RNA interference (RNAi) is a process of sequence-specific gene silencing and serves as a powerful molecular tool to manipulate gene expression in vitro and in vivo. RNAi technologies have been applied to study gene function and validate drug targets. Researchers are investigating RNAi-based compounds as novel therapeutics to treat a variety of human diseases that are currently lacking sufficient treatment. To date, numerous studies support that RNAi therapeutics can improve disease phenotypes in various rodent models of human disease. Here, we focus on the development of RNAi-based therapies aimed at treating neurological disorders for which reduction of mutant or toxic gene expression may provide clinical benefit. We review RNAi-based gene-silencing strategies, proof-of-concept studies testing therapeutic RNAi for CNS disorders, and highlight the most recent research aimed at transitioning RNAi-based therapeutics toward clinical trials.
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Article Synopsis
- RNA interference (RNAi) shows promise for treating neurological disorders but faces challenges like barriers in the central nervous system and unstable nucleic acids.
- The study created a virus-like non-viral gene delivery system using rabies virus glycoprotein-decorated liposomes (f(Lipo)-RVG29) specifically for delivering small interfering RNAs (siRNAs) to the brain, targeting Alzheimer's disease.
- The liposomal system effectively protects siRNA, improves gene delivery rates in brain tissue, and significantly reduces BACE1 expression in vivo, indicating its potential as a novel RNAi therapeutic tool.
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