UCLA IDDRC: Translational Core

临床翻译核心：摘要 UCLA IDDRC的翻译临床核心成立于2015年，其使命是支持高质量的， 多学科、协作的临床转化研究促进了对病因、预防、 智力和发育障碍(IDDS)的诊断和治疗。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；以及为国际发展研究中心调查员衔接提供的其他协助 具有基础科学的临床研究。该中心致力于推进IDD研究的评估方法 通过创新措施的制定、评估和传播。核心还支持外展， Biosecimen银行业务和数据管理。此外，核心还将参与IDDRC网络等 与IDDRC相关的多点临床调查。 在过去4年中，核心成功地建立了有效的程序，以支持正在进行的 和新的临床IDD研究，几乎是这一时间框架内支持的数量的两倍，并做出了贡献 现场进行了新的表型检测。核心在支持几项新研究方面发挥了影响，这些研究包括 新接触临床研究的早期职业研究人员。在这次更新中，我们通过增加两个 新的核心合作调查员(凯瑟琳·洛德博士和鲁朱塔·巴特·威尔逊博士)带来了社会、 诊断、行为、运动和神经学评估、缺碘干预试验、纵向研究和 对金标诊断仪器进行大规模培训。利用核心支持实现实质性的 成本效率，降低单个项目成本，并通过消除培训、质量控制和 保持个人研究的可靠性。然后，核心访问可以扩展研究工作，并 通过建立诊断、表型和操作的一致性来提高加州大学洛杉矶分校IDDRC研究的质量 临床研究。使用复杂的分析(如项目响应)来衡量开发工作 理论和机器学习，旨在解决困扰先前临床的方法论差距 与IDDS相关的研究努力，并通过提供更适合于捕获临床数据的工具来促进新的研究 基础生物学的表现，从而推进临床翻译。我们的培训旨在培养以下能力 IDD研究。我们的团队特别适合于最低限度地使用先进的评估方法 口头上的个人，以及面临IDDS风险的幼儿，这是我们团队的一大优势。核心调查人员都很好 以测量和范例开发、寿命研究和更高功能学科的研究而闻名 在一系列IDD中。

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.