Waisman Center, University of Wisconsin-Madison, Madison, WI, 53705, USA.
Department of Biostatistics and Medical Informatics, University of Wisconsin-Madison, Madison, WI, 53706, USA.
J Neurodev Disord. 2022 May 2;14(1):28. doi: 10.1186/s11689-022-09438-w.
Intellectual and Developmental Disabilities (IDDs), such as Down syndrome, Fragile X syndrome, Rett syndrome, and autism spectrum disorder, usually manifest at birth or early childhood. IDDs are characterized by significant impairment in intellectual and adaptive functioning, and both genetic and environmental factors underpin IDD biology. Molecular and genetic stratification of IDDs remain challenging mainly due to overlapping factors and comorbidity. Advances in high throughput sequencing, imaging, and tools to record behavioral data at scale have greatly enhanced our understanding of the molecular, cellular, structural, and environmental basis of some IDDs. Fueled by the "big data" revolution, artificial intelligence (AI) and machine learning (ML) technologies have brought a whole new paradigm shift in computational biology. Evidently, the ML-driven approach to clinical diagnoses has the potential to augment classical methods that use symptoms and external observations, hoping to push the personalized treatment plan forward. Therefore, integrative analyses and applications of ML technology have a direct bearing on discoveries in IDDs. The application of ML to IDDs can potentially improve screening and early diagnosis, advance our understanding of the complexity of comorbidity, and accelerate the identification of biomarkers for clinical research and drug development. For more than five decades, the IDDRC network has supported a nexus of investigators at centers across the USA, all striving to understand the interplay between various factors underlying IDDs. In this review, we introduced fast-increasing multi-modal data types, highlighted example studies that employed ML technologies to illuminate factors and biological mechanisms underlying IDDs, as well as recent advances in ML technologies and their applications to IDDs and other neurological diseases. We discussed various molecular, clinical, and environmental data collection modes, including genetic, imaging, phenotypical, and behavioral data types, along with multiple repositories that store and share such data. Furthermore, we outlined some fundamental concepts of machine learning algorithms and presented our opinion on specific gaps that will need to be filled to accomplish, for example, reliable implementation of ML-based diagnosis technology in IDD clinics. We anticipate that this review will guide researchers to formulate AI and ML-based approaches to investigate IDDs and related conditions.
智力和发育障碍(IDDs),如唐氏综合征、脆性 X 综合征、雷特综合征和自闭症谱系障碍,通常在出生或幼儿期表现出来。IDDs 的特点是智力和适应功能严重受损,遗传和环境因素是 IDD 生物学的基础。由于重叠因素和共病,对 IDDs 进行分子和遗传分层仍然具有挑战性。高通量测序、成像和大规模记录行为数据的工具的进步极大地提高了我们对一些 IDDs 的分子、细胞、结构和环境基础的理解。在“大数据”革命的推动下,人工智能(AI)和机器学习(ML)技术在计算生物学领域带来了全新的范式转变。显然,基于 ML 的临床诊断方法有可能增强使用症状和外部观察的经典方法,希望推动个性化治疗方案向前发展。因此,ML 技术的综合分析和应用与 IDDs 的发现直接相关。将 ML 应用于 IDDs 有可能改善筛查和早期诊断,加深我们对共病复杂性的理解,并加速鉴定用于临床研究和药物开发的生物标志物。五十多年来,IDDRC 网络支持了美国各地中心的研究人员网络,他们都在努力理解 IDD 背后各种因素的相互作用。在这篇综述中,我们介绍了快速增长的多模态数据类型,强调了使用 ML 技术阐明 IDD 相关因素和生物学机制的示例研究,以及 ML 技术的最新进展及其在 IDD 和其他神经疾病中的应用。我们讨论了各种分子、临床和环境数据收集模式,包括遗传、成像、表型和行为数据类型,以及存储和共享此类数据的多个存储库。此外,我们概述了机器学习算法的一些基本概念,并就需要填补的具体差距提出了我们的看法,例如,在 IDD 诊所中可靠地实施基于 ML 的诊断技术。我们预计,本综述将指导研究人员制定基于 AI 和 ML 的方法来研究 IDD 和相关疾病。