Banchs Isabel, Casasnovas Carlos, Albertí Antonia, De Jorge Laura, Povedano Mónica, Montero Jordi, Martínez-Matos Juan Antonio, Volpini Victor
Molecular Diagnosis Center of Inherited Diseases, Institut de Investigació Biomèdica de Bellvitge, Gran Via 199, 08907 L'Hospitalet de Llobregat, Barcelona, Spain.
J Biomed Biotechnol. 2009;2009:985415. doi: 10.1155/2009/985415. Epub 2009 Oct 8.
Charcot-Marie-Tooth (CMT) disease or hereditary motor and sensory neuropathy (HMSN) is a genetically heterogeneous group of conditions that affect the peripheral nervous system. The disease is characterized by degeneration or abnormal development of peripheral nerves and exhibits a range of patterns of genetic transmission. In the majority of cases, CMT first appears in infancy, and its manifestations include clumsiness of gait, predominantly distal muscular atrophy of the limbs, and deformity of the feet in the form of foot drop. It can be classified according to the pattern of transmission (autosomal dominant, autosomal recessive, or X linked), according to electrophysiological findings (demyelinating or axonal), or according to the causative mutant gene. The classification of CMT is complex and undergoes constant revision as new genes and mutations are discovered. In this paper, we review the most efficient diagnostic algorithms for the molecular diagnosis of CMT, which are based on clinical and electrophysiological data.
夏科-马里-图斯病(CMT)或遗传性运动感觉神经病(HMSN)是一组影响周围神经系统的遗传性异质性疾病。该疾病的特征是周围神经的退化或异常发育,并呈现出一系列遗传传递模式。在大多数情况下,CMT最早出现在婴儿期,其表现包括步态笨拙、四肢主要为远端肌肉萎缩以及足下垂形式的足部畸形。它可以根据传递模式(常染色体显性、常染色体隐性或X连锁)、根据电生理结果(脱髓鞘或轴索性)或根据致病突变基因进行分类。CMT的分类很复杂,随着新基因和突变的发现不断进行修订。在本文中,我们回顾了基于临床和电生理数据的CMT分子诊断最有效的诊断算法。