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连锁读取测序提供的大型神经肌肉疾病基因的单体型信息有提高诊断产量的潜力。

Haplotype information of large neuromuscular disease genes provided by linked-read sequencing has a potential to increase diagnostic yield.

机构信息

Centre for Molecular Medicine Norway (NCMM), University of Oslo, Oslo, Norway.

Institute for Molecular Medicine Finland (FIMM), HiLIFE, University of Helsinki, Helsinki, Finland.

出版信息

Sci Rep. 2024 Feb 21;14(1):4306. doi: 10.1038/s41598-024-54866-4.

Abstract

Rare or novel missense variants in large genes such as TTN and NEB are frequent in the general population, which hampers the interpretation of putative disease-causing biallelic variants in patients with sporadic neuromuscular disorders. Often, when the first initial genetic analysis is performed, the reconstructed haplotype, i.e. phasing information of the variants is missing. Segregation analysis increases the diagnostic turnaround time and is not always possible if samples from family members are lacking. To overcome this difficulty, we investigated how well the linked-read technology succeeded to phase variants in these large genes, and whether it improved the identification of structural variants. Linked-read sequencing data of nemaline myopathy, distal myopathy, and proximal myopathy patients were analyzed for phasing, single nucleotide variants, and structural variants. Variant phasing was successful in the large muscle genes studied. The longest continuous phase blocks were gained using high-quality DNA samples with long DNA fragments. Homozygosity increased the number of phase blocks, especially in exome sequencing samples lacking intronic variation. In our cohort, linked-read sequencing added more information about the structural variation but did not lead to a molecular genetic diagnosis. The linked-read technology can support the clinical diagnosis of neuromuscular and other genetic disorders.

摘要

在一般人群中,TTN 和 NEB 等大型基因中的罕见或新型错义变异很常见,这给散发性神经肌肉疾病患者中潜在致病双等位基因变异的解释带来了困难。通常,在进行第一次初始基因分析时,重建的单倍型(即变异的相位信息)缺失。如果缺乏来自家庭成员的样本,那么进行分离分析会增加诊断周转时间,而且并非总是可行。为了克服这一困难,我们研究了链接读取技术在这些大型基因中对变异进行相位分析的效果如何,以及它是否能提高结构变异的识别能力。对肌原纤维肌病、远端肌病和近端肌病患者的链接读取测序数据进行了相位分析、单核苷酸变异和结构变异分析。在所研究的大型肌肉基因中,变体相位分析是成功的。使用高质量的 DNA 样本和长 DNA 片段可以获得最长的连续相位块。纯合性增加了相位块的数量,尤其是在缺乏内含子变异的外显子测序样本中。在我们的队列中,链接读取测序增加了更多关于结构变异的信息,但并没有导致分子遗传学诊断。链接读取技术可以支持神经肌肉和其他遗传疾病的临床诊断。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/2fc0/10881483/3f32d3f229ca/41598_2024_54866_Fig1_HTML.jpg

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