The recent success of gene therapy across multiple clinical trials has inspired a great deal of hope regarding the treatment of previously intractable genetic diseases. This optimism has been extended to the prospect of gene therapy for mitochondrial disorders, which are not only particularly severe but also difficult to treat. However, this hope must be tempered by the reality of the mitochondrial organelle, which possesses specific biological properties that complicate genetic manipulation. In this perspective, we will discuss some of these complicating factors, including the unique pathways used to express and import mitochondrial proteins. We will also present some ways in which these challenges can be overcome by genetic manipulation strategies tailored specifically for mitochondrial diseases.

基因疗法在多个临床试验中的最新成功激发了人们对以前难治的遗传性疾病的治疗的巨大希望。这种乐观已经扩展到线粒体疾病的基因治疗的前景，线粒体疾病不仅特别严重，而且难以治疗。然而，这种希望必须通过线粒体细胞器的现实来缓和，它具有使基因操作复杂化的特定生物学特性。从这个角度出发，我们将讨论其中一些复杂因素，包括用于表达和导入线粒体蛋白质的独特途径。我们还将介绍一些方法，可以通过专门针对线粒体疾病量身定制的遗传操作策略来克服这些挑战。