Collardeau-Frachon Sophie
Institut de pathologie des hospices civils de Lyon, groupement hospitalier Est, 59, boulevard Pinel, 69677 Bron cedex, France.
Ann Pathol. 2024 Nov;44(6):432-452. doi: 10.1016/j.annpat.2024.09.010. Epub 2024 Oct 2.
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.
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。