Advanced nanoparticle strategies for optimizing RNA therapeutic delivery in neurodegenerative disorders

被引:7
作者
Naimi, Narges [1 ,2 ]
Seyedmirzaei, Homa [3 ]
Hassannejad, Zahra [2 ]
Khaboushan, Alireza Soltani [2 ,4 ,5 ]
机构
[1] Univ Tehran Med Sci, Fac Pharm, Dept Pharmaceut, Tehran, Iran
[2] Univ Tehran Med Sci, Childrens Med Ctr, Cell & Tissue Res Inst, Pediat Urol & Regenerat Med Res Ctr, Tehran, Iran
[3] Univ Tehran Med Sci, Sports Med Res Ctr, Neurosci Inst, Tehran, Iran
[4] Univ Tehran Med Sci, Students Scientif Res Ctr, Tehran, Iran
[5] Univ Tehran Med Sci, Sch Med, Tehran, Iran
关键词
RNA delivery; Nanoparticles; Gene therapy; Tissue engineering; Neurodegenerative disease; Blood -brain barrier; AMYLOID PRECURSOR PROTEIN; ACCELERATED BLOOD CLEARANCE; SOLID LIPID NANOPARTICLES; ALPHA-SYNUCLEIN; PARKINSONS-DISEASE; ALZHEIMERS-DISEASE; DRUG-DELIVERY; REPEATED INJECTION; ENDOSOMAL ESCAPE; BRAIN DELIVERY;
D O I
10.1016/j.biopha.2024.116691
中图分类号
R-3 [医学研究方法]; R3 [基础医学];
学科分类号
1001 ;
摘要
Neurodegenerative diseases affect many people worldwide, and as the population ages, the incidence of these conditions increases. Alzheimer's disease (AD) and Parkinson's disease (PD) are the most prevalent neurodegenerative disorders worldwide. Different medicines are being used to control symptoms related to these conditions, but no treatment has yet been approved. Both genetic and environmental factors are involved in disease pathogenesis, and research on the pathophysiological pathways is still ongoing. The role of subcellular pathways and dysregulation in RNA pathways has been highlighted in pathophysiological studies, and treatment strategies focused on these pathways can be a promising approach. Many experiments have been conducted on delivering RNA cargo to the CNS to modulate various pathways involved. Yet another challenge to be faced is the effective transport of desired molecules to targets, which can be greatly hindered by distinct barriers limiting transport to the CNS, most noticeably the blood-brain barrier (BBB). Nanotechnology and the use of different nano-carriers for the delivery of nucleotides, peptides, proteins, and drug molecules are currently of great interest as these carriers help with better delivery and protection and, as a result, improve the effectiveness of the cargo. Nanocarriers can protect susceptible RNA molecules from possible degradation or destruction and improve their ability to reach the brain by enhancing BBB penetration. Different mechanisms for this process have been hypothesized. This review will go through the therapeutic application of RNA molecules in the treatment of AD and PD and the role of nanocarriers in overcoming delivery challenges and enhancing efficacy.
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页数:12
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