UCLA IDDRC: Translational Core

临床翻译核心：摘要 加州大学洛杉矶分校IDDRC的转化临床核心的使命，成立于2015年，是支持高质量， 多学科，协作临床转化研究，推进病因，预防， 诊断和治疗智力和发育残疾。为了实现这一目标，核心将 提供研究咨询;获得降低成本，专业化，研究质量的诊断和表型 评估;招聘支持;培训;数据管理;以及其他协助IDDRC调查人员沟通的工具 临床研究与基础科学。该中心致力于改进缺碘症研究的评估方法 通过创新措施的制定、评估和传播。核心还支持外联， 生物样本库和数据管理。此外，核心将参与IDDRC网络和其他 IDDRC相关的多中心临床研究。 在过去4年中，核心小组成功地建立了有效的程序，以支持正在进行的 和新的IDD临床研究，在这段时间内支持的数量几乎翻了一番， 新的表型分析方法核心在支持几项新研究方面发挥了重要作用， 刚进入临床研究的早期职业研究者。在这次更新中，我们通过增加两个 新的核心合作研究者（Catherine Lord博士和Rujuta Bhatt Wilson博士），他们带来了社会， 诊断、行为、运动和神经评估，IDD干预试验，纵向研究，以及 大规模培训金标准诊断仪器。利用核心支持， 成本效率，降低单个项目成本，消除培训、质量控制和 保持个体研究的可靠性。核心访问然后能够扩大研究工作， 通过建立诊断、表型和行为的统一性，提高了加州大学洛杉矶分校IDDRC研究的质量。 临床研究。测量开发工作，利用复杂的分析，如项目响应 理论和机器学习旨在解决困扰先前临床研究的方法学差距。 与IDDs相关的研究工作，并通过提供更适合于捕获临床 潜在生物学的表现，从而推进临床翻译。我们的培训旨在培养能力， 缺碘症研究。我们的团队特别适合于推进评估方法，用于最低限度地 语言障碍者和有患IDDs风险的幼儿，这是我们团队的优势。核心调查人员 以测量和范式发展、寿命研究和更高功能主体的研究而闻名 一系列IDDs。

Clinical Translational Core: Abstract The mission of the Translational Clinical Core of the UCLA IDDRC, established in 2015, is to support high quality, multidisciplinary, collaborative clinical translational research advancing knowledge of causes, prevention, diagnosis, and treatment of intellectual and developmental disabilities (IDDs). To accomplish this, the Core will provide research consultation; access to reduced cost, specialized, research quality diagnostic and phenotypic assessments; recruitment support; training; data management; and other aids for IDDRC investigators bridging clinical research with basic science. The Core is engaged in advancing assessment methods for IDD research through innovative measure development, evaluation, and dissemination. The Core also supports outreach, biospecimen banking, and data management. In addition, the Core will participate in IDDRC Network and other IDDRC-related multi-site clinical investigations. Over the past 4 years, the Core has successfully established effective procedures for support of ongoing and new clinical IDD studies, nearly doubling the number supported during that timeframe, and has contributed new phenotyping measurements to the field. The Core has been impactful in supporting several new studies by early career investigators new to clinical studies. In this renewal, we are strengthening the Core by adding two new Core Co-Investigators (Drs. Catherine Lord and Rujuta Bhatt Wilson) who bring special expertise in social, diagnostic, behavioral, motor, and neurological assessments, IDD intervention trials, longitudinal studies, and large-scale training on gold standard diagnostic instruments. Core support is leveraged to achieve substantial cost efficiency, reducing individual project costs and by removing burdens of training, quality control, and maintenance of reliability from individual studies. Core access then enables expansion of research efforts and enhances quality of UCLA IDDRC research by establishing uniformity in diagnosis, phenotyping, and conduct of clinical studies. Measurement development efforts, utilizing sophisticated analytics such as Item Response Theory and machine learning, are aimed at addressing methodologic gaps that have plagued prior clinical research efforts relating to IDDs, and foster new research by providing tools better suited to capturing clinical manifestations of underlying biology, hence advancing clinical translation. Our training aims to build capacity for IDD research. Our team is especially well suited for advancing assessment approaches for use in minimally verbal individuals, and very young children at risk for IDDs, a strength of our team. Core investigators are well known for measurement and paradigm development, lifespan studies, and studies of higher functioning subjects across a range of IDDs.