New paradigms on siRNA local application
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Small interfering RNA (siRNA) functions through pairing withspecific mRNA sequences and results in the mRNA’s degradation. It is a potential therapeutic approach for many diseasescaused by altered gene expression. The delivery of siRNA isstill a major problem due to its rapid degradation in the circulation. Various strategies have been proposed to help with thecellular uptake of siRNA and short or small hairpin RNA(shRNA). Here, we reviewed recently published data regardinglocal applications of siRNA. Compared with systemic deliverymethods, local delivery of siRNA/shRNA has many advantages,such as targeting the specific tissues or organs, mimickinga gene knockout effect, or developing certain diseasesmodels. The eye, brain, and tumor tissues are ‘hot’ target tissues/organs for local siRNA delivery. The siRNA can be deliveredlocally, in naked form, with chemical modifications, or informulations with viral or non-viral vectors, such as liposomesand nanoparticles. This review provides a comprehensiveoverview of RNAi local administration and potential future applicationsin clinical treatment.
Abstract
Small interfering RNA (siRNA) functions through pairing withspecific mRNA sequences and results in the mRNA’s degradation. It is a potential therapeutic approach for many diseasescaused by altered gene expression. The delivery of siRNA isstill a major problem due to its rapid degradation in the circulation. Various strategies have been proposed to help with thecellular uptake of siRNA and short or small hairpin RNA(shRNA). Here, we reviewed recently published data regardinglocal applications of siRNA. Compared with systemic deliverymethods, local delivery of siRNA/shRNA has many advantages,such as targeting the specific tissues or organs, mimickinga gene knockout effect, or developing certain diseasesmodels. The eye, brain, and tumor tissues are ‘hot’ target tissues/organs for local siRNA delivery. The siRNA can be deliveredlocally, in naked form, with chemical modifications, or informulations with viral or non-viral vectors, such as liposomesand nanoparticles. This review provides a comprehensiveoverview of RNAi local administration and potential future applicationsin clinical treatment.
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