Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors
<p dir="ltr">The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing system has been the focus of intense research in the last decade due to its superior ability to desirably target and edit DNA sequences. The applicability of the CRISPR-Cas system to in...
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2020
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| _version_ | 1864513512898822144 |
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| author | Karim Shalaby (18718678) |
| author2 | Mustapha Aouida (417652) Omar El-Agnaf (2134222) |
| author2_role | author author |
| author_facet | Karim Shalaby (18718678) Mustapha Aouida (417652) Omar El-Agnaf (2134222) |
| author_role | author |
| dc.creator.none.fl_str_mv | Karim Shalaby (18718678) Mustapha Aouida (417652) Omar El-Agnaf (2134222) |
| dc.date.none.fl_str_mv | 2020-10-05T03:00:00Z |
| dc.identifier.none.fl_str_mv | 10.3390/ijms21197353 |
| dc.relation.none.fl_str_mv | https://figshare.com/articles/journal_contribution/Tissue-Specific_Delivery_of_CRISPR_Therapeutics_Strategies_and_Mechanisms_of_Non-Viral_Vectors/25957939 |
| dc.rights.none.fl_str_mv | CC BY 4.0 info:eu-repo/semantics/openAccess |
| dc.subject.none.fl_str_mv | Biological sciences Genetics Biomedical and clinical sciences Immunology CRISPR-Cas gene editing gene therapy non-viral vectors cell-penetrating peptides |
| dc.title.none.fl_str_mv | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| dc.type.none.fl_str_mv | Text Journal contribution info:eu-repo/semantics/publishedVersion text contribution to journal |
| description | <p dir="ltr">The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing system has been the focus of intense research in the last decade due to its superior ability to desirably target and edit DNA sequences. The applicability of the CRISPR-Cas system to in vivo genome editing has acquired substantial credit for a future in vivo gene-based therapeutic. Challenges such as targeting the wrong tissue, undesirable genetic mutations, or immunogenic responses, need to be tackled before CRISPR-Cas systems can be translated for clinical use. Hence, there is an evident gap in the field for a strategy to enhance the specificity of delivery of CRISPR-Cas gene editing systems for in vivo applications. Current approaches using viral vectors do not address these main challenges and, therefore, strategies to develop non-viral delivery systems are being explored. Peptide-based systems represent an attractive approach to developing gene-based therapeutics due to their specificity of targeting, scale-up potential, lack of an immunogenic response and resistance to proteolysis. In this review, we discuss the most recent efforts towards novel non-viral delivery systems, focusing on strategies and mechanisms of peptide-based delivery systems, that can specifically deliver CRISPR components to different cell types for therapeutic and research purposes.</p><h2>Other Information</h2><p dir="ltr">Published in: International Journal of Molecular Sciences<br>License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.3390/ijms21197353" target="_blank">https://dx.doi.org/10.3390/ijms21197353</a></p> |
| eu_rights_str_mv | openAccess |
| id | Manara2_301f44b005dc823b0083c3060d26283c |
| identifier_str_mv | 10.3390/ijms21197353 |
| network_acronym_str | Manara2 |
| network_name_str | Manara2 |
| oai_identifier_str | oai:figshare.com:article/25957939 |
| publishDate | 2020 |
| repository.mail.fl_str_mv | |
| repository.name.fl_str_mv | |
| repository_id_str | |
| rights_invalid_str_mv | CC BY 4.0 |
| spelling | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral VectorsKarim Shalaby (18718678)Mustapha Aouida (417652)Omar El-Agnaf (2134222)Biological sciencesGeneticsBiomedical and clinical sciencesImmunologyCRISPR-Casgene editinggene therapynon-viral vectorscell-penetrating peptides<p dir="ltr">The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing system has been the focus of intense research in the last decade due to its superior ability to desirably target and edit DNA sequences. The applicability of the CRISPR-Cas system to in vivo genome editing has acquired substantial credit for a future in vivo gene-based therapeutic. Challenges such as targeting the wrong tissue, undesirable genetic mutations, or immunogenic responses, need to be tackled before CRISPR-Cas systems can be translated for clinical use. Hence, there is an evident gap in the field for a strategy to enhance the specificity of delivery of CRISPR-Cas gene editing systems for in vivo applications. Current approaches using viral vectors do not address these main challenges and, therefore, strategies to develop non-viral delivery systems are being explored. Peptide-based systems represent an attractive approach to developing gene-based therapeutics due to their specificity of targeting, scale-up potential, lack of an immunogenic response and resistance to proteolysis. In this review, we discuss the most recent efforts towards novel non-viral delivery systems, focusing on strategies and mechanisms of peptide-based delivery systems, that can specifically deliver CRISPR components to different cell types for therapeutic and research purposes.</p><h2>Other Information</h2><p dir="ltr">Published in: International Journal of Molecular Sciences<br>License: <a href="https://creativecommons.org/licenses/by/4.0/" target="_blank">https://creativecommons.org/licenses/by/4.0/</a><br>See article on publisher's website: <a href="https://dx.doi.org/10.3390/ijms21197353" target="_blank">https://dx.doi.org/10.3390/ijms21197353</a></p>2020-10-05T03:00:00ZTextJournal contributioninfo:eu-repo/semantics/publishedVersiontextcontribution to journal10.3390/ijms21197353https://figshare.com/articles/journal_contribution/Tissue-Specific_Delivery_of_CRISPR_Therapeutics_Strategies_and_Mechanisms_of_Non-Viral_Vectors/25957939CC BY 4.0info:eu-repo/semantics/openAccessoai:figshare.com:article/259579392020-10-05T03:00:00Z |
| spellingShingle | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors Karim Shalaby (18718678) Biological sciences Genetics Biomedical and clinical sciences Immunology CRISPR-Cas gene editing gene therapy non-viral vectors cell-penetrating peptides |
| status_str | publishedVersion |
| title | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| title_full | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| title_fullStr | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| title_full_unstemmed | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| title_short | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| title_sort | Tissue-Specific Delivery of CRISPR Therapeutics: Strategies and Mechanisms of Non-Viral Vectors |
| topic | Biological sciences Genetics Biomedical and clinical sciences Immunology CRISPR-Cas gene editing gene therapy non-viral vectors cell-penetrating peptides |