Slone Jesse, Huang Taosheng
Division of Human Genetics, Cincinnati Children's Hospital Medical Center, 3333 Burnet Avenue, Cincinnati, OH 45229 USA.
NPJ Genom Med. 2020 Mar 2;5:7. doi: 10.1038/s41525-020-0116-5. eCollection 2020.
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.
近期多个临床试验中基因治疗的成功，激发了人们对治疗以往难以治愈的遗传疾病的巨大希望。这种乐观情绪已延伸到线粒体疾病的基因治疗前景，线粒体疾病不仅特别严重，而且难以治疗。然而，这种希望必须因线粒体细胞器的实际情况而有所缓和，线粒体具有特定的生物学特性，使基因操作变得复杂。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。我们还将介绍一些通过专门针对线粒体疾病定制的基因操作策略来克服这些挑战的方法。
