New Paradigms on siRNA Local Application.

New Paradigms on siRNA Local Application.
BMB Rep. 2014 Jul 31;
Authors: Pan M, Ni J, He H, Gao S, Duan X
Abstract
Small interfering RNA (siRNA) functions through pairing with specific mRNA sequences and results in mRNA degradation, which is becoming a potential therapeutic approach for many diseases caused by altered gene expression. The delivery is a major problem for siRNA application due to its rapid degradation in blood circulation. Various strategies have been proposed to help cellular uptake of siRNA/short or small hairpin RNA (shRNA) successfully. Here we reviewed the recent published data regarding local applications of siRNA. Compared with systematic delivery methods, local delivery of siRNA/shRNA demonstrates many advantages, such as targeting the specific tissues or organs, mimicking the gene knockout effect or setting up certain diseases models. Eye, brain or tumor tissues are the hot target tissues/organs for local siRNA delivery. The siRNA is locally delivered either in naked form, or with chemical modifications or in formulations with viral vector and nonviral vectors such as liposomes and nanoparticles. This review will give a comprehensive insight of RNAi local administration and the potential applications in the future clinic treatment.
PMID: 25081998 [PubMed - as supplied by publisher]

Source: BMB Reports - July 31, 2014 Category: Biochemistry Authors: Pan M, Ni J, He H, Gao S, Duan X Tags: BMB Rep Source Type: research