Background: The lack of an outright treatment for Parkinson’s disease (PD) is a pivotal
concern in medicine and has driven the search for novel alternatives for treating the disease. Among
the proposed approaches, small interfering RNA (siRNA)-based therapy is attracting significant attention
as a potential method for the treatment of PD; however, siRNAs delivery possesses potential
drawbacks, such as reduced stability in blood circulation and low capacity for reaching the target site.
Objective: This review aims to explore siRNA-based approaches to PD and the latest advances for designing
nanoparticles that effectively target siRNAs to the action site and that protect these against
degradation in blood circulation.
Results: siRNA-based approaches provide an interesting option for designing new strategies for treating
PD through the silencing of genes, whose abnormal expressions contribute to the pathophysiology
of the disease; however, siRNA delivery to the brain is a key issue that remains unsolved to date. Current
research efforts are focused on designing vectors that effectively transport and protect siRNAs. In
this regard, nanoparticles are being developed as carriers for siRNAs with controlled delivery efficiency
and low toxicity profiles, and these represent an alternative to common vectors.
Conclusion: Identification of putative gene targets for siRNA therapy of PD has set the pace for researching
non-viral vectors; however, the technological aspects for tackling the challenge that siRNAs
targeting to the brain represents are essentials. In this respect, the formulation of siRNAs in nanoparticles
would avoid harmful side effects, such as immunogenic and oncogenic drawbacks.