VOLUME 8 | FEBRUARY 2009 | Kathryn A. Whitehead*, Robert Langer *† and Daniel G. Anderson†
The article provides an overview of the progress in the development and application of small interfering RNA (siRNA) for therapeutic purposes. It highlights the challenges and advancements in siRNA delivery systems, emphasizing the need for safe and effective methods to deliver siRNA to target tissues. The authors discuss the mechanisms of RNA interference, the role of siRNA in gene silencing, and the importance of chemical modifications to enhance siRNA's therapeutic potential. They also review various synthetic materials used for siRNA delivery, including liposomes, lipidoids, polymers, and polyethylenimine (PEI). The article concludes by discussing ongoing clinical trials for siRNA-based therapies, particularly for age-related macular degeneration and respiratory syncytial virus, and outlines future directions for improving siRNA delivery and therapeutic efficacy.The article provides an overview of the progress in the development and application of small interfering RNA (siRNA) for therapeutic purposes. It highlights the challenges and advancements in siRNA delivery systems, emphasizing the need for safe and effective methods to deliver siRNA to target tissues. The authors discuss the mechanisms of RNA interference, the role of siRNA in gene silencing, and the importance of chemical modifications to enhance siRNA's therapeutic potential. They also review various synthetic materials used for siRNA delivery, including liposomes, lipidoids, polymers, and polyethylenimine (PEI). The article concludes by discussing ongoing clinical trials for siRNA-based therapies, particularly for age-related macular degeneration and respiratory syncytial virus, and outlines future directions for improving siRNA delivery and therapeutic efficacy.