Fabry disease: diagnosis by alpha-galactosidase activities in tears. 1975

D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick

The enzymatic diagnosis of hemizygotes with Fabry disease and heterozygous carriers was accomplished by the fluorometric determination of alpha-galactosidase activities in tears. Two components of total alpha-galactosidase activity were differentiated by their relative thermostabilities and by chromatography on DEAE-cellulose. The major component, alpha-galactosidase A, was thermolabile and represented approximately 90% of total activity; the remaining activity was thermostable, eluted at a slightly higher salt concentration and was designated alpha-galactosidase B. A single, symmetric pH optimum was observed for total alpha-galactosidase activities from heterozygotes and normal individuals, whereas the total activity from hemizgotes, which was about 10% of that in normal controls, had a broad pH profile, identical to those for alpha-galactosidase B activities from all individuals studied. The apparent Km values for total activities were 3.2, 4.0, and greater than 13 mM for normal individuals, heterozygotes, and hemizygotes, respectively. In contrast, apparent Km values for alpha-galactosidase B activities were greater than 13 mM for all individuals, further suggesteng that the residual activity in hemizygotes with Fabry disease represented the alpha-galactosidase B component. of the potential inhibitors studied, alpha-D-melibiose was found to competitively inhibit total alpha-galactosidase activity (Ki approximately 10 mM). These studies demonstrate that tears provide an easily obtainable source of freshly secreted enzyme for the diagnosis of hemizygotes and heterozygotes with Fabry disease and suggest that tears may be useful for the diagnosis of other inborn errors of metabolism.

UI MeSH Term Description Entries
D007700 Kinetics The rate dynamics in chemical or physical systems.
D008297 Male Males
D004355 Drug Stability The chemical and physical integrity of a pharmaceutical product. Drug Shelf Life,Drugs Shelf Lives,Shelf Life, Drugs,Drug Stabilities,Drugs Shelf Life,Drugs Shelf Live,Life, Drugs Shelf,Shelf Life, Drug,Shelf Live, Drugs,Shelf Lives, Drugs
D005260 Female Females
D005696 Galactosidases A family of galactoside hydrolases that hydrolyze compounds with an O-galactosyl linkage. EC 3.2.1.-. Galactosidase
D006579 Heterozygote An individual having different alleles at one or more loci regarding a specific character. Carriers, Genetic,Genetic Carriers,Carrier, Genetic,Genetic Carrier,Heterozygotes
D006596 Hexosaminidases Enzymes that catalyze the hydrolysis of N-acylhexosamine residues in N-acylhexosamides. Hexosaminidases also act on GLUCOSIDES; GALACTOSIDES; and several OLIGOSACCHARIDES. Galactosaminidases,Hexosaminidase,Galactosaminidase,Glucosaminidase,Glucosaminidases
D006720 Homozygote An individual in which both alleles at a given locus are identical. Homozygotes
D006801 Humans Members of the species Homo sapiens. Homo sapiens,Man (Taxonomy),Human,Man, Modern,Modern Man
D006863 Hydrogen-Ion Concentration The normality of a solution with respect to HYDROGEN ions; H+. It is related to acidity measurements in most cases by pH pH,Concentration, Hydrogen-Ion,Concentrations, Hydrogen-Ion,Hydrogen Ion Concentration,Hydrogen-Ion Concentrations

Related Publications

D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
January 1998, Ryoikibetsu shokogun shirizu,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
December 1995, Nihon rinsho. Japanese journal of clinical medicine,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
November 2004, Clinical chemistry,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
February 1997, Rinsho byori. The Japanese journal of clinical pathology,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
January 2007, Clinical therapeutics,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
June 1994, The Journal of the Kentucky Medical Association,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
January 2005, Clinical chemistry and laboratory medicine,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
May 2016, Orphanet journal of rare diseases,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
May 1991, International journal of dermatology,
D L Johnson, and M A Del Monte, and E Cotlier, and R J Desnick
March 1997, Proceedings of the National Academy of Sciences of the United States of America,
Copied contents to your clipboard!