In vivo and ex vivo gene transfer to the brain

The use of gene transfer techniques to express novel proteins within different cellular populations has provided insights into the function and plasticity of the brain. Recently, this technique has been successfully used to explore physiological processes within the CNS and to intervene in neurodegenerative disease and cancer. Progress in manipulating transgene products in vivo and in achieving cell-specific targeting of genetic material offers promise for enhancing the usefulness of this technique and its therapeutic potential for treating human disorders of the CNS.