A mouse model of human mucopolysaccharidosis IX exhibits osteoarthritis

Hum Mol Genet. 2008 Jul 1;17(13):1904-15. doi: 10.1093/hmg/ddn088. Epub 2008 Mar 15.

Abstract

Hyaluronidases are endoglycosidases that hydrolyze hyaluronan (HA), an abundant component of the extracellular matrix of vertebrate connective tissues. Six human hyaluronidase-related genes have been identified to date. Mutations in one of these genes cause a deficiency of hyaluronidase 1 (HYAL1) resulting in a lysosomal storage disorder, mucopolysaccharidosis (MPS) IX. We have characterized a mouse model of MPS IX and compared its phenotype with the human disease. The targeted Hyal1 allele in this model had a neomycin resistance cassette in exon 2 that replaced 753 bp of the coding region containing the predicted enzyme active site. As a result, Hyal1(-/-) animals had no detectable wild-type Hyal1 transcript, protein or serum activity. Hyal1 null animals were viable, fertile and showed no gross abnormalities at 1 year and 8 months of age. Histological studies of the knee joint showed a loss of proteoglycans occurring as early as 3 months that progressed with age. An increased number of chondrocytes displaying intense pericellular and/or cytoplasmic HA staining were detected in the epiphyseal and articular cartilage of null mice, demonstrating an accumulation of HA. Elevations of HA were not detected in the serum or non-skeletal tissues, indicating that osteoarthritis is the key disease feature in a Hyal1 deficiency. Hyal3 expression was elevated in Hyal1 null mice, suggesting that Hyal3 may compensate in HA degradation in non-skeletal tissues. Overall, the murine MPS IX model displays the key features of the human disease.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Disease Models, Animal
  • Female
  • Gene Targeting
  • Glycosaminoglycans / metabolism
  • Humans
  • Hyaluronic Acid / blood
  • Hyaluronoglucosaminidase / genetics*
  • Hyaluronoglucosaminidase / metabolism*
  • Joints / pathology
  • Male
  • Mice
  • Mice, Knockout
  • Mucopolysaccharidoses / complications
  • Mucopolysaccharidoses / genetics
  • Mucopolysaccharidoses / physiopathology*
  • Osteoarthritis / complications
  • Osteoarthritis / genetics
  • Osteoarthritis / metabolism
  • Osteoarthritis / physiopathology*
  • Phenotype

Substances

  • Glycosaminoglycans
  • Hyaluronic Acid
  • Hyal1 protein, mouse
  • Hyaluronoglucosaminidase