripotent stem cells

AU - Brookhouser, Nicholas

AU - Raman, Sreedevi

AU - Potts, Christopher

AU - Brafman, David

PY - 2017/3/1

Y1 - 2017/3/1

N2 - In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods.

AB - In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods.

KW - CRISPR/Cas9

KW - Genome editing

KW - Homology-directed repair

KW - Human induced pluripotent stem cells (hiPSCs)

KW - TALEN

KW - ZFN

UR - http://www.scopus.com/inward/record.url?scp=85050579485&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85050579485&partnerID=8YFLogxK

U2 - 10.3390/cells6010005

DO - 10.3390/cells6010005

M3 - Review article

VL - 6

JO - Cells

JF - Cells

SN - 2073-4409

IS - 1

M1 - 5

ER -