Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components
| العنوان: | Lipid-Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Components |
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| المؤلفون: | Pardis Kazemian, Si-Yue Yu, Sarah B. Thomson, Alexandra Birkenshaw, Blair R. Leavitt, Colin J. D. Ross |
| المصدر: | Molecular Pharmaceutics. 19:1669-1686 |
| بيانات النشر: | American Chemical Society (ACS), 2022. |
| سنة النشر: | 2022 |
| مصطلحات موضوعية: | Gene Editing, Liposomes, Drug Discovery, Gene Transfer Techniques, Nanoparticles, Pharmaceutical Science, Molecular Medicine, CRISPR-Cas Systems, Lipids |
| الوصف: | 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. |
| تدمد: | 1543-8392 1543-8384 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::831d33a4edf06031d01bdea358e3975a https://doi.org/10.1021/acs.molpharmaceut.1c00916 |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....831d33a4edf06031d01bdea358e3975a |
| قاعدة البيانات: | OpenAIRE |
Find this issue from the American Chemical Society | تدمد: | 15438392 15438384 |
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