Gene Therapy Using Neural Stem Cells

Cell replacement and gene transfer approaches for the diseased or injured CNS have provided the basis for the development of potentially powerful new therapeutic strategies for a broad spectrum of brain diseases. Transplantation of cells engineered to produce growth factors or molecules with antitumor effects shows the potential of grafted cells as vectors for protein delivery. However, for any possible clinical application, evidence that the gene transfer is completely innocuous and safe for the recipient must be obtained. To this regard, special cautions must be taken not to interfere with other brain functions apart from the one to be targeted. The grafted cells must integrate without inducing further damage to the recipient brain, with no apparent systemic or local side effects, such as uncontrolled growth, production/release of harmful compounds, or inflammatory response.