[Antisense targeting in neurology]

Antisense targeting refers to the use of synthetic short lengths of single stranded DNA, or RNA with base sequences complementary to a specific gene or its mRNA. Commonly, synthetic oligonucleotides are designed to hybridize to specific mRNA and thus preventing its translation in a specific protein.

The use of this technology as research tool is well known since two decades ago, but it has been in the last few years, when it has been proposed as a promising tool for the development of a new generation of drugs with high specificity, relative ease of production and low rate of toxicity. Antisense therapeutics is currently being evaluated in clinical trials for cancer, inflammation, and viral diseases. In the field of Neuropharmacology, it has become in a very valuable tool to block the expression of specific genes in vitro as well in the living brain. In this article, we review the contributions of this technology in the field of the Neurosciences, and also give an overview concerning the advances of the antisense strategy in the design of possible new treatments for certain neurological disorders. Other clinically relevant information regarding molecular biology, pharmacokinetics, mechanism of action, and side effects of antisense oligonucleotides has been collected and summarized.

In the neuropharmacological area is the Neurooncology the most intensively researched; nevertheless, the lack of oligos that cross the blood-brain barrier in sufficient amount continues being one of the main difficulties for the successful application of this technique on the central nervous system.