Delivery of gene editing therapeutics

Bhavesh D. Kevadiya; Farhana Islam; Pallavi Deol; Lubaba A. Zaman; Dina A. Mosselhy; Md Ashaduzzaman; Neha Bajwa; Nanda Kishore Routhu; Preet Amol Singh; Shilpa Dawre; Lalitkumar K. Vora; Sumaiya Nahid; Deepali Mathur; Mohammad Ullah Nayan; Ashish Baldi; Ramesh Kothari; Tapan A. Patel; Jitender Madan; Zahra Gounani; Jitender Bariwal; Kenneth S. Hettie; Howard E. Gendelman

doi:10.1016/j.nano.2023.102711

Delivery of gene editing therapeutics

Bhavesh D. Kevadiya^*
, Farhana Islam
, Pallavi Deol
, Lubaba A. Zaman
, Dina A. Mosselhy
, Md Ashaduzzaman
, Neha Bajwa
, Nanda Kishore Routhu
, Preet Amol Singh
, Shilpa Dawre
, Lalitkumar K. Vora
, Sumaiya Nahid
, Deepali Mathur
, Mohammad Ullah Nayan
, Ashish Baldi
, Ramesh Kothari
, Tapan A. Patel
, Jitender Madan
, Zahra Gounani
, Jitender Bariwal

Kenneth S. Hettie, Howard E. Gendelman^*

^*Corresponding author for this work

School of Pharmacy

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

5 Citations (Scopus)

Abstract

For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. Statement of significance: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.

Original language	English
Article number	102711
Journal	Nanomedicine: Nanotechnology, Biology, and Medicine
Volume	54
Early online date	12 Oct 2023
DOIs	https://doi.org/10.1016/j.nano.2023.102711
Publication status	Published - Nov 2023

Bibliographical note

Funding Information:
This work was supported by NIMH ( R01 MH121402-01A1 ), NINDS ( R01 NS034239-28 ), NIDA ( P01 DA028555-06A1 ), UNMC student assistantships and the Hirschfeld Family Foundation.

Funding Information:
The authors would like to express gratitude towards Tom Barger and Nicholas Conoan of the Electron Microscopy Core Facility (EMCF) at the University of Nebraska Medical Center for technical assistance. The EMCF is supported by state funds from the Nebraska Research Initiative and the University of Nebraska Foundation and institutionally by the Office of the Vice Chancellor for Research ; Victoria Smith and Holly Britton of the UNMC Flow Cytometry Research Facility. The UNMC Flow Cytometry Research Facility is administered through the Office of the Vice Chancellor for Research and supported by state funds from the Fred and Pamela Buffett Cancer Center's National Cancer Institute Cancer Support Grant. Some of the Figure panels were made with BioRender.com . This publication's contents are the sole responsibility of the authors and do not represent the official views of the funding agencies.

Publisher Copyright:
© 2023

Keywords

CRISPR-Cas9
Gene editing
Lipid nanoparticles
Viral vector delivery

ASJC Scopus subject areas

Bioengineering
Medicine (miscellaneous)
Molecular Medicine
Biomedical Engineering
General Materials Science
Pharmaceutical Science

UN SDGs

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

Access to Document

10.1016/j.nano.2023.102711Licence: Unspecified

Cite this

Kevadiya, B. D., Islam, F., Deol, P., Zaman, L. A., Mosselhy, D. A., Ashaduzzaman, M., Bajwa, N., Routhu, N. K., Singh, P. A., Dawre, S., Vora, L. K., Nahid, S., Mathur, D., Nayan, M. U., Baldi, A., Kothari, R., Patel, T. A., Madan, J., Gounani, Z., ... Gendelman, H. E. (2023). Delivery of gene editing therapeutics. Nanomedicine: Nanotechnology, Biology, and Medicine, 54, Article 102711. https://doi.org/10.1016/j.nano.2023.102711

@article{831155dba57c4f88a3853c67a2837075,

title = "Delivery of gene editing therapeutics",

abstract = "For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. Statement of significance: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.",

keywords = "CRISPR-Cas9, Gene editing, Lipid nanoparticles, Viral vector delivery",

author = "Kevadiya, \{Bhavesh D.\} and Farhana Islam and Pallavi Deol and Zaman, \{Lubaba A.\} and Mosselhy, \{Dina A.\} and Md Ashaduzzaman and Neha Bajwa and Routhu, \{Nanda Kishore\} and Singh, \{Preet Amol\} and Shilpa Dawre and Vora, \{Lalitkumar K.\} and Sumaiya Nahid and Deepali Mathur and Nayan, \{Mohammad Ullah\} and Ashish Baldi and Ramesh Kothari and Patel, \{Tapan A.\} and Jitender Madan and Zahra Gounani and Jitender Bariwal and Hettie, \{Kenneth S.\} and Gendelman, \{Howard E.\}",

note = "Funding Information: This work was supported by NIMH ( R01 MH121402-01A1 ), NINDS ( R01 NS034239-28 ), NIDA ( P01 DA028555-06A1 ), UNMC student assistantships and the Hirschfeld Family Foundation. Funding Information: The authors would like to express gratitude towards Tom Barger and Nicholas Conoan of the Electron Microscopy Core Facility (EMCF) at the University of Nebraska Medical Center for technical assistance. The EMCF is supported by state funds from the Nebraska Research Initiative and the University of Nebraska Foundation and institutionally by the Office of the Vice Chancellor for Research ; Victoria Smith and Holly Britton of the UNMC Flow Cytometry Research Facility. The UNMC Flow Cytometry Research Facility is administered through the Office of the Vice Chancellor for Research and supported by state funds from the Fred and Pamela Buffett Cancer Center's National Cancer Institute Cancer Support Grant. Some of the Figure panels were made with BioRender.com . This publication's contents are the sole responsibility of the authors and do not represent the official views of the funding agencies. Publisher Copyright: {\textcopyright} 2023",

year = "2023",

month = nov,

doi = "10.1016/j.nano.2023.102711",

language = "English",

volume = "54",

journal = "Nanomedicine: Nanotechnology, Biology, and Medicine",

issn = "1549-9634",

publisher = "Elsevier Inc.",

}

Kevadiya, BD, Islam, F, Deol, P, Zaman, LA, Mosselhy, DA, Ashaduzzaman, M, Bajwa, N, Routhu, NK, Singh, PA, Dawre, S, Vora, LK, Nahid, S, Mathur, D, Nayan, MU, Baldi, A, Kothari, R, Patel, TA, Madan, J, Gounani, Z, Bariwal, J, Hettie, KS & Gendelman, HE 2023, 'Delivery of gene editing therapeutics', Nanomedicine: Nanotechnology, Biology, and Medicine, vol. 54, 102711. https://doi.org/10.1016/j.nano.2023.102711

TY - JOUR

T1 - Delivery of gene editing therapeutics

AU - Kevadiya, Bhavesh D.

AU - Islam, Farhana

AU - Deol, Pallavi

AU - Zaman, Lubaba A.

AU - Mosselhy, Dina A.

AU - Ashaduzzaman, Md

AU - Bajwa, Neha

AU - Routhu, Nanda Kishore

AU - Singh, Preet Amol

AU - Dawre, Shilpa

AU - Vora, Lalitkumar K.

AU - Nahid, Sumaiya

AU - Mathur, Deepali

AU - Nayan, Mohammad Ullah

AU - Baldi, Ashish

AU - Kothari, Ramesh

AU - Patel, Tapan A.

AU - Madan, Jitender

AU - Gounani, Zahra

AU - Bariwal, Jitender

AU - Hettie, Kenneth S.

AU - Gendelman, Howard E.

N1 - Funding Information: This work was supported by NIMH ( R01 MH121402-01A1 ), NINDS ( R01 NS034239-28 ), NIDA ( P01 DA028555-06A1 ), UNMC student assistantships and the Hirschfeld Family Foundation. Funding Information: The authors would like to express gratitude towards Tom Barger and Nicholas Conoan of the Electron Microscopy Core Facility (EMCF) at the University of Nebraska Medical Center for technical assistance. The EMCF is supported by state funds from the Nebraska Research Initiative and the University of Nebraska Foundation and institutionally by the Office of the Vice Chancellor for Research ; Victoria Smith and Holly Britton of the UNMC Flow Cytometry Research Facility. The UNMC Flow Cytometry Research Facility is administered through the Office of the Vice Chancellor for Research and supported by state funds from the Fred and Pamela Buffett Cancer Center's National Cancer Institute Cancer Support Grant. Some of the Figure panels were made with BioRender.com . This publication's contents are the sole responsibility of the authors and do not represent the official views of the funding agencies. Publisher Copyright: © 2023

PY - 2023/11

Y1 - 2023/11

N2 - For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. Statement of significance: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.

AB - For the past decades, gene editing demonstrated the potential to attenuate each of the root causes of genetic, infectious, immune, cancerous, and degenerative disorders. More recently, Clustered Regularly Interspaced Short Palindromic Repeats-CRISPR-associated protein 9 (CRISPR-Cas9) editing proved effective for editing genomic, cancerous, or microbial DNA to limit disease onset or spread. However, the strategies to deliver CRISPR-Cas9 cargos and elicit protective immune responses requires safe delivery to disease targeted cells and tissues. While viral vector-based systems and viral particles demonstrate high efficiency and stable transgene expression, each are limited in their packaging capacities and secondary untoward immune responses. In contrast, the nonviral vector lipid nanoparticles were successfully used for as vaccine and therapeutic deliverables. Herein, we highlight each available gene delivery systems for treating and preventing a broad range of infectious, inflammatory, genetic, and degenerative diseases. Statement of significance: CRISPR-Cas9 gene editing for disease treatment and prevention is an emerging field that can change the outcome of many chronic debilitating disorders.

KW - CRISPR-Cas9

KW - Gene editing

KW - Lipid nanoparticles

KW - Viral vector delivery

U2 - 10.1016/j.nano.2023.102711

DO - 10.1016/j.nano.2023.102711

M3 - Review article

C2 - 37813236

AN - SCOPUS:85173608629

SN - 1549-9634

VL - 54

JO - Nanomedicine: Nanotechnology, Biology, and Medicine

JF - Nanomedicine: Nanotechnology, Biology, and Medicine

M1 - 102711

ER -

Delivery of gene editing therapeutics

Abstract

Bibliographical note

Keywords

ASJC Scopus subject areas

UN SDGs

Access to Document

Fingerprint

Cite this