Fragile X syndrome and associated disorders: Clinical aspects and pathology

被引:82
作者
Salcedo-Arellano, Maria Jimena [1 ,2 ,3 ,4 ,5 ]
Dufour, Brett [3 ,4 ,5 ]
McLennan, Yingratana [1 ,2 ,3 ,4 ]
Martinez-Cerdeno, Veronica [2 ,3 ,4 ,5 ]
Hagerman, Randi [1 ,2 ]
机构
[1] Univ Calif Davis, Sch Med, Dept Pediat, Sacramento, CA 95817 USA
[2] Univ Calif Davis, Med Invest Neurodev Disorders MIND Inst, Sacramento, CA 95817 USA
[3] Inst Pediat Regenerat Med, Sacramento, CA USA
[4] Shriners Hosp Children Northern Calif, Sacramento, CA USA
[5] UC Davis Sch Med, Dept Pathol & Lab Med, Sacramento, CA USA
基金
美国国家卫生研究院;
关键词
Fragile X syndrome; Fragile X-associated tremor/ataxia syndrome; FXTAS; FMR1; gene; Neuropathology; MENTAL-RETARDATION PROTEIN; TREMOR/ATAXIA SYNDROME; INTRANUCLEAR INCLUSIONS; PREMUTATION CARRIERS; RAN TRANSLATION; CIRCUIT DYSFUNCTION; IRON ACCUMULATION; POTENTIAL ROLE; CGG REPEATS; MOUSE MODEL;
D O I
10.1016/j.nbd.2020.104740
中图分类号
Q189 [神经科学];
学科分类号
071006 ;
摘要
This review aims to assemble many years of research and clinical experience in the fields of neurodevelopment and neuroscience to present an up-to-date understanding of the clinical presentation, molecular and brain pathology associated with Fragile X syndrome, a neurodevelopmental condition that develops with the full mutation of the FMR1 gene, located in the q27.3 loci of the X chromosome, and Fragile X-associated tremor/ataxia syndrome a neurodegenerative disease experienced by aging premutation carriers of the FMR1 gene. It is important to understand that these two syndromes have a very distinct clinical and pathological presentation while sharing the same origin: the mutation of the FMR1 gene; revealing the complexity of expansion genetics.
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页数:8
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