遗传增强
基因传递
清脆的
脾脏
基因
生物
计算生物学
DNA
医学
生物信息学
免疫学
遗传学
作者
Kelly Godbout,Jacques P. Tremblay
出处
期刊:Pharmaceutics
[MDPI AG]
日期:2022-10-07
卷期号:14 (10): 2129-2129
被引量:22
标识
DOI:10.3390/pharmaceutics14102129
摘要
Gene therapy holds great promise in the treatment of genetic diseases. It is now possible to make DNA modifications using the CRISPR system. However, a major problem remains: the delivery of these CRISPR-derived technologies to specific organs. Lipid nanoparticles (LNPs) have emerged as a very promising delivery method. However, when delivering LNPs intravenously, most of the cargo is trapped by the liver. Alternatively, injecting them directly into organs, such as the brain, requires more invasive procedures. Therefore, developing more specific LNPs is crucial for their future clinical use. Modifying the composition of the lipids in the LNPs allows more specific deliveries of the LNPs to some organs. In this review, we have identified the most effective compositions and proportions of lipids for LNPs to target specific organs, such as the brain, lungs, muscles, heart, liver, spleen, and bones.
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