Jatzkewitz H, Sandhoff K
Arch Psychiatr Nervenkr (1970). 1976 Apr 30;221(3):213-25. doi: 10.1007/BF00418481.
A short survey on the sphingolipid storage diseases is presented. The chemical nature of the accumulated substances is related to the genetically induced enzymic blocks on their biodegradation. Two disorders are stressed with alter the nervous system: metachromatic leukodystrophy and familiar infantile amaurotic idiocy (GM2-gangliosidosis). The difficulties in the causal interpretation of three variants of the latter disease due to the involvement of isoenzymes are dealt with. The relationship between the enzyme defect in these disorders and their time of clinical onset is discussed. Finally, the diagnostic possibilities are presented which are a prerequisite for preventing a further dissemination of these therapy-resistent inborn errors of metabolism.
本文对鞘脂贮积病进行了简要综述。所积累物质的化学性质与它们生物降解过程中基因诱导的酶阻断有关。重点介绍了两种影响神经系统的疾病:异染性脑白质营养不良和家族性婴儿黑蒙性白痴(GM2神经节苷脂沉积症)。探讨了由于同工酶的参与,后一种疾病三种变体在病因解释上的困难。讨论了这些疾病中酶缺陷与其临床发病时间的关系。最后,介绍了诊断方法,这是防止这些抗治疗的先天性代谢错误进一步传播的先决条件。