
浏览全部资源
扫码关注微信
四川大学华西第二医院儿科
纸质出版日期:2017,
扫 描 看 全 文
鄢力, 杨凡. 2例ACADM基因复合杂合突变所致的中链酰基辅酶A脱氢酶缺乏症[J]. 重庆医科大学学报, 2017,42(5):601-605.
Yan Li, Yang Fan. Two cases of medium chain acyl-Co A dehydrogenase deficiency caused by ACADM gene compound heterozygous mutations[J]. Journal of Chongqing Medical University, 2017,42(5):601-605.
鄢力, 杨凡. 2例ACADM基因复合杂合突变所致的中链酰基辅酶A脱氢酶缺乏症[J]. 重庆医科大学学报, 2017,42(5):601-605. DOI: 10.13406/j.cnki.cyxb.001223.
Yan Li, Yang Fan. Two cases of medium chain acyl-Co A dehydrogenase deficiency caused by ACADM gene compound heterozygous mutations[J]. Journal of Chongqing Medical University, 2017,42(5):601-605. DOI: 10.13406/j.cnki.cyxb.001223.
目的:对2例生后串联质谱遗传代谢病检测发现疑似中链酰基辅酶A脱氢酶缺乏症的患儿进行临床和基因检测
并进行随访
以明确诊断。方法:采集患儿外周血检测遗传代谢病代谢产物、肝功、采集尿检测尿有机酸
采集患儿及其父母的外周血进行高通量测序和Sanger测序验证中链酰基辅酶A脱氢酶基因ACADM
并对基因异常进行分析。结果:(1)外周血串联质谱LC-MS检测发现患儿中链脂酰肉碱(C6
C
10)升高;(2)肝功能均出现异常;(3)尿有机酸检测其中一例患者甘油和双羧酸略有升高
另一例未见异常;(4)基因检测显示患儿均为ACADM基因复合杂合子
基因异常分别来至无症状的单突变携带者父亲和母亲
复合杂合突变导致合成的中链酰基辅酶A脱氢酶活性异常。结论:ACADM基因复合突变导致合成中链酰基辅酶A脱氢酶活性降低
患儿分解中链脂肪酸能力下降
导致代谢产物异常和肝功能异常。
Objective:To perform clinical and genetic testing and follow-up on two neonates to confirm the diagnosis of suspected medium chain acyl-coenzyme A dehydrogenase deficiency(MCADD)by newborn screening using tandem mass spectrometry(MS/MS).Methods:Acylcarnitine profile was measured by liquid chromatography tandem mass spectrometry(LC-MS/MS)in peripheral blood and liver function. Urinary organic acid was tested by gas chromatography/mass spectrometry(GS/MS). Medium chain acyl-Co A dehydrogenase gene(ACADM)of both patients and their parents was tested by high-throughput sequencing and Sanger sequencing.Results:(1)Both children’s acyl carnitine(C6-C10)was elevated.(2)Liver function was abnormal.(3)One patient’s glycerin and carboxy acid increased slightly
while the other’s showed normal.(4)Both children’s ACADM gene was compound heterozygotes. The genetic abnormality was inherited from the father and mother respectively
who were asymptomatic single mutant carriers. The compound heterozygous mutations resulted in abnormal activity of ACADM. Conclusion:The compound mutations of ACADM gene cause the decrease of MCAD activity and the patients ’abilities of decomposing the medium-chain fatty acid and then the abnormality of metabolites and liver fuction.
中链酰基辅酶A脱氢酶缺乏症中链酰基辅酶A脱氢酶基因复合杂合突变
medium chain acyl-Co A dehydrogenase deficiencymedium chain acyl-Co A dehydrogenase genecompound heterozygous mutations
0
浏览量
210
下载量
1
CSCD
关联资源
相关文章
相关作者
相关机构
京公网安备11010802024621