Chabrol Brigitte, Caillaud Catherine, Minassian Berge
Reference Center for Hereditary Metabolic Illnesses, Hôpital Timone Enfants, Marseilles, France.
Handb Clin Neurol. 2013;113:1701-6. doi: 10.1016/B978-0-444-59565-2.00038-1.
Neuronal ceroid lipofuscinoses (NCL) represent a group of autosomal recessive neurodegenerative disorders, presenting with myoclonic epilepsy, psychomotor delay, progressive loss of vision, and early death. Four main clinical forms have been delineated (infantile, late infantile, juvenile, and adult), but many other variants have also been described. At least 14 genetically distinct NCL, designated CLN1 to CLN14, are presently known.The identification of the deficient protein and/or the genetic defect is required for a specific diagnosis, which is necessary for a reliable genetic counseling in at-risk families.
神经元蜡样脂褐质沉积症(NCL)是一组常染色体隐性神经退行性疾病,表现为肌阵挛性癫痫、精神运动发育迟缓、进行性视力丧失和早亡。已明确四种主要临床类型(婴儿型、晚婴儿型、青少年型和成人型),但也描述了许多其他变异型。目前已知至少有14种基因不同的NCL,命名为CLN1至CLN14。特定诊断需要鉴定缺陷蛋白和/或基因缺陷,这对于有患病风险家庭进行可靠的遗传咨询是必要的。