Gene therapy for diseases of the nervous system in childhood

Gene therapy seems very promising for numerous diseases of the developing nervous system of infants. The efficacy of such an approach relies on different parameters: 1) choice of a target gene; 2) selection of a promoter permitting a robust and protracted expression of the delivered gene in the target cells; 3) selection of a vector permitting to deliver the gene to the right cells with efficacy and without side-effects; 4) choice of the way to administer the vector to the target cells. Although experimental and methodological tools have greatly improved during the last decade, gene therapy of child nervous system diseases is still in the field of basic research.