Errico Alessia, Ballabio Andrea, Rugarli Elena I
Telethon Institute of Genetics and Medicine (TIGEM), II University of Naples, Naples, Italy.
Hum Mol Genet. 2002 Jan 15;11(2):153-63. doi: 10.1093/hmg/11.2.153.
Hereditary spastic paraplegia (HSP) is characterized by progressive weakness and spasticity of the lower limbs, caused by the specific degeneration of the corticospinal tracts, the longest axons in humans. Most cases of the autosomal dominant form of the disease are due to mutations in the SPG4 gene, which encodes spastin, an ATPase belonging to the AAA family. The cellular pathways in which spastin operates and its role in causing degeneration of motor axons are currently unknown. By expressing wild-type or ATPase-defective spastin in several cell types, we now show that spastin interacts dynamically with microtubules. Spastin association with the microtubule cytoskeleton is mediated by the N-terminal region of the protein, and is regulated through the ATPase activity of the AAA domain. Expression of all the missense mutations into the AAA domain, which were previously identified in patients, leads to constitutive binding to microtubules in transfected cells and induces the disappearance of the aster and the formation of thick perinuclear bundles, suggesting a role of spastin in microtubule dynamics. Consistently, wild-type spastin promotes microtubule disassembly in transfected cells. These data suggest that spastin may be involved in microtubule dynamics similarly to the highly homologous microtubule-severing protein, katanin. Impairment of fine regulation of the microtubule cytoskeleton in long axons, due to spastin mutations, may underlie pathogenesis of HSP.
遗传性痉挛性截瘫(HSP)的特征是下肢进行性无力和痉挛,由人类最长轴突——皮质脊髓束的特异性退化引起。该疾病常染色体显性形式的大多数病例是由于SPG4基因突变所致,该基因编码一种属于AAA家族的ATP酶——痉挛蛋白。目前尚不清楚痉挛蛋白发挥作用的细胞途径及其在导致运动轴突退化中的作用。通过在几种细胞类型中表达野生型或ATP酶缺陷型痉挛蛋白,我们现在表明痉挛蛋白与微管动态相互作用。痉挛蛋白与微管细胞骨架的结合由该蛋白的N端区域介导,并通过AAA结构域的ATP酶活性进行调节。先前在患者中鉴定出的所有错义突变在AAA结构域中的表达,导致在转染细胞中与微管的组成型结合,并诱导星状体消失和核周粗大束状结构的形成,这表明痉挛蛋白在微管动态变化中发挥作用。同样,野生型痉挛蛋白在转染细胞中促进微管解聚。这些数据表明,痉挛蛋白可能与高度同源的微管切断蛋白katanin类似,参与微管动态变化。由于痉挛蛋白突变导致长轴突中微管细胞骨架精细调节受损,可能是HSP发病机制的基础。