Suppr超能文献

与NFU1突变相关的新型痉挛性截瘫表型。

New spastic paraplegia phenotype associated to mutation of NFU1.

作者信息

Tonduti Davide, Dorboz Imen, Imbard Apolline, Slama Abdelhamid, Boutron Audrey, Pichard Samia, Elmaleh Monique, Vallée Louis, Benoist Jean François, Ogier Heléne, Boespflug-Tanguy Odile

机构信息

Paris Diderot University - Sorbonne Paris Cité; Inserm U1141, DHU PROTECT, Robert Debré Hospital, Paris, France.

Department of Brain and Behavioral Sciences, Unit of Child Neurology and Psychiatry, University of Pavia, Pavia, Italy.

出版信息

Orphanet J Rare Dis. 2015 Feb 8;10:13. doi: 10.1186/s13023-015-0237-6.

Abstract

Recently an early onset lethal encephalopathy has been described in relation to mutations of NFU1, one of the genes involved in iron-sulfur cluster metabolism. We report a new NFU1 mutated patient presenting with a milder phenotype characterized by a later onset, a slowly progressive spastic paraparesis with relapsing-remitting episodes, mild cognitive impairment and a long survival. The early white matter abnormalities observed on MRI was combined with a mixed sensory-motor neuropathy in the third decade. Our case clearly suggests the importance of considering NFU1 mutation in slowly evolving leukoencephalopathy with high glycine concentration.

摘要

最近,已经描述了一种与铁硫簇代谢相关基因之一的NFU1突变有关的早发性致死性脑病。我们报告了一名新的NFU1突变患者,其表现出较轻的表型,特征为发病较晚、缓慢进展的痉挛性截瘫伴复发-缓解发作、轻度认知障碍和长期存活。在第三个十年中,MRI上观察到的早期白质异常与混合性感觉运动性神经病变并存。我们的病例清楚地表明,在高甘氨酸浓度的缓慢进展性白质脑病中考虑NFU1突变的重要性。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/57db/4333890/2df22ff81c79/13023_2015_237_Fig1_HTML.jpg

相似文献

1
New spastic paraplegia phenotype associated to mutation of NFU1.
Orphanet J Rare Dis. 2015 Feb 8;10:13. doi: 10.1186/s13023-015-0237-6.
2
A genetic mimic of cerebral palsy: Homozygous NFU1 mutation with marked intrafamilial phenotypic variation.
Brain Dev. 2020 Nov;42(10):756-761. doi: 10.1016/j.braindev.2020.07.009. Epub 2020 Aug 1.
3
Phenotypic continuum of NFU1-related disorders.
Ann Clin Transl Neurol. 2022 Dec;9(12):2025-2035. doi: 10.1002/acn3.51679. Epub 2022 Oct 18.
5
A presenilin 1 mutation (Arg278Ser) associated with early onset Alzheimer's disease and spastic paraparesis.
J Neurol Sci. 2007 Sep 15;260(1-2):78-82. doi: 10.1016/j.jns.2007.04.013. Epub 2007 May 15.
6
Atypical adult onset complicated spastic paraparesis with thin corpus callosum in two patients carrying a novel FA2H mutation.
Eur J Neurol. 2012 Nov;19(11):e127-9. doi: 10.1111/j.1468-1331.2012.03838.x. Epub 2012 Aug 27.
7
A leaky splicing mutation in NFU1 is associated with a particular biochemical phenotype. Consequences for the diagnosis.
Mitochondrion. 2016 Jan;26:72-80. doi: 10.1016/j.mito.2015.12.004. Epub 2015 Dec 11.
9
No association of spastic paraparesis genes in PSEN1 Alzheimer's disease with spastic paraparesis.
Neuroreport. 2007 Aug 6;18(12):1267-9. doi: 10.1097/WNR.0b013e3282405209.
10
A novel presenilin 1 mutation (Y154N) in a patient with early onset Alzheimer's disease with spastic paraparesis.
Neurosci Lett. 2004 Sep 30;368(3):319-22. doi: 10.1016/j.neulet.2004.07.057.

引用本文的文献

3
Phenotypic continuum of NFU1-related disorders.
Ann Clin Transl Neurol. 2022 Dec;9(12):2025-2035. doi: 10.1002/acn3.51679. Epub 2022 Oct 18.
10
"Idiopathic" pulmonary arterial hypertension in early infancy: Excluding NFU1 deficiency.
Ann Pediatr Cardiol. 2019 Sep-Dec;12(3):325-328. doi: 10.4103/apc.APC_136_18.

本文引用的文献

1
Leukoencephalopathy with cysts and hyperglycinemia may result from NFU1 deficiency.
Mitochondrion. 2014 Mar;15:59-64. doi: 10.1016/j.mito.2014.01.003. Epub 2014 Jan 22.
5
Maturation of iron-sulfur proteins in eukaryotes: mechanisms, connected processes, and diseases.
Annu Rev Biochem. 2008;77:669-700. doi: 10.1146/annurev.biochem.76.052705.162653.
6
Diagnostic criteria for respiratory chain disorders in adults and children.
Neurology. 2002 Nov 12;59(9):1406-11. doi: 10.1212/01.wnl.0000033795.17156.00.

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