| Autor: |
Zhen Liu, Zhenghua Li, Bin Li |
| Jazyk: |
angličtina |
| Rok vydání: |
2022 |
| Předmět: |
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| Zdroj: |
Advanced NanoBiomed Research, Vol 2, Iss 11, Pp n/a-n/a (2022) |
| Druh dokumentu: |
article |
| ISSN: |
2699-9307 |
| DOI: |
10.1002/anbr.202200082 |
| Popis: |
The clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR‐associated (Cas) genome editing system provides a powerful toolbox for genetic engineering. However, safe and efficient in vivo delivery of all CRISPR components into the nuclei of target organs remains challenging toward CRISPR/Cas clinical translation. With the rapid development of nanotechnology, the nonviral CRISPR/Cas mRNA delivery systems are emerging as one of the most clinically advanced platforms. CRISPR/Cas mRNA formulated in lipid nanoparticles is currently under clinical trials for treating hereditary transthyretin amyloidosis. Nonviral delivery of CRISPR/Cas systems in mRNA format not only minimizes immunogenicity and carcinogenicity, but also enables transient genome editing, thereby reducing potential off‐target effects. The recent advances in the nonviral CRISPR/Cas mRNA delivery systems should facilitate CRISPR technologies toward clinical applications. |
| Databáze: |
Directory of Open Access Journals |
| Externí odkaz: |
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