Seneca Sara, Goemans Nathalie, Van Coster Rudy, Givron Patrice, Reybrouck Tony, Sciot Raf, Meulemans Ann, Smet Joel, Van Hove Johan L K
Center of Medical Genetics, Free University Brussels, Brussels, Belgium.
Am J Med Genet A. 2005 Aug 30;137(2):170-5. doi: 10.1002/ajmg.a.30854.
Several mutations in mitochondrial transfer RNA (tRNA) genes can cause mitochondrial myopathy. We describe a young girl who presented with pronounced exercise intolerance. The anaerobic threshold and the maximal oxygen consumption were decreased. She had decreased complex I and IV enzyme activity and ragged red fibers on muscle biopsy. An A to G transition at nucleotide position 7526 in tRNA Aspartate (tRNA(Asp)) gene was heteroplasmic in several of the patient's tissues. We were unable to detect the mutation in muscle tissue from the patient's mother. This case adds a new genetic etiology for mitochondrial myopathy. It also illustrates for patients with combined deficiency of the complex I and IV enzyme activity the value of sequencing in the affected tissue muscle, and not only in blood, all mitochondrial tRNA genes including those not commonly affected, such as in this case mt tRNA(Asp).
线粒体转运RNA(tRNA)基因的几种突变可导致线粒体肌病。我们描述了一名表现出明显运动不耐受的年轻女孩。无氧阈值和最大耗氧量降低。她的复合体I和IV酶活性降低,肌肉活检显示有破碎红纤维。天冬氨酸转运RNA(tRNA(Asp))基因第7526位核苷酸处的A到G转换在患者的多个组织中呈异质性。我们在患者母亲的肌肉组织中未检测到该突变。该病例为线粒体肌病增加了一种新的遗传病因。它还说明了对于复合体I和IV酶活性联合缺乏的患者,不仅要对血液进行测序,还要对受影响的组织肌肉进行测序,对所有线粒体tRNA基因进行测序的价值,包括那些通常不受影响的基因,如本例中的线粒体天冬氨酸转运RNA(mt tRNA(Asp))。