Mizukami Keijiro, Raj Karthik, Osborne Carl, Giger Urs
Section of Medical Genetics, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA, United States of America.
Veterinary Clinical Sciences, College of Veterinary Medicine, University of Minnesota, Saint Paul, MN, United States of America.
PLoS One. 2016 Jul 12;11(7):e0159247. doi: 10.1371/journal.pone.0159247. eCollection 2016.
Cystinuria is a classical inborn error of metabolism characterized by a selective proximal renal tubular defect affecting cystine, ornithine, lysine, and arginine (COLA) reabsorption, which can lead to uroliths and urinary obstruction. In humans, dogs and mice, cystinuria is caused by variants in one of two genes, SLC3A1 and SLC7A9, which encode the rBAT and bo,+AT subunits of the bo,+ basic amino acid transporter system, respectively. In this study, exons and flanking regions of the SLC3A1 and SLC7A9 genes were sequenced from genomic DNA of cats (Felis catus) with COLAuria and cystine calculi. Relative to the Felis catus-6.2 reference genome sequence, DNA sequences from these affected cats revealed 3 unique homozygous SLC7A9 missense variants: one in exon 5 (p.Asp236Asn) from a non-purpose-bred medium-haired cat, one in exon 7 (p.Val294Glu) in a Maine Coon and a Sphinx cat, and one in exon 10 (p.Thr392Met) from a non-purpose-bred long-haired cat. A genotyping assay subsequently identified another cystinuric domestic medium-haired cat that was homozygous for the variant originally identified in the purebred cats. These missense variants result in deleterious amino acid substitutions of highly conserved residues in the bo,+AT protein. A limited population survey supported that the variants found were likely causative. The remaining 2 sequenced domestic short-haired cats had a heterozygous variant at a splice donor site in intron 10 and a homozygous single nucleotide variant at a branchpoint in intron 11 of SLC7A9, respectively. This study identifies the first SLC7A9 variants causing feline cystinuria and reveals that, as in humans and dogs, this disease is genetically heterogeneous in cats.
胱氨酸尿症是一种典型的先天性代谢缺陷病,其特征为选择性近端肾小管缺陷,影响胱氨酸、鸟氨酸、赖氨酸和精氨酸(COLA)的重吸收,可导致尿路结石和尿路梗阻。在人类、犬类和小鼠中,胱氨酸尿症由两个基因之一的变异引起,即SLC3A1和SLC7A9,它们分别编码bo,+碱性氨基酸转运系统的rBAT和bo,+AT亚基。在本研究中,从患有COLAuria和胱氨酸结石的猫(家猫)的基因组DNA中对SLC3A1和SLC7A9基因的外显子及侧翼区域进行了测序。相对于家猫-6.2参考基因组序列,这些患病猫的DNA序列显示出3种独特的纯合SLC7A9错义变异:一种来自一只非定向培育的中毛猫的外显子5(p.Asp236Asn),一种来自缅因库恩猫和一只斯芬克斯猫的外显子7(p.Val294Glu),以及一种来自一只非定向培育的长毛猫的外显子10(p.Thr392Met)。随后的基因分型检测又鉴定出另一只患有胱氨酸尿症的家养中毛猫,它对于最初在纯种猫中鉴定出的变异是纯合的。这些错义变异导致bo,+AT蛋白中高度保守残基的有害氨基酸替代。有限的群体调查支持所发现的变异可能是致病的。其余2只测序的家养短毛猫分别在SLC7A9基因内含子10的剪接供体位点有一个杂合变异,在内含子11的分支点有一个纯合单核苷酸变异。本研究鉴定出了首个导致猫胱氨酸尿症的SLC7A9变异,并揭示出,与人类和犬类一样,这种疾病在猫中也是基因异质性的。