用于递送CRISPR/Cas9的基于脂质和聚合物的纳米颗粒系统
Lipid- and Polymer-Based Nanoparticle Systems for the Delivery of CRISPR/Cas9.
作者信息
Ashok Bhaargavi, Peppas Nicholas A, Wechsler Marissa E
机构信息
Department of Chemical Engineering, The University of Texas at Austin, Austin, TX, USA.
Institute for Biomaterials, Drug Delivery, and Regenerative Medicine, The University of Texas at Austin, Austin TX, USA.
出版信息
J Drug Deliv Sci Technol. 2021 Oct;65. doi: 10.1016/j.jddst.2021.102728. Epub 2021 Jul 11.
Abstract
The discovery of clustered regularly interspaced short palindromic repeat (CRISPR)/ CRISPR-associated (Cas) genome editing systems and their applications in human health and medicine has heralded a new era of biotechnology. However, the delivery of CRISPR therapeutics is arguably the most difficult barrier to overcome for translation to clinical administration. Appropriate delivery methods are required to efficiently and selectively transport all gene editing components to specific target cells and tissues of interest, while minimizing off-target effects. To overcome this challenge, we discuss and critic nanoparticle delivery strategies, focusing on the use of lipid-based and polymeric-based matrices herein.
摘要
成簇规律间隔短回文重复序列（CRISPR）/CRISPR相关蛋白（Cas）基因组编辑系统的发现及其在人类健康和医学中的应用开创了生物技术的新纪元。然而，CRISPR疗法的递送可以说是转化为临床应用时最难克服的障碍。需要合适的递送方法来高效且选择性地将所有基因编辑组件转运到特定的目标细胞和感兴趣的组织，同时将脱靶效应降至最低。为了克服这一挑战，我们讨论并评析了纳米颗粒递送策略，本文重点关注基于脂质和基于聚合物的基质的应用。
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