CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective
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 modul...
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| Format: | Article |
| Language: | English |
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Wiley
2023-07-01
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| Series: | Advanced Science |
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| Online Access: | https://doi.org/10.1002/advs.202207512 |
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| author | 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 |
| author_facet | 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 |
| author_sort | Deepak Kumar Sahel |
| collection | DOAJ |
| description | 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. |
| first_indexed | 2024-03-13T01:04:53Z |
| format | Article |
| id | doaj.art-6ea600b638bb42159c25220db05a713b |
| institution | Directory Open Access Journal |
| issn | 2198-3844 |
| language | English |
| last_indexed | 2024-03-13T01:04:53Z |
| publishDate | 2023-07-01 |
| publisher | Wiley |
| record_format | Article |
| series | Advanced Science |
| spelling | doaj.art-6ea600b638bb42159c25220db05a713b2023-07-06T07:39:03ZengWileyAdvanced Science2198-38442023-07-011019n/an/a10.1002/advs.202207512CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational PerspectiveDeepak Kumar Sahel0Lalitkumar K. Vora1Aishwarya Saraswat2Saurabh Sharma3Jasmin Monpara4Anisha A. D'Souza5Deepakkumar Mishra6Kamatham Pushpa Tryphena7Satoru Kawakita8Shahid Khan9Mohd Azhar10Dharmendra Kumar Khatri11Ketan Patel12Raghu Raj Singh Thakur13Department of Pharmacy Birla Institute of Technology and Science‐Pilani BITS‐Pilani, Vidya Vihar Pilani Rajasthan 333031 IndiaSchool of Pharmacy Queen's University Belfast 97 Lisburn Road Belfast BT9 7BL UKCollege of Pharmacy & Health Sciences St. John's University Queens NY 11439 USATerasaki Institute for Biomedical Innovation Los Angeles CA 90064 USADepartment of Pharmaceutical Sciences University of Sciences Philadelphia PA 19104 USAGraduate School of Pharmaceutical Sciences and School of Pharmacy Duquesne University Pittsburgh PA 15282 USASchool of Pharmacy Queen's University Belfast 97 Lisburn Road Belfast BT9 7BL UKMolecular and Cellular Neuroscience Lab Department of Pharmacology and Toxicology National Institute of Pharmaceutical Education and Research (NIPER)‐Hyderabad Telangana 500037 IndiaDepartment of Biomedical Engineering University of California Davis CA 95616 USATerasaki Institute for Biomedical Innovation Los Angeles CA 90064 USAResearch and Development Tata Medical and Diagnostics Limited Mumbai Maharashtra 400001 IndiaMolecular and Cellular Neuroscience Lab Department of Pharmacology and Toxicology National Institute of Pharmaceutical Education and Research (NIPER)‐Hyderabad Telangana 500037 IndiaCollege of Pharmacy & Health Sciences St. John's University Queens NY 11439 USASchool of Pharmacy Queen's University Belfast 97 Lisburn Road Belfast BT9 7BL UKAbstract 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.https://doi.org/10.1002/advs.202207512CRISPR/Cas9gene editingin vivo deliverynanomedicine |
| spellingShingle | 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 CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective Advanced Science CRISPR/Cas9 gene editing in vivo delivery nanomedicine |
| title | CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective |
| title_full | CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective |
| title_fullStr | CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective |
| title_full_unstemmed | CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective |
| title_short | CRISPR/Cas9 Genome Editing for Tissue‐Specific In Vivo Targeting: Nanomaterials and Translational Perspective |
| title_sort | crispr cas9 genome editing for tissue specific in vivo targeting nanomaterials and translational perspective |
| topic | CRISPR/Cas9 gene editing in vivo delivery nanomedicine |
| url | https://doi.org/10.1002/advs.202207512 |
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