Research progress in nasal delivery of siRNA for the treatment of brain diseases
ZHOU Mei-ling
SU Ping
ZHANG Ming-wan
HUANG Yuan
QU Ke-run
JIANG Jin-jin
LI Rui
Abstract：There still lacks of effective therapeutic drugs and therapeutic targets for the treatment of brain diseases such as neurodegenerative disorders and brain tumors at present. With the pathogenesis is being clarified at genetic level, gene therapy strategy for brain diseases has attracted increasing attention. Small interfering RNA (siRNA)is widely regarded as one of the most promising gene therapy drugs. However, the application of siRNA is extremely limited in vivo due to its large molecular weight, hydrophilic nature, easy degradation by nuclease, and poor permeability through the blood-brain barrier (BBB ). Nasal drug delivery systems can directly deliver therapeutics into the brain by passing the BBB, which provides a safe, effective and convenient delivery strategy for siRNA to treat brain diseases. This review summarizes the research progress in intranasal siRNA transport and nanoscale siRNA delivery systems so as to offer reliable references to achieve better therapeutic effect of siRNA delivered through the nose-brain pathway.
Keywords：brain diseasesgene therapy strategysmall interfering RNAnose-brain pathwaynanoscale siRNA delivery systems
Publication Date：2018-01-01
Online Publishing Date：2026-08-14(First online date of this platform, not the publication date of the document)
Pages：5( 887-891 )