Demina A, Boas E, Beutler E
Scripps Research Institute, Department of Molecular and Experimental Medicine, La Jolla, California 92037, USA.
Hematopathol Mol Hematol. 1998;11(2):63-71.
Both the L-type pyruvate kinase gene (PKLR) and glucocerebrosidase (GBA) gene are on band q21 of chromosome 1 in humans. Two overlapping P1 bacteriophage clones containing PKLR and GBA were identified and mapped, defining the locations of these two genes as well as those of the GBA pseudogene (psi GBA) metaxin (MTX), the MTX pseudogene (psi MTX), and thrombospondin 3 (THBS3). The distance between the 5' ends of GBA and PKLR was determined to be 71 kb. The direction of transcription PKLR gene was convergent to that of the GBA gene. All 195 Gaucher disease patients homozygous for the 1226G mutation, representing 390 chromosomes with the 1226G mutation, had a PvuII -/- GBA haplotype and a C/C at nt 1705 of the PKLR gene (-/- haplotype). All 56 Gaucher disease patients who were 1226G/84GG compound heterozygotes manifested a -/+ GBA haplotype and 55 of 56 patients were -/+ at PKLR nt 1705. Only 1 patient with 1226G/84GG genotype showed a crossover with the PKLR polymorphism, with a -/- haplotype at nt 1705. Similarly, 9 patients deficient in pyruvate kinase with the PKLR 1529A/1529A genotype were all found to have the same -/- GBA haplotype.
L型丙酮酸激酶基因(PKLR)和葡糖脑苷脂酶(GBA)基因都位于人类1号染色体的q21带。鉴定并定位了两个包含PKLR和GBA的重叠P1噬菌体克隆,确定了这两个基因以及GBA假基因(psi GBA)、介连蛋白(MTX)、MTX假基因(psi MTX)和血小板反应蛋白3(THBS3)的位置。GBA和PKLR的5'端之间的距离确定为71 kb。PKLR基因的转录方向与GBA基因的转录方向相反。所有195例1226G突变纯合的戈谢病患者,代表390条带有1226G突变的染色体,都具有PvuII -/- GBA单倍型和PKLR基因第1705位核苷酸处的C/C(-/-单倍型)。所有56例1226G/84GG复合杂合子的戈谢病患者均表现为-/+ GBA单倍型,56例患者中有55例在PKLR第1705位核苷酸处为-/+。只有1例1226G/84GG基因型患者表现出与PKLR多态性的交叉,在第1705位核苷酸处为-/-单倍型。同样,9例PKLR 1529A/1529A基因型的丙酮酸激酶缺乏患者均被发现具有相同的-/- GBA单倍型。