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Advanced nanoparticle strategies for optimizing RNA therapeutic delivery in neurodegenerative disorders - 29/05/24

Doi : 10.1016/j.biopha.2024.116691 
Narges Naimi a, b, Homa Seyedmirzaei c, Zahra Hassannejad b, , Alireza Soltani Khaboushan b, d, e,
a Departement of Pharmaceutics, Faculty of Pharmacy, Tehran University of Medical Sciences, Tehran, Iran 
b Pediatric Urology and Regenerative Medicine Research Center, Gene, Cell and Tissue Research Institute, Children’s Medical Center, Tehran University of Medical Science, Tehran, Iran 
c Sports Medicine Research Center, Neuroscience Institute, Tehran University of Medical Sciences, Tehran, Iran 
d Students’ Scientific Research Center, Tehran University of Medical Sciences, Tehran, Iran 
e School of Medicine, Tehran University of Medical Sciences, Tehran, Iran 

Corresponding author.⁎⁎Corresponding author at: Pediatric Urology and Regenerative Medicine Research Center, Gene, Cell and Tissue Research Institute, Children’s Medical Center, Tehran University of Medical Science, Tehran, Iran.Pediatric Urology and Regenerative Medicine Research Center, Gene, Cell and Tissue Research Institute, Children’s Medical Center, Tehran University of Medical ScienceTehranIran

Abstract

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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Il testo completo di questo articolo è disponibile in PDF.

Abbreviations : AD, PD, CNS, BBB, MRNA, RNAi, SiRNA, RISC, MiRNA, ShRNA, GLUT, TfR, LDLR, LDL, , APP, APPα, RVG29, EGCG, PEG, APO A-1, SRB1, TrkA, NGF, QDs, R-BMSCs, SiSNCA, LDH, PPG, PLA, PHEA, HPMA

Keywords : RNA delivery, Nanoparticles, Gene therapy, Tissue engineering, Neurodegenerative disease, Blood-brain barrier


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© 2024  The Authors. Pubblicato da Elsevier Masson SAS. Tutti i diritti riservati.
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