Biochemistry and Molecular Genetics Department, Hospital Clinic of Barcelona, IDIBAPS (Institut de Investigacions Biomèdiques August Pi I Sunyer), 08036 Barcelona, Spain.
CIBER of Rare Diseases (CIBERER), 08036 Barcelona, Spain.
Genes (Basel). 2023 Mar 28;14(4):813. doi: 10.3390/genes14040813.
Neurological disorders (ND) are diseases that affect the brain and the central and autonomic nervous systems, such as neurodevelopmental disorders, cerebellar ataxias, Parkinson's disease, or epilepsies. Nowadays, recommendations of the American College of Medical Genetics and Genomics strongly recommend applying next generation sequencing (NGS) as a first-line test in patients with these disorders. Whole exome sequencing (WES) is widely regarded as the current technology of choice for diagnosing monogenic ND. The introduction of NGS allows for rapid and inexpensive large-scale genomic analysis and has led to enormous progress in deciphering monogenic forms of various genetic diseases. The simultaneous analysis of several potentially mutated genes improves the diagnostic process, making it faster and more efficient. The main aim of this report is to discuss the impact and advantages of the implementation of WES into the clinical diagnosis and management of ND. Therefore, we have performed a retrospective evaluation of WES application in 209 cases referred to the Department of Biochemistry and Molecular Genetics of the Hospital Clinic of Barcelona for WES sequencing derived from neurologists or clinical geneticists. In addition, we have further discussed some important facts regarding classification criteria for pathogenicity of rare variants, variants of unknown significance, deleterious variants, different clinical phenotypes, or frequency of actionable secondary findings. Different studies have shown that WES implementation establish diagnostic rate around 32% in ND and the continuous molecular diagnosis is essential to solve the remaining cases.
神经紊乱(ND)是影响大脑和中枢及自主神经系统的疾病,例如神经发育障碍、小脑共济失调、帕金森病或癫痫。如今,美国医学遗传学与基因组学学院的建议强烈推荐将下一代测序(NGS)作为此类疾病患者的一线测试方法。全外显子组测序(WES)被广泛认为是诊断单基因 ND 的当前首选技术。NGS 的引入允许快速且廉价的大规模基因组分析,并且在解析各种遗传疾病的单基因形式方面取得了巨大进展。同时分析几个潜在突变的基因可改善诊断过程,使其更快、更高效。本报告的主要目的是讨论将 WES 引入 ND 的临床诊断和管理中的影响和优势。因此,我们对在巴塞罗那临床医院生物化学和分子遗传学系进行 WES 测序的 209 例由神经科医生或临床遗传学家转诊的病例进行了回顾性评估。此外,我们还进一步讨论了一些关于罕见变异致病性、意义不明的变异、有害变异、不同临床表型或可操作性次要发现频率的分类标准的重要事实。不同的研究表明,WES 的实施在 ND 中建立了约 32%的诊断率,而持续的分子诊断对于解决剩余病例至关重要。