Non-Viral Delivery To Enable Genome Editing

Yuan Rui; David R. Wilson; Jordan J. Green

doi:10.1016/j.tibtech.2018.08.010

Non-Viral Delivery To Enable Genome Editing

Yuan Rui, David R. Wilson, Jordan J. Green

School of Medicine

Research output: Contribution to journal › Review article › peer-review

37 Scopus citations

Abstract

Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.

Original language	English (US)
Pages (from-to)	281-293
Number of pages	13
Journal	Trends in Biotechnology
Volume	37
Issue number	3
DOIs	https://doi.org/10.1016/j.tibtech.2018.08.010
State	Published - Mar 2019

Keywords

CRISPR/Cas
TALEN
ZFN
gene editing
non-viral

ASJC Scopus subject areas

Biotechnology
Bioengineering

Access to Document

10.1016/j.tibtech.2018.08.010

Cite this

@article{70bc00d8788d4e68b22bea47d12f95bf,

title = "Non-Viral Delivery To Enable Genome Editing",

abstract = "Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.",

keywords = "CRISPR/Cas, TALEN, ZFN, gene editing, non-viral",

author = "Yuan Rui and Wilson, {David R.} and Green, {Jordan J.}",

note = "Publisher Copyright: {\textcopyright} 2018 Elsevier Ltd",

year = "2019",

month = mar,

doi = "10.1016/j.tibtech.2018.08.010",

language = "English (US)",

volume = "37",

pages = "281--293",

journal = "Trends in Biotechnology",

issn = "0167-7799",

publisher = "Elsevier Limited",

number = "3",

}

TY - JOUR

T1 - Non-Viral Delivery To Enable Genome Editing

AU - Rui, Yuan

AU - Wilson, David R.

AU - Green, Jordan J.

PY - 2019/3

Y1 - 2019/3

N2 - Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.

AB - Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.

KW - CRISPR/Cas

KW - TALEN

KW - ZFN

KW - gene editing

KW - non-viral

UR - http://www.scopus.com/inward/record.url?scp=85054058084&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85054058084&partnerID=8YFLogxK

U2 - 10.1016/j.tibtech.2018.08.010

DO - 10.1016/j.tibtech.2018.08.010

M3 - Review article

C2 - 30278987

AN - SCOPUS:85054058084

SN - 0167-7799

VL - 37

SP - 281

EP - 293

JO - Trends in Biotechnology

JF - Trends in Biotechnology

IS - 3

ER -

Non-Viral Delivery To Enable Genome Editing

Abstract

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this