Gene therapy and pharmacological treatment of inherited neurological disorders

Abstract

The identification of mutant genes responsible for inherited neurological disorders gives an opportunity to consider new approaches to their treatment. Although replacement of defective genes in postmitotic neurons is unlikely to be possible in the near future, the identification of gene products, and definitive delineation of the cellular dysfunction and death that result from mutated gene products, may suggest new therapeutic options. This article reviews these advances and considers approaches that may provide hope for alleviation of these disorders.