CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective
DOI
10.1002/advs.202207512
Document Type
Journal Article
Publication Date
7-6-2023
Publication Title
Advanced Science
Volume
10
Issue
19
Keywords
CRISPR/Cas9, gene editing, in vivo delivery, nanomedicine
Abstract
Clustered randomly interspaced short palindromic repeats (CRISPRs) and its associated endonuclease protein, i.e., Cas9, have been discovered as an immune system in bacteria and archaea; nevertheless, they are now being adopted as mainstream biotechnological/molecular scissors that can modulate ample genetic and nongenetic diseases via insertion/deletion, epigenome editing, messenger RNA editing, CRISPR interference, etc. Many Food and Drug Administration-approved and ongoing clinical trials on CRISPR adopt ex vivo strategies, wherein the gene editing is performed ex vivo, followed by reimplantation to the patients. However, the in vivo delivery of the CRISPR components is still under preclinical surveillance. This review has summarized the nonviral nanodelivery strategies for gene editing using CRISPR/Cas9 and its recent advancements, strategic points of view, challenges, and future aspects for tissue-specific in vivo delivery of CRISPR/Cas9 components using nanomaterials.
Open Access
Gold
Repository Citation
Sahel, D., Vora, L., Saraswat, A., Sharma, S., Monpara, J., D'Souza, A., Mishra, D., Tryphena, K., Kawakita, S., Khan, S., Azhar, M., Khatri, D., Patel, K., & Singh Thakur, R. (2023). CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective. Advanced Science, 10 (19). https://doi.org/10.1002/advs.202207512