CRISPR/Cas9 Genome Editing for Tissue-Specific In Vivo Targeting: Nanomaterials and Translational Perspective.

Autor: Sahel DK; Department of Pharmacy, Birla Institute of Technology and Science-Pilani, BITS-Pilani, Vidya Vihar, Pilani, Rajasthan, 333031, India., Vora LK; School of Pharmacy, Queen's University Belfast, 97 Lisburn Road, Belfast, BT9 7BL, UK., Saraswat A; College of Pharmacy & Health Sciences, St. John's University, Queens, NY, 11439, USA., Sharma S; Terasaki Institute for Biomedical Innovation, Los Angeles, CA, 90064, USA., Monpara J; Department of Pharmaceutical Sciences, University of Sciences, Philadelphia, PA, 19104, USA., D'Souza AA; Graduate School of Pharmaceutical Sciences and School of Pharmacy, Duquesne University, Pittsburgh, PA, 15282, USA., Mishra D; School of Pharmacy, Queen's University Belfast, 97 Lisburn Road, Belfast, BT9 7BL, UK., Tryphena KP; Molecular and Cellular Neuroscience Lab, Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana, 500037, India., Kawakita S; Department of Biomedical Engineering, University of California, Davis, CA, 95616, USA., Khan S; Terasaki Institute for Biomedical Innovation, Los Angeles, CA, 90064, USA., Azhar M; Research and Development Tata Medical and Diagnostics Limited, Mumbai, Maharashtra, 400001, India., Khatri DK; Molecular and Cellular Neuroscience Lab, Department of Pharmacology and Toxicology, National Institute of Pharmaceutical Education and Research (NIPER)-Hyderabad, Telangana, 500037, India., Patel K; College of Pharmacy & Health Sciences, St. John's University, Queens, NY, 11439, USA., Singh Thakur RR; School of Pharmacy, Queen's University Belfast, 97 Lisburn Road, Belfast, BT9 7BL, UK.
Jazyk: angličtina
Zdroj: Advanced science (Weinheim, Baden-Wurttemberg, Germany) [Adv Sci (Weinh)] 2023 Jul; Vol. 10 (19), pp. e2207512. Date of Electronic Publication: 2023 May 11.
DOI: 10.1002/advs.202207512
Abstrakt: 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.
(© 2023 The Authors. Advanced Science published by Wiley-VCH GmbH.)
Databáze: MEDLINE
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