Varga Matthew J, Richardson Marcy E, Chamberlin Adam
Ambry Genetics, Aliso Viejo, CA, USA.
Ambry Genetics, Aliso Viejo, CA, USA.
Am J Hum Genet. 2025 May 1;112(5):984-992. doi: 10.1016/j.ajhg.2025.03.010. Epub 2025 Apr 14.
Structural biology offers a powerful lens through which to assess genetic variants by providing insights into their impact on clinically relevant protein structure and function. Due to the availability of new, user-friendly, web-based tools, structural analyses by wider audiences have become more mainstream. These new tools, including AlphaMissense and AlphaFold, have recently been in the limelight due to their initial success and projected future promise; however, the intricacies and limitations of using these tools still need to be disseminated to the more general audience that is likely to use them in variant analysis. Here, we expound on frameworks applying structural biology to variant interpretation by examining two accompanying articles. To this end, we explore the nuances of choosing the correct protein model, compare and contrast various structural approaches, and highlight both the advantages and limitations of employing structural biology in variant interpretation. Using two articles published in this issue of The American Journal of Human Genetics as a baseline, we focus on case studies in TP53 and BRCA1 to illuminate gene-specific differences in the applications of structural information, which illustrate the complexities inherent in this field. Additionally, we discuss the implications of recent advancements, such as AlphaFold, and provide practical guidance for researchers navigating variant interpretation using structural biology.
结构生物学提供了一个强大的视角,通过深入了解基因变异对临床相关蛋白质结构和功能的影响来评估这些变异。由于新的、用户友好的基于网络的工具的出现,更广泛的受众进行结构分析已变得更加主流。这些新工具,包括AlphaMissense和AlphaFold,由于其初步成功和预期的未来前景,最近备受关注;然而,使用这些工具的复杂性和局限性仍需要向更可能在变异分析中使用它们的普通受众进行传播。在此,我们通过审视两篇配套文章阐述了将结构生物学应用于变异解读的框架。为此,我们探讨选择正确蛋白质模型的细微差别,比较和对比各种结构方法,并强调在变异解读中采用结构生物学的优势和局限性。以本期《美国人类遗传学杂志》发表 的两篇文章为基线,我们重点关注TP53和BRCA1的案例研究,以阐明结构信息应用中的基因特异性差异,这说明了该领域固有的复杂性。此外,我们讨论了诸如AlphaFold等最新进展的影响,并为使用结构生物学进行变异解读的研究人员提供实用指导。