Freitas Regina Toni Loureiro de, Zanoteli Edmar, Morita Maria da Penha Ananias, Oliveira Acary Souza Bulle
Disciplina de Neurologia da Universidade Federal de São Paulo, Brazil.
Arq Neuropsiquiatr. 2005 Jun;63(2B):514-8. doi: 10.1590/s0004-282x2005000300027. Epub 2005 Jul 25.
Congenital muscular dystrophy (CMD) composes a group of disorders characterized by hypotonia and muscular weakness noticed in the first year of life. The Ullrich's form is characterized by proximal joint contractures and distal hiperextensibility. About 40% of these patients present mutations in one of the genes that codify the sub-units of the collagen VI protein (COL6), producing total or partial deficiency of the protein expression. We analyzed, through immunohistochemistry, the expression of COL6 in muscle fragments of 50 patients with CMD; 20 of them presented merosin expression deficiency. We identified 4 cases with total COL6 deficiency (8% of the total), representing 13% of the cases with normal merosin expression. The histological findings of patients with deficiency of COL6 were indistinguishable from other forms of CMD, but milder than that abnormalities observed in merosin deficient patients. In three COL6 deficient patients were observed hypotonia and weakness in the neonatal period, delayed of motor milestones, muscular retractions of knees and elbows, distal joint hiperextensibility and congenital hip dislocation (two patients). One patient lost the ability to walk; and one died due to respiratory problems. The analysis of COL6 expression, as well as merosin expression, in the muscle tissue from CMD patients, can be important for identification and phenotypic characterization of different CMD subtypes.
先天性肌营养不良(CMD)是一组以出生后第一年出现肌张力低下和肌无力为特征的疾病。乌尔里希型的特点是近端关节挛缩和远端关节过度伸展。这些患者中约40%在编码胶原蛋白VI(COL6)亚基的基因之一中存在突变,导致该蛋白表达完全或部分缺失。我们通过免疫组织化学分析了50例CMD患者肌肉片段中COL6的表达;其中20例存在merosin表达缺失。我们鉴定出4例COL6完全缺乏的病例(占总数的8%),占merosin表达正常病例的13%。COL6缺乏患者的组织学表现与其他形式的CMD无法区分,但比merosin缺乏患者观察到的异常情况要轻。在3例COL6缺乏的患者中,新生儿期出现肌张力低下和肌无力、运动发育迟缓、膝部和肘部肌肉挛缩、远端关节过度伸展以及先天性髋关节脱位(2例)。1例患者失去行走能力;1例因呼吸问题死亡。分析CMD患者肌肉组织中COL6以及merosin的表达,对于不同CMD亚型的鉴定和表型特征分析可能具有重要意义。