LYSOSOMAL CYSTINE COUNTER-TRANSPORT IN HETEROZYGOTES FOR CYSTINOSIS

  • 1 January 1984
    • journal article
    • research article
    • Vol. 36  (2) , 277-282
Abstract
Heterozygotes for cystinosis exhibited approximately half the normal rate of Cys counter-transport into isolated leukocyte lysosomes. This gene-dosage effect strongly supports previous findings demonstrating that the basic defect in cystinosis is impaired Cys transport across the lysosomal membrane. The method was used to determine the cystinosis carrier status for siblings of affected children in 2 families with cystinosis.