At birth, mice that are homozygous null for the <i>Idua</i> gene are viable, fertile, normal in size and do not display any gross physical or behavioral abnormalities. No alpha-L-iduronidase enzyme activity or mRNA is detected. By three weeks of age, homozygous null mice develop a flattened facial profile and a thickening of digits. Defective bone formation is noticeable by fifteen weeks, characterized by a broadening and thickening of long bones. Evidence of lysosomal storage disorder is apparent in cells of the reticuloendothelial system at four weeks. By eight weeks progressive evidence of lysosomal storage is seen in Kupffer cells, splenic sinusoidal lining cells, chondrocytes, glial and Purkinje cells. This animal model is suitable for use in studies investigating the lysosomal storage disorder mucopolysaccharidosis type I (MPS I).

These NOD/SCID <i> Idua </i> knock-out mice exhibit abnormal development of facial profile, defective bone formation, and lysosomal storage disorder and will be useful for studies looking into the ability of human marrow stem cells (especially bone marrow cells) transduced with gene therapy vectors to repopulate and alter phenotype such as for Hurler syndrome patients.




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