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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...

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Autores principales: Kazemian, Pardis, Yu, Si-Yue, Thomson, Sarah B., Birkenshaw, Alexandra, Leavitt, Blair R., Ross, Colin J. D.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Chemical Society 2022
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.
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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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