GENE-THERAPY FOR DISEASES OF THE NERVOUS- SYSTEM IN CHILDHOOD

Gene therapy seems very promising for numerous diseases of the develop ing 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 delive red gene in the target cells; 3) selection of a vector permitting to d eliver the gene to the right cells with efficacy and without side-effe cts; 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.