Giebel L B, Tripathi R K, Strunk K M, Hanifin J M, Jackson C E, King R A, Spritz R A
Department of Medical Genetics, University of Wisconsin, Madison 53706.
Am J Hum Genet. 1991 Jun;48(6):1159-67.
We have identified three different tyrosinase gene mutant alleles in four unrelated patients with type IB ("yellow") oculocutaneous albinism (OCA) and thus have demonstrated that type IB OCA is allelic to type IA (tyrosinase negative) OCA. In an inbred Amish kindred, type IB OCA results from homozygosity for a Pro----Leu substitution at codon 406. In the second family, type IB OCA results from compound heterozygosity for a type IA OCA allele (codon 81 Pro----Leu) and a novel type IB allele (codon 275 Val----Phe). In the third patient, type IB OCA results from compound heterozygosity for the same type IB allele (codon 275 Val----Phe) and a novel type IB OCA allele. In a fourth patient, type IB OCA results from compound heterozygosity for the codon 81 type IA OCA allele and a type IB allele that contains no identifiable abnormalities; dysfunction of this type IB allele apparently results from a mutation either well within one of the large introns or at some distance from the tyrosinase gene. In vitro expression of the Amish type IB allele in nonpigmented HeLa cells demonstrates that the Pro----Leu substitution at codon 406 greatly reduces but does not abolish tyrosinase enzymatic activity, a finding consistent with the clinical phenotype.
我们在4名患IB型(“黄色”)眼皮肤白化病(OCA)的非亲缘患者中鉴定出3种不同的酪氨酸酶基因突变等位基因,从而证明IB型OCA与IA型(酪氨酸酶阴性)OCA是等位基因。在一个近亲结婚的阿米什家族中,IB型OCA是由第406密码子发生脯氨酸→亮氨酸替换的纯合子所致。在第二个家族中,IB型OCA是由一个IA型OCA等位基因(第81密码子脯氨酸→亮氨酸)和一个新的IB型等位基因(第275密码子缬氨酸→苯丙氨酸)的复合杂合子所致。在第三名患者中,IB型OCA是由同一个IB型等位基因(第275密码子缬氨酸→苯丙氨酸)和一个新的IB型OCA等位基因的复合杂合子所致。在第四名患者中,IB型OCA是由第81密码子的IA型OCA等位基因和一个无明显异常的IB型等位基因的复合杂合子所致;这个IB型等位基因的功能障碍显然是由一个大内含子内的突变或距酪氨酸酶基因有一段距离的突变引起的。在无色素的HeLa细胞中对阿米什家族的IB型等位基因进行体外表达,结果表明第406密码子的脯氨酸→亮氨酸替换极大地降低了酪氨酸酶的酶活性,但并未使其完全丧失,这一发现与临床表型相符。