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在一名年轻女孩中,MRI 显示 17p 端粒缺失(包括 YWHAE,但不包括 PAFAH1B1)与正常脑结构相关的表达分析。

Expression analysis of a 17p terminal deletion, including YWHAE, but not PAFAH1B1, associated with normal brain structure on MRI in a young girl.

机构信息

Division of Medical Genetics, Kanagawa Children's Medical Center, Yokohama, Japan.

出版信息

Am J Med Genet A. 2012 Sep;158A(9):2347-52. doi: 10.1002/ajmg.a.35542. Epub 2012 Aug 7.

Abstract

Tyrosine 3-monooxygenase/tryptophan 5-monooxygenase activation protein, epsilon polypeptide (YWHAE), on chromosome 17p13.3, has been shown to play a crucial role in neuronal development. The deletion of YWHAE, but not platelet-activating factor acetylhydrolase, isoform 1b, subunit 1 (PAFAH1B1), underlies a newly recognized neurodevelopmental disorder, characterized by significant growth retardation, developmental delay/intellectual disability (DD/ID), distinctive facial appearance, and brain abnormalities. Here, we report on a girl with a terminal deletion of 17p13.3, including YWHAE but not PAFAH1B1, showing normal brain structure on MRI. She had mild developmental delay, a distinctive facial appearance, and severe growth retardation despite normal growth hormone levels, which was improved by growth hormone therapy. Expression analysis of YWHAE and PAFAH1B1 yielded results consistent with array CGH and FISH results. These results indicate that the dosage effect of YWHAE varies from severe to very mild structural brain abnormalities, and suggest that the expression of YWHAE is associated with a complex mechanism of neuronal development.

摘要

色氨酸 5-单加氧酶激活蛋白 ε 多肽(YWHAE)位于 17p13.3 染色体上,已被证明在神经元发育中发挥着关键作用。YWHAE 的缺失,但不是血小板激活因子乙酰水解酶 1b 亚基 1(PAFAH1B1),是一种新认识的神经发育障碍的基础,其特征是明显的生长迟缓、发育迟缓/智力残疾(DD/ID)、独特的面部特征和脑异常。在这里,我们报告了一个女孩患有 17p13.3 末端缺失,包括 YWHAE 但不包括 PAFAH1B1,其 MRI 显示正常的脑结构。她有轻度的发育迟缓,独特的面部特征和严重的生长迟缓,尽管生长激素水平正常,生长激素治疗后有所改善。YWHAE 和 PAFAH1B1 的表达分析结果与 array CGH 和 FISH 结果一致。这些结果表明,YWHAE 的剂量效应从严重到非常轻微的结构脑异常,提示 YWHAE 的表达与神经元发育的复杂机制有关。

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