[Prospects for gene treatment in paediatric neurology]

The current advances in the field of human genetics allow for the development of alternative therapies for the treatment of diseases affecting the central nervous system (CNS). One of the most promising new therapies is the use of therapeutic genes or gene therapy.

To briefly review the recent advances for the treatment of diseases affecting the CNS.

We consider the therapeutic strategies currently in study for gene therapy of diseases with neurologic symptoms as monogenic diseases, CNS malignancies and neurodegenerative diseases. We describe the pros and cons of viral, non viral vectors and cell based therapy.

The current gene therapy approaches allow for specific and efficient administration of therapeutic genes to the CNS. Despite the need of optimization of the available vectors, mainly regarding to safety and efficacy, gene therapy has an important potential for the treatment of neuropediatric diseases.