Department of Biomedical Engineering, Marshall University, Huntington, WV 25755, USA.
Department of Genetics, Yale University School of Medicine, New Haven, CT 06519, USA.
Exp Neurol. 2021 Jan;335:113523. doi: 10.1016/j.expneurol.2020.113523. Epub 2020 Nov 4.
Congenital hydrocephalus (CH) is caused by genetic mutations, but whether factors impacting human genetic mutations are disease-specific remains elusive. Given two factors associated with high mutation rates, we reviewed how many disease-susceptible genes match with (i) proximity to telomeres or (ii) high adenine and thymine (A + T) content in human CH as compared to other disorders of the central nervous system (CNS). We extracted genomic information using a genome data viewer. Importantly, 98 of 108 genes causing CH satisfied (i) or (ii), resulting in >90% matching rate. However, such a high accordance no longer sustained as we checked two factors in Alzheimer's disease (AD) and/or familial Parkinson's disease (fPD), resulting in 84% and 59% matching, respectively. A disease-specific matching of telomere proximity or high A + T content predicts causative genes of CH much better than neurodegenerative diseases and other CNS conditions, likely due to sufficient number of known causative genes (n = 108) and precise determination and classification of the genotype and phenotype. Our analysis suggests a need for identifying genetic basis of both factors before human clinical studies, to prioritize putative genes found in preclinical models into the likely (meeting at least one) and more likely candidate (meeting both), which predisposes human genes to mutations.
先天性脑积水 (CH) 是由基因突变引起的,但影响人类基因突变的因素是否具有疾病特异性仍不清楚。鉴于与高突变率相关的两个因素,我们回顾了有多少疾病易感基因与 (i) 端粒的接近程度或 (ii) 在人类 CH 中与其他中枢神经系统 (CNS) 疾病相比,腺嘌呤和胸腺嘧啶 (A+T) 含量较高相匹配。我们使用基因组数据查看器提取了基因组信息。重要的是,导致 CH 的 108 个基因中有 98 个满足 (i) 或 (ii),匹配率超过 90%。然而,当我们检查阿尔茨海默病 (AD) 和/或家族性帕金森病 (fPD) 中的两个因素时,这种高度的一致性不再维持,分别导致 84%和 59%的匹配。端粒接近或高 A+T 含量的疾病特异性匹配预测 CH 的致病基因要好于神经退行性疾病和其他 CNS 疾病,这可能是由于已知致病基因的数量足够多(n=108),以及基因型和表型的精确确定和分类。我们的分析表明,在进行人类临床研究之前,需要确定这两个因素的遗传基础,以便将临床前模型中发现的假定基因优先划分为可能(至少满足一个)和更可能的候选基因(均满足),这使人类基因更容易发生突变。