Brain diseases are the most serious health problems; represent a significant and worldwide public health
problem. Small interfering RNAs (siRNAs) can initiate specific silencing of genes and are potential therapeutic
agents for many genetically influenced diseases including brain disease. However, on systemic administration the
blood-brain barrier (BBB) poses most significant obstacle for the therapeutic siRNAs delivery to the brain. Therefore,
the development of successful approaches to enhance siRNA delivery to the brain is of immense interest in
clinical and pharmaceutical research. At present, intranasal delivery approach serves as an effective mode of direct
delivery of siRNAs to brain by bypassing BBB. In this review, we describe the principles of RNA interference
(RNAi) machinery; challenges associated with siRNAs in therapeutics brain targeting and summarize the recent
progress made in the use of vector based siRNA technology. Further, it is anticipated that intranasal delivery approach
will have a very important role to play in the future for the translation of siRNAs therapeutics from bench to
bedside for different brain diseases.
Keywords: siRNA, brain diseases, intranasal delivery, brain targeting, nanotechnology, delivery vehicles.
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