Aldave Anthony J, Gutmark Julie G, Yellore Vivek S, Affeldt John A, Meallet Mario A, Udar Nitin, Rao Narsing A, Small Kent W, Klintworth Gordon K
Cornea Service, The Jules Stein Eye Institute, 100 Stein Plaza, Los Angeles, CA 90095, USA.
Am J Ophthalmol. 2004 Nov;138(5):772-81. doi: 10.1016/j.ajo.2004.06.021.
To report a phenotypic variant of lattice corneal dystrophy associated with two missense changes, Ala546Asp and Pro551Gln, in the transforming growth factor-beta-induced gene (TGFBI).
Experimental study.
Genomic DNA was obtained from the proband as well as affected and unaffected family members. Exons 4, 11, 12, and 14 of the TGFBI gene were amplified and sequenced. Additionally, a corneal button excised from the proband was examined by light and transmission electron microscopy. Haplotype analysis was performed on the proband's family and members of a previously identified pedigree with the same TGFBI gene missense changes.
Bilateral, symmetric, radially arranged, branching refractile lines within and surrounding an area of central anterior stromal haze were noted in the proband. Multiple polymorphic, refractile deposits were noted in the mid and posterior stroma in both the proband and her daughter. Light and electron microscopic analyses demonstrated amyloid and excluded the presence of deposits characteristic of granular corneal dystrophy. Screening of TGFBI exon 12 in the proband and her affected daughter revealed two missense changes, Ala546Asp and Pro551Gln (both absent in 250 control chromosomes). Haplotype analysis suggested that the mutations in this family and in a previously identified pedigree reflect a founder effect, rather than an independent occurrence.
We present a phenotypic variant of lattice corneal dystrophy associated with the Ala546Asp and Pro551Gln missense changes in exon 12 of the TGFBI gene. A common ancestor appears to account for the missense mutations observed in this pedigree and in a previously reported family.
报告一种与转化生长因子β诱导基因(TGFBI)中的两个错义变化Ala546Asp和Pro551Gln相关的格子状角膜营养不良的表型变异。
实验研究。
从先证者以及受影响和未受影响的家庭成员中获取基因组DNA。对TGFBI基因的第4、11、12和14外显子进行扩增和测序。此外,对从先证者切除的角膜纽扣进行光镜和透射电镜检查。对先证者家族以及先前鉴定的具有相同TGFBI基因错义变化的家系成员进行单倍型分析。
在先证者中观察到中央前基质混浊区域内及周围有双侧、对称、放射状排列、分支状的折光性线条。先证者及其女儿的角膜基质中部和后部均可见多个多形性、折光性沉积物。光镜和电镜分析显示为淀粉样物质,排除了颗粒状角膜营养不良特征性沉积物的存在。对先证者及其受影响女儿的TGFBI第12外显子进行筛查,发现两个错义变化,Ala546Asp和Pro551Gln(在250条对照染色体中均未出现)。单倍型分析表明,该家族和先前鉴定的家系中的突变反映了奠基者效应,而非独立发生。
我们报告了一种与TGFBI基因第12外显子中的Ala546Asp和Pro551Gln错义变化相关的格子状角膜营养不良的表型变异。一个共同祖先似乎可以解释在这个家系和先前报道的一个家族中观察到的错义突变。