Gene Therapy to Address Unmet Needs in Neurology2

This Neurobiology of Disease Workshop, held at Neuroscience 2017, embraces the breadth of "gene therapy," including viral vectors, oligonucleotides, and cell therapies used in promising preclinical studies and clinical trials for a variety of neurological disorders long thought to be incurable. These new methods involve DNA engineering, gene replacement using virus vectors and the patient's own genetically modified cells, oligonucleotides that can "revive" beneficial gene functions or suppress toxic ones, and viruses and cells armed to tackle brain tumors.