The design and exogenous delivery of siRNA for post-transcriptional gene silencing.
RNA interference (RNAi) is a natural cellular process that effects post-transcriptional gene silencing in eukaryotic systems. Small interfering RNA (siRNA) molecules are the key intermediaries in this process which when exogenously administered can inhibit or "silence" the expression of any given target gene. Thus, siRNA molecules hold great promise as biological tools and as potential therapeutic agents for targeted inhibition of disease-causing genes. However, key challenges to the effective and widespread use of these polyanionic, macromolecular duplexes of RNA are their appropriate design and efficient delivery to cells in vitro and in vivo. This review highlights the current strategies used in the design of effective siRNA molecules and also summarises the main strategies being considered for the exogenous delivery of siRNA for both in vitro and in vivo applications.
Gilmore
Ian R
IR
Centre for Genome-based Therapeutics, The Welsh School of Pharmacy, Cardiff University, Redwood Building, King Edward VII Avenue, Cardiff CF10 3XF, UK.
Fox
Stephen P
SP
Hollins
Andrew J
AJ
Sohail
Muhammad
M
Akhtar
Saghir
S
D000818:Animals; D016503:Drug Delivery Systems; D018014:Gene Transfer Techniques; D006801:Humans; D034622:RNA Interference; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000737:chemistry / Q000235:genetics*
D034741:RNA, Small Interfering