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2015 ; 48
(3
): 147-52
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New paradigms on siRNA local application
#MMPMID25081998
Pan M
; Ni J
; He H
; Gao S
; Duan X
BMB Rep
2015[Mar]; 48
(3
): 147-52
PMID25081998
show ga
Small interfering RNA (siRNA) functions through pairing with specific mRNA
sequences and results in the mRNA's degradation. It is a potential therapeutic
approach for many diseases caused by altered gene expression. The delivery of
siRNA is still a major problem due to its rapid degradation in the circulation.
Various strategies have been proposed to help with the cellular uptake of siRNA
and short or small hairpin RNA (shRNA). Here, we reviewed recently published data
regarding local applications of siRNA. Compared with systemic delivery methods,
local delivery of siRNA/shRNA has many advantages, such as targeting the specific
tissues or organs, mimicking a gene knockout effect, or developing certain
diseases models. The eye, brain, and tumor tissues are 'hot' target
tissues/organs for local siRNA delivery. The siRNA can be delivered locally, in
naked form, with chemical modifications, or in formulations with viral or
non-viral vectors, such as liposomes and nanoparticles. This review provides a
comprehensive overview of RNAi local administration and potential future
applications in clinical treatment.
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