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Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components
[Image: see text] Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome...
Autores principales: | , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Chemical Society
2022
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9176214/ https://www.ncbi.nlm.nih.gov/pubmed/35594500 http://dx.doi.org/10.1021/acs.molpharmaceut.1c00916 |
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author | Kazemian, Pardis Yu, Si-Yue Thomson, Sarah B. Birkenshaw, Alexandra Leavitt, Blair R. Ross, Colin J. D. |
author_facet | Kazemian, Pardis Yu, Si-Yue Thomson, Sarah B. Birkenshaw, Alexandra Leavitt, Blair R. Ross, Colin J. D. |
author_sort | Kazemian, Pardis |
collection | PubMed |
description | [Image: see text] Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome editing. Lipid nanoparticles (LNPs) have become an attractive nonviral delivery platform for CRISPR-mediated genome editing due to their low immunogenicity and application flexibility. In this review, we provide a background of CRISPR-mediated gene therapy, as well as LNPs and their applicable characteristics for delivering CRISPR components. We then highlight the challenges of CRISPR delivery, which have driven the significant development of new, safe, and optimized LNP formulations in the past decade. Finally, we discuss considerations for using LNPs to deliver CRISPR and future perspectives on clinical translation of LNP-CRISPR gene editing. |
format | Online Article Text |
id | pubmed-9176214 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2022 |
publisher | American Chemical Society |
record_format | MEDLINE/PubMed |
spelling | pubmed-91762142023-05-20 Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components Kazemian, Pardis Yu, Si-Yue Thomson, Sarah B. Birkenshaw, Alexandra Leavitt, Blair R. Ross, Colin J. D. Mol Pharm [Image: see text] Gene editing mediated by CRISPR/Cas9 systems is due to become a beneficial therapeutic option for treating genetic diseases and some cancers. However, there are challenges in delivering CRISPR components which necessitate sophisticated delivery systems for safe and effective genome editing. Lipid nanoparticles (LNPs) have become an attractive nonviral delivery platform for CRISPR-mediated genome editing due to their low immunogenicity and application flexibility. In this review, we provide a background of CRISPR-mediated gene therapy, as well as LNPs and their applicable characteristics for delivering CRISPR components. We then highlight the challenges of CRISPR delivery, which have driven the significant development of new, safe, and optimized LNP formulations in the past decade. Finally, we discuss considerations for using LNPs to deliver CRISPR and future perspectives on clinical translation of LNP-CRISPR gene editing. American Chemical Society 2022-05-20 2022-06-06 /pmc/articles/PMC9176214/ /pubmed/35594500 http://dx.doi.org/10.1021/acs.molpharmaceut.1c00916 Text en © 2022 The Authors. Published by American Chemical Society https://creativecommons.org/licenses/by-nc-nd/4.0/Permits non-commercial access and re-use, provided that author attribution and integrity are maintained; but does not permit creation of adaptations or other derivative works (https://creativecommons.org/licenses/by-nc-nd/4.0/). |
spellingShingle | Kazemian, Pardis Yu, Si-Yue Thomson, Sarah B. Birkenshaw, Alexandra Leavitt, Blair R. Ross, Colin J. D. Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components |
title | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing
Components |
title_full | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing
Components |
title_fullStr | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing
Components |
title_full_unstemmed | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing
Components |
title_short | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing
Components |
title_sort | lipid-nanoparticle-based delivery of crispr/cas9 genome-editing
components |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9176214/ https://www.ncbi.nlm.nih.gov/pubmed/35594500 http://dx.doi.org/10.1021/acs.molpharmaceut.1c00916 |
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