清脆的
基因组编辑
纳米载体
计算生物学
基因组
Cas9
纳米技术
生物
微泡
计算机科学
药物输送
基因
材料科学
遗传学
小RNA
作者
Elizabeth Maria Clarissa,Mamata Karmacharya,Hyun‐Min Choi,Sumit Kumar,Yoon‐Kyoung Cho
出处
期刊:Small
[Wiley]
日期:2025-02-03
标识
DOI:10.1002/smll.202409353
摘要
Abstract The advent of Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)‐based genome editing technologies has opened up groundbreaking possibilities for treating a wide spectrum of genetic disorders and diseases. However, the success of these technologies relies heavily on the development of efficient and safe delivery systems. Among the most promising approaches are natural and synthetic nanocarrier‐mediated delivery systems, including viral vectors, extracellular vesicles (EVs), engineered cellular membrane particles, liposomes, and various nanoparticles. These carriers enhance the efficacy of the CRISPR system by providing a unique combination of efficiency, specificity, and reduced immunogenicity. Synthetic carriers such as liposomes and nanoparticles facilitate CRISPR delivery with high reproducibility and customizable functions. Viral vectors, renowned for their high transduction efficiency and broad tropism, serve as powerful vehicles for delivering CRISPR components to various cell types. EVs, as natural carriers of RNA and proteins, offer a stealth mechanism to evade immune detection, allowing for the targeted delivery of genome editors with minimal off‐target effects. Engineered cellular membrane particles further improve delivery by simulating the cellular environment, enhancing uptake, and minimizing immune response. This review explores the innovative integration of CRISPR genome editors with various nanocarrier systems, focusing on recent advancements, applications, and future directions in therapeutic genome editing.
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