分子分析扩展了与 GLI3 突变相关表型谱。
Molecular analysis expands the spectrum of phenotypes associated with GLI3 mutations.
机构信息
Genetic Disease Research Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, Maryland 20892-4472, USA.
出版信息
Hum Mutat. 2010 Oct;31(10):1142-54. doi: 10.1002/humu.21328.
A range of phenotypes including Greig cephalopolysyndactyly and Pallister-Hall syndromes (GCPS, PHS) are caused by pathogenic mutation of the GLI3 gene. To characterize the clinical variability of GLI3 mutations, we present a subset of a cohort of 174 probands referred for GLI3 analysis. Eighty-one probands with typical GCPS or PHS were previously reported, and we report the remaining 93 probands here. This includes 19 probands (12 mutations) who fulfilled clinical criteria for GCPS or PHS, 48 probands (16 mutations) with features of GCPS or PHS but who did not meet the clinical criteria (sub-GCPS and sub-PHS), 21 probands (6 mutations) with features of PHS or GCPS and oral-facial-digital syndrome, and 5 probands (1 mutation) with nonsyndromic polydactyly. These data support previously identified genotype-phenotype correlations and demonstrate a more variable degree of severity than previously recognized. The finding of GLI3 mutations in patients with features of oral-facial-digital syndrome supports the observation that GLI3 interacts with cilia. We conclude that the phenotypic spectrum of GLI3 mutations is broader than that encompassed by the clinical diagnostic criteria, but the genotype-phenotype correlation persists. Individuals with features of either GCPS or PHS should be screened for mutations in GLI3 even if they do not fulfill clinical criteria.
一系列表型,包括 Greig 颅面多并指综合征和 Pallister-Hall 综合征(GCPS、PHS),是由 GLI3 基因的致病性突变引起的。为了描述 GLI3 突变的临床变异性,我们展示了一个 174 名先证者队列的子集,这些先证者因 GLI3 分析而被转诊。先前已经报道了 81 名具有典型 GCPS 或 PHS 的先证者,我们在此报告其余 93 名先证者。这包括 19 名符合 GCPS 或 PHS 临床标准的先证者(12 种突变)、48 名具有 GCPS 或 PHS 特征但不符合临床标准的先证者(亚 GCPS 和亚 PHS)、21 名具有 PHS 或 GCPS 和口腔面指综合征特征的先证者,以及 5 名具有非综合征性多指的先证者(1 种突变)。这些数据支持先前确定的基因型-表型相关性,并表明严重程度比以前认识到的更为多变。在具有口腔面指综合征特征的患者中发现 GLI3 突变支持 GLI3 与纤毛相互作用的观察结果。我们得出结论,GLI3 突变的表型谱比临床诊断标准所包含的更广泛,但基因型-表型相关性仍然存在。即使不符合临床标准,具有 GCPS 或 PHS 特征的个体也应筛查 GLI3 突变。