Ryan Allen F, Dazert Stefan
Adv Otorhinolaryngol. 2009;66:1-12. doi: 10.1159/000218203. Epub 2009 Jun 2.
Since the recognition of genes as the discrete units of heritability, and of DNA as their molecular substrate, the utilization of genes for therapeutic purposes has been recognized as a potential means of correcting genetic disorders. The tools of molecular biology, which allow the manipulation of DNA sequence, provided the means to put this concept into practice. However, progress in the implementation of these ideas has been slow. Here we review the history of the idea of gene therapy and the complexity of genetic disorders. We also discuss the requirements for sequence-based therapy to be accomplished for different types of inherited diseases, as well as the methods available for gene manipulation. The challenges that have limited the applications of gene therapy are reviewed, as are ethical concerns. Finally, we discuss the promise of gene therapy to address inherited and acquired disorders of the inner ear.
自从基因被确认为遗传的离散单位，以及DNA被确认为其分子底物以来，利用基因进行治疗已被视为纠正遗传疾病的一种潜在手段。分子生物学工具能够对DNA序列进行操作，为将这一概念付诸实践提供了手段。然而，这些想法在实施过程中的进展一直很缓慢。在此，我们回顾基因治疗理念的历史以及遗传疾病的复杂性。我们还讨论了针对不同类型遗传性疾病实现基于序列治疗的要求，以及可用于基因操作的方法。回顾了限制基因治疗应用的挑战以及伦理问题。最后，我们讨论基因治疗在解决内耳遗传性和获得性疾病方面的前景。
