In recent pre-clinical work, we have shown that pharmacologic agents targeting the immune system can prevent disease in the Ndufs4(KO) model of Leigh syndrome, indicating that the immune system plays a causal role in the pathogenesis of at least this form of mitochondrial disease. Intriguingly, a number of case reports have indicated that immune-targeting therapeutics may be beneficial in the setting of genetic mitochondrial disease. Here, we summarize clinical and pre-clinical evidence suggesting a key role for the immune system in mediating the pathogenesis of at least some forms of genetic mitochondrial disease.
Significant clinical and pre-clinical evidence indicates a key role for the immune system as a significant in the pathogenesis of at least some forms of genetic mitochondrial disease.
在最近的临床前工作中,我们已经证明,靶向免疫系统的药物可以预防疾病的Ndufs4(KO)Leigh综合征的模型,表明免疫系统在至少这种形式的线粒体疾病的发病机理中起着因果作用。有趣的是,大量病例报告表明,免疫靶向治疗可能对遗传性线粒体疾病有益.这里,我们总结了临床和临床前证据,这些证据表明免疫系统在介导至少某些形式的遗传性线粒体疾病的发病机制中起关键作用。
重要的临床和临床前证据表明免疫系统在至少某些形式的遗传性线粒体疾病的发病机理中起着重要作用。