Non-viral siRNA delivery to the lung.

TitleNon-viral siRNA delivery to the lung.
Publication TypeJournal Article
Year of Publication2007
AuthorsThomas, M, Lu, JJ, Chen, J, Klibanov, AM
JournalAdv Drug Deliv Rev
Date Published2007 Mar 30
KeywordsAnimals, Drug Delivery Systems, Gene Silencing, Humans, Lung, Lung Diseases, RNA, Small Interfering, Treatment Outcome

SiRNAs exert their biological effect by guiding the degradation of their cognate mRNA sequence, thereby shutting down the corresponding protein production (gene silencing by RNA interference or RNAi). Due to this property, siRNAs are emerging as promising therapeutic agents for the treatment of inherited and acquired diseases, as well as research tools for the elucidation of gene function in both health and disease. Because of their lethality and prevalence, lung diseases have attracted particular attention as targets of siRNA-mediated cures. In addition, lung is accessible to therapeutic agents via multiple routes, e.g., through the nose and the mouth, thus obviating the need for targeting and making it an appealing target for RNAi-based therapeutic strategies. The clinical success of siRNA-mediated interventions critically depends upon the safety and efficacy of the delivery methods and agents. Delivery of siRNAs relevant to lung diseases has been attempted through multiple routes and using various carriers in animal models. This review focuses on the recent progress in non-viral delivery of siRNAs for the treatment of lung diseases, particularly infectious diseases. The rapid progress will put siRNA-based therapeutics on fast track to the clinic.

Alternate JournalAdv. Drug Deliv. Rev.
PubMed ID17459519
Grant ListAI56267 / AI / NIAID NIH HHS / United States
EB000244 / EB / NIBIB NIH HHS / United States
P50 CA112967 / CA / NCI NIH HHS / United States
U54 CA119349 / CA / NCI NIH HHS / United States