CRISPR/Cas9 genome editing for tissue‐specific in vivo targeting: nanomaterials and translational perspective

Deepak Kumar Sahel, Lalitkumar K. Vora*, Aishwarya Saraswat, Saurabh Sharma, Jasmin Monpara, Anisha A. D'Souza, Deepakkumar Mishra, Kamatham Pushpa Tryphena, Satoru Kawakita, Shahid Khan, Mohd Azhar, Dharmendra Kumar Khatri*, Ketan Patel, Raghu Raj Singh Thakur

*Corresponding author for this work

Research output: Contribution to journalReview articlepeer-review

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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.

Original languageEnglish
Article number2207512
Number of pages38
JournalAdvanced Science
Volume10
Issue number19
Early online date11 May 2023
DOIs
Publication statusPublished - 11 May 2023

Keywords

  • Review
  • Reviews
  • CRISPR/Cas9
  • gene editing
  • in vivo delivery
  • nanomedicine
  • Biochemistry, Genetics and Molecular Biology (miscellaneous)
  • Medicine (miscellaneous)
  • General Chemical Engineering
  • General Materials Science
  • General Engineering
  • General Physics and Astronomy

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