Suppr超能文献

遗传性痉挛性截瘫:从诊断到新兴治疗方法。

Hereditary spastic paraplegia: from diagnosis to emerging therapeutic approaches.

机构信息

Reta Lila Weston Institute of Neurological Studies, UCL Queen Square Institute of Neurology, London, UK.

Cambridge Institute for Medical Research and Department of Medical Genetics, University of Cambridge, Cambridge, UK.

出版信息

Lancet Neurol. 2019 Dec;18(12):1136-1146. doi: 10.1016/S1474-4422(19)30235-2. Epub 2019 Jul 31.

Abstract

Hereditary spastic paraplegia (HSP) describes a heterogeneous group of genetic neurodegenerative diseases characterised by progressive spasticity of the lower limbs. The pathogenic mechanism, associated clinical features, and imaging abnormalities vary substantially according to the affected gene and differentiating HSP from other genetic diseases associated with spasticity can be challenging. Next generation sequencing-based gene panels are now widely available but have limitations and a molecular diagnosis is not made in most suspected cases. Symptomatic management continues to evolve but with a greater understanding of the pathophysiological basis of individual HSP subtypes there are emerging opportunities to provide targeted molecular therapies and personalised medicine.

摘要

遗传性痉挛性截瘫(HSP)描述了一组异质性的遗传性神经退行性疾病,其特征是下肢进行性痉挛。发病机制、相关临床特征和影像学异常根据受影响的基因而有很大差异,因此 HSP 与其他伴有痉挛的遗传性疾病的鉴别具有挑战性。基于下一代测序的基因面板现在已经广泛应用,但存在局限性,在大多数疑似病例中都无法做出分子诊断。对症治疗仍在不断发展,但随着对个体 HSP 亚型病理生理基础的认识不断加深,为提供有针对性的分子治疗和个体化医疗提供了新的机会。

文献AI研究员

20分钟写一篇综述,助力文献阅读效率提升50倍。

立即体验

用中文搜PubMed

大模型驱动的PubMed中文搜索引擎

马上搜索

文档翻译

学术文献翻译模型,支持多种主流文档格式。

立即体验