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 - 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
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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 -