[Therapeutic RNA interference for neurodegenerative diseases]

Abstract

Introduction: The discovery of RNA interference (RNAi), a biological way to control gene expression, has revolutionised the field of biomedical research. Of the many applications of RNAi, perhaps its use for therapeutic purposes is the most promising. One group of diseases where the use of therapeutic RNAi is being actively explored is that of neurodegenerative disorders.

Aim: To provide both the clinical neurologist and the basic researcher with an updated summary of the development of therapeutic RNAi for neurodegenerative diseases.

Development: The technical progress made in the manipulation of RNAi in neurons and the recent advances in our knowledge of the pathogenesis of neurodegenerative processes have been valuable aids in designing therapeutic RNAi for both hereditary and idiopathic diseases. Several pre-clinical trials have been successfully completed in animal models, thus clearing the way towards the design of clinical trials in humans. Nevertheless, before reaching that point more experimental studies need to be carried out in animals to prove the effectiveness of this mode of therapy and, still more important, to be able to predict the possible side effects of this procedure in the human brain.

Conclusions: Although it is still in the pre-clinical stages, the use of RNAi for therapeutic purposes in neurodegenerative diseases is producing some very promising results. The evolution of this field over the next few years may be critical for beginning its clinical application.