核酸纳米材料在线粒体功能障碍治疗中的应用
Lan Yao1, Qing Hai2, Tao Zhang1
1State Key Laboratory of Oral Diseases, National Clinical Research Center for Oral Diseases, West China School of Stomatology, Sichuan University, Chengdu, China.
Current drug metabolism
|June 15, 2023
概括
框架核酸为线粒体基因治疗提供了一种新的,无毒的输送系统. 这些结构能够精确地准线粒体,潜在地治疗神经退行等疾病.
科学领域:
- 生物医学工程 生物医学工程
- 分子生物学分子生物学
- 线粒体医学 线粒体医学
背景情况:
- 线粒体功能障碍与癌症,代谢障碍和神经退行等疾病有关.
- 传统的治疗方法面临着副作用和治疗效率的挑战.
- 线粒体基因疗法通过调节基因表达提供了一个有希望的替代方案.
研究的目的:
- 探索框架核酸 (FNA) 作为线粒体基因治疗的先进输送载体.
- 突出FNAs相对于传统的输送方法 (如脂质体) 的优势.
- 讨论FNA在治疗线粒体功能障碍,特别是神经退行症方面的潜力.
主要方法:
- 利用FNAs独特的结构特性,在没有转染试剂的情况下进入细胞.
- 利用FNAs的核酸性质进行结构编辑和药物加载.
- 设计具有可控制尺寸的FNA,以提高生物屏障的透率,包括血脑屏障.
主要成果:
- FNAs显示出有效和有针对性的传递到线粒体的潜力.
- 它们的生物相容性和稳定性支持体内应用.
- 可控尺寸允许穿越生物障碍,使中枢神经系统能够针对神经退行.
结论:
- 框架核酸代表了线粒体基因治疗的有希望的,生物相容的,高效的输送系统.
- FNA在精确准,降低细胞毒性和增强跨越生物障碍的传递方面具有优势.
- 对基于FNA的系统的进一步研究具有治疗线粒体功能障碍和相关疾病的巨大潜力.
相关概念视频
Electron Transport Chain: Complex I and II
14.6K
The mitochondrial electron transport chain (ETC) is the main energy generation system in the eukaryotic cells. However, mitochondria also produce cytotoxic reactive oxygen species (ROS) due to the large electron flow during oxidative phosphorylation. While Complex I is one of the primary sources of superoxide radicals, ROS production by Complex II is uncommon and may only be observed in cancer cells with mutated complexes.
ROS generation is regulated and maintained at moderate levels necessary...
ROS generation is regulated and maintained at moderate levels necessary...
14.6K
Mitochondrial Membranes
11.6K
A single mitochondrion is a bean-shaped organelle enclosed by a double-membrane system. The outer membrane of mitochondria is smooth and contains many porins - the integral membrane transporters. Porins enable free diffusion of ions and small uncharged molecules through the outer mitochondrial membrane but limit the transport of molecules larger than 5000 Daltons. Further, the outer mitochondrial membrane forms a unique structure called membrane contact sites with other subcellular organelles,...
11.6K
Animal Mitochondrial Genetics
7.7K
Among all the organelles in an animal cell, only mitochondria have their own independent genomes. Animal mitochondrial DNA is a double-stranded, closed-circular molecule with around 20,000 base pairs. Mitochondrial DNA is unique in that one of its two strands, the heavy, or H, -strand is guanine rich, whereas the complementary strand is cytosine rich and called the light, or L, -strand. Compared to nuclear DNA, mitochondrial DNA has a very low percentage of non-coding regions and is marked by...
7.7K
Mitochondria
14.0K
Mitochondria are eukaryotic cellular organelles that are known to produce energy through a process called oxidative phosphorylation. Besides their primary function, mitochondria are involved in various cellular processes, including cell growth, differentiation, signaling, metabolism, and senescence. Age-related changes cause a decline in mitochondrial quality and integrity due to increased mitochondrial mutations and oxidative damage. Thus, aging can severely impact mitochondrial functions,...
14.0K


