Department of Medical Genetics, Centre for Molecular Medicine and Therapeutics, University of British Columbia, Vancouver, Canada.
Am J Med Genet B Neuropsychiatr Genet. 2013 Dec;162B(8):864-71. doi: 10.1002/ajmg.b.32193. Epub 2013 Aug 30.
Intermediate alleles (27-35 CAG, IAs) for Huntington disease (HD) usually do not confer the disease phenotype but are prone to CAG repeat instability. Consequently, offspring are at-risk of inheriting an expanded allele in the HD range (≥36 CAG). IAs that expand into a new mutation have been hypothesized to be more susceptible to instability compared to IAs identified on the non-HD side of a family from the general population. Frequency estimates for IAs are limited and have largely been determined using clinical samples of HD or related disorders, which may result in an ascertainment bias. This study aimed to establish the frequency of IAs in a sample of a British Columbia's (B.C.) general population with no known association to HD and examine the haplotype of new mutation and general population IAs. CAG sizing was performed on 1,600 DNA samples from B.C.'s general population. Haplotypes were determined using 22 tagging SNPs across the HTT gene. 5.8% of individuals were found to have an IA, of which 60% were on HD-associated haplotypes. There was no difference in the haplotype distribution of new mutation and general population IAs. These findings suggest that IAs are relatively frequent in the general population and are often found on haplotypes associated with expanded CAG lengths. There is likely no difference in the propensity of new mutation and general population IAs to expand into the disease range given that they are both found on disease-associated haplotypes. These findings have important implications for clinical practice.
中间等位基因(27-35CAG,IA)通常不会导致亨廷顿病(HD)表型,但容易发生 CAG 重复不稳定。因此,后代有继承 HD 范围内(≥36CAG)扩展等位基因的风险。假设与一般人群中家族的非 HD 侧鉴定的 IA 相比,扩展为新突变的 IA 更容易不稳定。IA 的频率估计值有限,并且主要是使用 HD 或相关疾病的临床样本确定的,这可能导致鉴定偏差。本研究旨在确定不列颠哥伦比亚省(BC)一般人群中无已知与 HD 相关的 IA 频率,并检查新突变和一般人群 IA 的单倍型。对来自 BC 一般人群的 1600 个 DNA 样本进行了 CAG 定序。使用 HTT 基因中的 22 个标记 SNP 确定了单倍型。发现 5.8%的个体存在 IA,其中 60%存在于与 HD 相关的单倍型上。新突变和一般人群 IA 的单倍型分布没有差异。这些发现表明,IA 在一般人群中相对常见,并且经常在与扩展 CAG 长度相关的单倍型上发现。鉴于它们都存在于疾病相关的单倍型上,新突变和一般人群 IA 扩展到疾病范围的倾向可能没有差异。这些发现对临床实践具有重要意义。