Martin J B
Department of Neurology, University of California, San Francisco 94143-0402.
Trends Biotechnol. 1995 Jan;13(1):28-35. doi: 10.1016/S0167-7799(00)88898-8.
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.
确定导致遗传性神经疾病的突变基因，为考虑其治疗新方法提供了契机。尽管在不久的将来，不太可能在有丝分裂后的神经元中替换缺陷基因，但确定基因产物，以及明确由突变基因产物导致的细胞功能障碍和死亡，可能会提示新的治疗选择。本文回顾了这些进展，并探讨了可能为缓解这些疾病带来希望的方法。
