Blood dolichol in lysosomal diseases

Abstract
Highly elevated serum total dolichol (free dolichol + dolichyl ester) concentrations have recently been found in two lysosomal storage diseases, aspartylglucosaminuria (AGU) and mannosidosis. The present study demonstrates that the increase of serum dolichol in AGU patients is caused by an increase of serum free dolichol. In 15 patients the mean serum level of free dolichol (227 ± 16 ng/mL) was 1.9 times higher (p < 0.001) than that in healthy controls (120 ± 6 ng/mL), while the amounts of dolichol fatty acid esters were similar in the patients and controls (110 ± 9 vs. 118 ± 6 ng/mL). In contrast, 10 patients with neuronal ceroid-lipofuscinosis (NCL) (three with infantile, four with juvenile, and three with variant late infantile NCL) had significantly (p < 0.01) lower mean serum levels of both free (79 ± 5 ng/mL) and total (159 ± 6 ng/mL) dolichol than age-adjusted healthy controls (free, 100 ± 6 ng/mL; total, 206 ± 14 ng/mL). Decreased blood dolichol has not been reported earlier for any other disease. We conclude that the increased serum free dolichol in AGU reflects disturbed lysosomal function and that the decreased free and esterified dolichols in NCLs speak against their presumed primary lysosomal nature.Key words: serum dolichol, aspartylglucosaminuria, neuronal ceroid-lipofuscinosis, lysosomal function, lysosomal storage disease.