清脆的
基因组编辑
Cas9
计算生物学
基因传递
纳米技术
从长凳到床边
计算机科学
遗传增强
生物
基因
材料科学
医学
遗传学
医学物理学
作者
Zoya Iqbal,Khurrum Rehman,Jiang Xia,Maryam Shabbir,Muhammad Zaman,Yujie Liang,Li Duan
出处
期刊:Biomaterials Science
[The Royal Society of Chemistry]
日期:2023-01-01
卷期号:11 (11): 3762-3783
被引量:10
摘要
RISPR-Cas9 has exhibited enormous potential in gene therapy. It can perform genome editing with single-nucleotide precision in various types of cell and tissue, providing a powerful breakthrough technology for genome editing in therapeutic development. But the limited delivery methods pose substantial challenges pertinent to safe and effective CRISPR/Cas9 delivery, thus hindering its application. These challenges should be tackled to develop next-generation genetic therapies. Biomaterial-based drug delivery systems can overcome these issues, for example using biomaterials as carriers for CRISPR/Cas9 targeted delivery, and conditional control of its function can improve precision, furnish on-demand and transient gene editing and reduce adverse consequences such as off-target events and immunogenicity, representing a promising direction for modern precision medicine. This review describes the application status and research progress of current CRISPR/Cas9 delivery approaches, including polymeric nanoparticles, liposomes, extracellular vesicles, inorganic nanoparticles and hydrogels. The unique properties of light-controlled and small-molecule drugs for spatially and temporally controlled genome editing are also illustrated. In addition, targetable delivery vehicles for the active delivery of CRISPR systems are also discussed. The perspectives to overcome the current limitations in the CRISPR/Cas9 delivery and their bench-to-bedside translation are also highlighted.
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