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[成人和儿童贮积症:溶酶体、脂质和糖原贮积病]

[Adult and pediatric thesaurismosis: Lysosomal, lipid and glycogen storage diseases].

作者信息

Collardeau-Frachon Sophie

机构信息

Institut de pathologie des hospices civils de Lyon, groupement hospitalier Est, 59, boulevard Pinel, 69677 Bron cedex, France.

出版信息

Ann Pathol. 2024 Nov;44(6):432-452. doi: 10.1016/j.annpat.2024.09.010. Epub 2024 Oct 2.

DOI:10.1016/j.annpat.2024.09.010
PMID:39358197
Abstract

Thesaurismosis or storage diseases are rare genetic disorders due to an abnormal accumulation of an organic compound or its metabolite within cells. These conditions are either secondary to a defect in catabolism caused by enzymatic dysfunction or to a deficiency in transport proteins. They encompass lysosomal storage diseases, lipid storage diseases or dyslipidemias, and glycogen storage disorders or glycogenoses. Diagnosis is typically based on clinical and biological anomalies but may be made or suggested by the pathologist when symptoms are atypical or when biochemical or genetic tests are challenging to interpret. For accurate diagnosis, it is crucial to freeze a portion of the samples. Special staining and electronic microscopy can also aid in the diagnostic process. As the diagnosis is multidisciplinary, collaboration with clinicians, biochemists and geneticists is essential.

摘要

贮积症或储存疾病是罕见的遗传性疾病,由于有机化合物或其代谢产物在细胞内异常蓄积所致。这些病症要么继发于酶功能障碍引起的分解代谢缺陷,要么继发于转运蛋白缺乏。它们包括溶酶体贮积病、脂质贮积病或血脂异常,以及糖原贮积症或糖原病。诊断通常基于临床和生物学异常,但当症状不典型或生化或基因检测难以解释时,病理学家也可做出诊断或提出诊断建议。为了准确诊断,冷冻一部分样本至关重要。特殊染色和电子显微镜检查也有助于诊断过程。由于诊断是多学科的,与临床医生、生物化学家和遗传学家合作至关重要。

相似文献

1
[Adult and pediatric thesaurismosis: Lysosomal, lipid and glycogen storage diseases].[成人和儿童贮积症:溶酶体、脂质和糖原贮积病]
Ann Pathol. 2024 Nov;44(6):432-452. doi: 10.1016/j.annpat.2024.09.010. Epub 2024 Oct 2.
2
Heritable metabolic storage diseases.遗传性代谢性贮积病
J Cutan Pathol. 1985 Jun-Aug;12(3-4):348-57. doi: 10.1111/j.1600-0560.1985.tb01638.x.
3
Lysosomal storage in human skeletal muscle.人类骨骼肌中的溶酶体储存。
Hum Pathol. 1986 Jul;17(7):683-703. doi: 10.1016/s0046-8177(86)80179-4.
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[Lysosomal storage diseases].[溶酶体贮积症]
Z Rheumatol. 2010 Aug;69(6):527-38. doi: 10.1007/s00393-010-0627-z.
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Enzymatic and biochemical diagnosis of inborn lysosomal diseases with neurological symptoms.伴有神经症状的先天性溶酶体疾病的酶学和生化诊断。
Eur Neurol. 1972;7(1):101-18. doi: 10.1159/000114417.
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Methylamine accumulation in cultured cells as a measure of the aqueous storage compartment in the laboratory diagnosis of genetic lysosomal diseases.在遗传性溶酶体疾病的实验室诊断中,培养细胞中甲胺的积累作为水性储存区室的一种衡量指标。
Am J Med Genet. 1996 May 3;63(1):198-202. doi: 10.1002/(SICI)1096-8628(19960503)63:1<198::AID-AJMG35>3.0.CO;2-H.
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[Adult lysosomal and peroxisomal enzyme diseases and glycogenoses. I. Enzyme diseases with predominant symptoms in the fields of internal medicine, dermatology, orthopedics and ophthalmology].[成人溶酶体和过氧化物酶体酶病及糖原贮积病。I. 在内科、皮肤科、骨科和眼科领域以主要症状为主的酶病]
Sb Lek. 1994;95(3):221-30.
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Mass spectrometry in the study of lysosomal storage disorders.溶酶体贮积症研究中的质谱分析
Cell Mol Biol (Noisy-le-grand). 2003 Jul;49(5):769-77.
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[Adult lysosomal and peroxisomal enzyme diseases and glycogenoses. II. Enzyme diseases with predominant psychiatric and neurologic symptoms].[成人溶酶体和过氧化物酶体酶病及糖原贮积病。II. 以精神和神经症状为主的酶病]
Sb Lek. 1994;95(3):231-8.
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Fine structure and silver-staining patterns of lysosome-like bodies in absorptive cells of the small intestine in normal children and children with a lysosomal storage disease.正常儿童和患有溶酶体贮积病儿童小肠吸收细胞中类溶酶体小体的精细结构和银染模式
Virchows Arch B Cell Pathol. 1973 Jun 25;13(2):119-44. doi: 10.1007/BF02889303.

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