5 RELATED LEBANESE INDIVIDUALS WITH HIGH PLASMA LYSOSOMAL HYDROLASES - A NEW DEFECT IN MANNOSE-6-PHOSPHATE RECEPTOR RECOGNITION

  • 1 January 1984
    • journal article
    • research article
    • Vol. 36  (5) , 1001-1014
Abstract
Five healthy related individuals in 3 generations of a Lebanese family have highly elevated plasma lysosomal enzyme levels inherited as a dominant Mendelian trait. The same enzymes in other extracellular fluids were within normal limits. While the pattern and extent of plasma enzyme elevation was similar to that found in mucolipidoses II and III, the physicochemical properties of the elevated enzymes were different from those of both control and I-cell disease plasma. Secretion of lysosomal hydrolases into cell media by fibroblasts from one of the individuals was increased 2-7 times more than that from controls. The results suggest faulty recognition between lysosomal hydrolases and mannose-6-phosphate receptors. This could be caused by a defect either in the phosphodiesterase that normally uncovers mannose-6-phosphate hydrolase markers or in the mannose-6-phosphate receptor itself.