Knocking down barriers: advances in siRNA delivery.
In the 10 years that have passed since the Nobel prize-winning discovery of RNA interference (RNAi), billions of dollars have been invested in the therapeutic application of gene silencing in humans. Today, there are promising data from ongoing clinical trials for the treatment of age-related macular degeneration and respiratory syncytial virus. Despite these early successes, however, the widespread use of RNAi therapeutics for disease prevention and treatment requires the development of clinically suitable, safe and effective drug delivery vehicles. Here, we provide an update on the progress of RNAi therapeutics and highlight novel synthetic materials for the encapsulation and intracellular delivery of nucleic acids.
Whitehead
Kathryn A
KA
Department of Chemical Engineering, Massachusetts Institute of Technology, Cambridge, Massachusetts 02142, USA.
Langer
Robert
R
Anderson
Daniel G
DG
D000818:Animals; D002986:Clinical Trials as Topic; D016503:Drug Delivery Systems / Q000379:methods* / Q000639:trends; D006801:Humans; D008081:Liposomes; D053758:Nanoparticles / Q000008:administration & dosage*; D034622:RNA Interference / Q000187:drug effects / Q000502:physiology*; D034741:RNA, Small Interfering / Q000008:administration & dosage* / Q000494:pharmacology
D008081:Liposomes; D034741:RNA, Small Interfering