ABSTRACT
The realization of gene therapy for the central nervous system has advanced to the realms
of clinical practice. The brain and spinal cord, once inaccessible to certain therapies, have
been unlocked by gene therapy vectors allowing medical practitioners to target areas of the
brain in a manner never before attainable. For example, certain vectors can target tumors,
replicate in those cells, and deliver toxic substances to rid the brain of cancers. Also, we
can now deliver genes to certain cell types of the brain and allow continuous gene delivery,
delivering a therapeutic gene to the exact cells that are perishing in neurological disorders
or injury. These discoveries have advanced the concept of gene therapy for the nervous
system to a clinical reality.
