Identification of a mutation in the arylsulfatase A gene of a patient with adult-type metachromatic leukodystrophy

Am J Hum Genet. 1991 May;48(5):971-8.

Abstract

To analyze the genetic abnormality in a Japanese patient with adult-type metachromatic leukodystrophy (MLD), we first elucidated the genomic organization of the human arylsulfatase A (ASA) gene and then compared the nucleotide sequences of exons and splice junctions of the mutant ASA gene to those of a normal control. We have identified a new mutation, a G-to-A transition in exon 2, which results in amino acid substitution of Asp for 99Gly. In a transient expression study, COS cells transfected with the mutant cDNA carrying 99Gly----Asp did not show an increase of ASA activity, which confirms that the mutation is a cause of adult-type MLD.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Base Sequence
  • Blotting, Western
  • Cerebroside-Sulfatase / genetics*
  • Exons*
  • Female
  • Humans
  • Introns*
  • Leukodystrophy, Metachromatic / enzymology
  • Leukodystrophy, Metachromatic / genetics*
  • Molecular Sequence Data
  • Mutation / genetics*

Substances

  • Cerebroside-Sulfatase