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

50 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

Funding Information:
B. E. G. acknowledges fellowship support from the Alfred P. Sloan Foundation, the University of Iowa Graduate College, and the National GEM Consortium. E. J. Devor acknowledges support from the University of Iowa Department of Obstetrics and Gynecology Research Development Fund. A.K.S. acknowledges support from the National Cancer Institute at the National Institutes of Health (5P30CA086862) and the Lyle and Sharon Bighley Chair of Pharmaceutical Sciences.

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 = "Funding Information: B. E. G. acknowledges fellowship support from the Alfred P. Sloan Foundation, the University of Iowa Graduate College, and the National GEM Consortium. E. J. Devor acknowledges support from the University of Iowa Department of Obstetrics and Gynecology Research Development Fund. A.K.S. acknowledges support from the National Cancer Institute at the National Institutes of Health (5P30CA086862) and the Lyle and Sharon Bighley Chair of Pharmaceutical Sciences. 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.

N1 - Funding Information: B. E. G. acknowledges fellowship support from the Alfred P. Sloan Foundation, the University of Iowa Graduate College, and the National GEM Consortium. E. J. Devor acknowledges support from the University of Iowa Department of Obstetrics and Gynecology Research Development Fund. A.K.S. acknowledges support from the National Cancer Institute at the National Institutes of Health (5P30CA086862) and the Lyle and Sharon Bighley Chair of Pharmaceutical Sciences. Publisher Copyright: © 2018, American Association of Pharmaceutical Scientists.

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

VL - 20

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