MRC Holland B.V., Amsterdam, The Netherlands.
PLoS One. 2019 Jul 24;14(7):e0220211. doi: 10.1371/journal.pone.0220211. eCollection 2019.
Spinal Muscular Atrophy (SMA) is a disorder characterized by the degeneration of motor neurons in the spinal cord, leading to muscular atrophy. In the majority of cases, SMA is caused by the homozygous absence of the SMN1 gene. The disease severity of SMA is strongly influenced by the copy number of the closely related SMN2 gene. In addition, an SMN variant lacking exons 7 and 8 has been reported in 8% and 23% of healthy Swedish and Spanish individuals respectively. We tested 1255 samples from the 1000 Genomes Project using a new version of the multiplex ligation-dependent probe amplification (MLPA) P021 probemix that covers each SMN exon. The SMN variant lacking exons 7 and 8 was present in up to 20% of individuals in several Caucasian populations, while being almost completely absent in various Asian and African populations. This SMN1/2Δ7-8 variant appears to be derived from an ancient deletion event as the deletion size is identical in 99% of samples tested. The average total copy number of SMN1, SMN2 and the SMN1/2Δ7-8 variant combined was remarkably comparable in all populations tested, ranging from 3.64 in Asian to 3.75 in African samples.
脊髓性肌萎缩症(SMA)是一种以脊髓运动神经元退化导致肌肉萎缩为特征的疾病。在大多数情况下,SMA 是由 SMN1 基因的纯合缺失引起的。SMA 的疾病严重程度受密切相关的 SMN2 基因拷贝数的强烈影响。此外,据报道,在分别来自瑞典和西班牙的 8%和 23%的健康个体中,存在一种缺乏外显子 7 和 8 的 SMN 变体。我们使用新的多重连接依赖性探针扩增(MLPA)P021 探针混合物对 1000 基因组计划中的 1255 个样本进行了测试,该混合物覆盖每个 SMN 外显子。在几个白种人群体中,多达 20%的个体存在缺乏外显子 7 和 8 的 SMN 变体,而在各种亚洲和非洲人群体中,这种变体几乎完全不存在。这种 SMN1/2Δ7-8 变体似乎源自一个古老的缺失事件,因为在 99%的测试样本中,缺失大小完全相同。在所有测试的人群中,SMN1、SMN2 和 SMN1/2Δ7-8 变体的总拷贝数平均值非常相似,范围从亚洲的 3.64 到非洲的 3.75。