Karageorgos L, Hill B, Bawden M J, Hopwood J J
Lysosomal Diseases Research Unit, Department of Genetic Medicine, Children, Youth and Women's Health Service, 72 King William Road, North Adelaide, 5006, South Australia, Australia.
J Inherit Metab Dis. 2007 Jun;30(3):358-64. doi: 10.1007/s10545-007-0539-5. Epub 2007 Apr 24.
Mucopolysaccharidosis IIIB, an autosomal recessive lysosomal storage disorder of heparan sulfate caused by mutations in the alpha-N-acetylglucosaminidase (NAGLU) gene, was recently discovered in cattle. Clinical signs include progressive ataxia, stumbling gait, swaying and difficulty in balance and walking. These clinical signs are usually first observed at approximately 2 years of age and then develop progressively over the lifespan of the animals. Affected bulls were found to be homozygous for the missense mutation E452K (c.1354G > A). The availability of mutational analysis permits screening for the NAGLU mutation to eradicate this mutation from the cattle breeding population.
粘多糖贮积症IIIB型是一种常染色体隐性溶酶体贮积症,由α-N-乙酰氨基葡萄糖苷酶(NAGLU)基因突变引起,导致硫酸乙酰肝素贮积,最近在牛身上被发现。临床症状包括进行性共济失调、蹒跚步态、摇摆以及平衡和行走困难。这些临床症状通常在大约2岁时首次出现,然后在动物的生命周期中逐渐发展。受影响的公牛被发现对于错义突变E452K(c.1354G > A)是纯合子。突变分析的可行性使得能够对NAGLU突变进行筛查,从而从牛繁殖群体中消除这种突变。