Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

Brittany E. Givens; Youssef W. Naguib; Sean M. Geary; Eric J. Devor; Aliasger K. Salem

doi:10.1208/s12248-018-0267-9

Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

Brittany E. Givens, Youssef W. Naguib, Sean M. Geary, Eric J. Devor, Aliasger K. Salem

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

73 Scopus citations

Abstract

The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

Original language	English
Article number	108
Journal	AAPS Journal
Volume	20
Issue number	6
DOIs	https://doi.org/10.1208/s12248-018-0267-9
State	Published - Nov 1 2018

Bibliographical note

Publisher Copyright:
© 2018, American Association of Pharmaceutical Scientists.

Keywords

CRISPR/Cas9
gene delivery
gene editing
nanoparticle

ASJC Scopus subject areas

Pharmaceutical Science

Access to Document

10.1208/s12248-018-0267-9

Cite this

@article{71e0287f8d4847eb9cf1cd093849f295,

title = "Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics",

abstract = "The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.",

keywords = "CRISPR/Cas9, gene delivery, gene editing, nanoparticle",

author = "Givens, {Brittany E.} and Naguib, {Youssef W.} and Geary, {Sean M.} and Devor, {Eric J.} and Salem, {Aliasger K.}",

note = "Publisher Copyright: {\textcopyright} 2018, American Association of Pharmaceutical Scientists.",

year = "2018",

month = nov,

day = "1",

doi = "10.1208/s12248-018-0267-9",

language = "English",

volume = "20",

number = "6",

}

TY - JOUR

T1 - Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

AU - Givens, Brittany E.

AU - Naguib, Youssef W.

AU - Geary, Sean M.

AU - Devor, Eric J.

AU - Salem, Aliasger K.

PY - 2018/11/1

Y1 - 2018/11/1

N2 - The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

AB - The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

KW - CRISPR/Cas9

KW - gene delivery

KW - gene editing

KW - nanoparticle

UR - http://www.scopus.com/inward/record.url?scp=85054645390&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=85054645390&partnerID=8YFLogxK

U2 - 10.1208/s12248-018-0267-9

DO - 10.1208/s12248-018-0267-9

M3 - Review article

C2 - 30306365

AN - SCOPUS:85054645390

SN - 1550-7416

VL - 20

JO - AAPS Journal

JF - AAPS Journal

IS - 6

M1 - 108

ER -

Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

Access to Document

Other files and links

Fingerprint

Cite this