Mucopolysaccharidosis type IVA: common double deletion in the N-acetylgalactosamine-6-sulfatase gene (GALNS)

Genomics. 1995 Apr 10;26(3):535-42. doi: 10.1016/0888-7543(95)80172-i.

Abstract

Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive disorder caused by a deficiency in N-acetylgalactosamine-6-sulfatase (GALNS). We found two separate deletions of nearly 8.0 and 6.0 kb in the GALNS gene, including some exons. There are Alu repetitive elements near the breakpoints of the 8.0-kb deletion, and this deletion resulted from an Alu-Alu recombination. The other 6.0-kb deletion involved illegitimate recombinational events between incomplete short direct repeats of 8 bp at deletion breakpoints. The same rearrangement has been observed in a heteroallelic state in four unrelated patients. This is the first documentation of a common double deletion a gene that is not a member of a gene cluster.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Base Sequence
  • Blotting, Southern
  • Chondroitinsulfatases / genetics*
  • DNA
  • Humans
  • Molecular Sequence Data
  • Mucopolysaccharidosis IV / genetics*
  • Sequence Deletion*

Substances

  • DNA
  • Chondroitinsulfatases

Associated data

  • GENBANK/D45223
  • GENBANK/D45224