siRNA as a tool to improve the treatment of brain diseases: Mechanism, targets and delivery.

摘要: As the population ages, brain pathologies such as neurodegenerative diseases and cancer increase their incidence, being need to find successful treatments of upmost importance. Drug delivery central nervous system (CNS) is required in order reach causes treat them. However, biological barriers, mainly blood-brain barrier (BBB), are key obstacles that prevent effectiveness possible due ability strongly limit perfusion compounds into brain. Over past decades, new approaches towards overcoming BBB its efflux transporters had been proposed. One these here reviewed through small interfering RNA (siRNA), which capable specifically target one gene silence it a post-transcriptional way. There different functional proteins at BBB, ones responsible for transport or just tightness, could be siRNA target. important effective way anatomical site action. This where nanotechnology-based systems may help, by protecting circulation providing cell/tissue-targeting intracellular delivery. After an initial overview on incidence basic features CNS, pumps, this review focuses recent strategies based siRNA, how diseases.