Antisense as a neuroscience tool and therapeutic agent.

Gene expression in the mammalian brain is highly complex and requires an immensely powerful functional genomics tool to unravel it. Antisense has the potential to meet this requirement, but has always been plagued by biological and technological hurdles that have made the technology unreliable. With recent progress in developing potent, low-toxicity nucleic acid chemistries and novel drug delivery methods to cross the blood-brain barrier, the use of antisense is gathering momentum.