Core B: Clinical Translational Core

项目总结：临床翻译核心（CTC） Hawk-IDDRC的临床转化核心（CTC）旨在解决那些 通过为研究人员提供资源，将发现从 实验室转化为临床实践，并使人们能够理解临床观察背后的生物学， 以推进转化研究。CTC通过提供招聘基础设施而与众不同 农村人口以及处于生命周期关键过渡点的参与者，包括： 青春期和青春期，成年期不同程度的独立，以及成年后的 专业护理人员发挥主要作用。这些过渡点代表了影响 长期的健康，但我们对每一个人的了解都是有限的。重要的是农村 人群1）获得适当护理的机会减少，2）有不同的潜在IDD暴露， 研究不够; 3）缺乏研究基础设施。霍克-IDDRC CTC准备满足这些需求， 因此，将通过携带来自未充分研究人群的独特临床数据和生物样本， （1）促进和加快招募和临床评估受缺碘症影响的 来自不同地区的各个生命阶段的患者，将他们及其家人与临床研究联系起来， 资源; 2）为缺碘症研究人员提供生物库和生物信息学服务; 3）促进和支持 通过为临床提供成功的架构，设计、开发和测试新型IDD疗法 为IDD研究人员提供试验和药物制造服务。反恐委员会的专门知识和资源 主管（马克·穆兰和亚历山大·巴苏克）涵盖了广泛的能力，从 产前风险评估、人类受试者招募以及临床数据和生物样本的库。具体 爱荷华州大学（UI）覆盖生命周期的资产包括CoLab（全球妊娠协作）， 由40个研究中心组成的国际合作，专注于妊娠结局研究，共享临床数据， 生物样本; SPARK的临床招募中心，一项全国性的自闭症遗传研究; UI领导的全州范围内 农村和城市地区的合作诊所、学校和供应商网络;以及智力残疾- 精神疾病（ID-MI）诊所，全国仅有的几个住院单位之一，专注于患者 智力残疾和精神疾病并存反恐委员会将作为这些活动的中心协调中心。 资源，简化调查人员的访问，并使其能够支持31个联邦资助的项目 每年总计1260万美元。获得统一、协调的临床数据、生物样本和 临床队列允许CTC与所有核心合作进行回顾性和前瞻性IDD研究。 反恐委员会的资源将用于支持霍克-非洲发展问题国际十年的使命，即提供一个组织结构 它整合了从受孕到成年的整个生命周期的基础和临床研究， 预防、诊断、治疗和改善缺碘症，并针对得不到充分服务的农村人口。

PROJECT SUMMARY: CLINICAL TRANSLATIONAL CORE (CTC) The Clinical Translational Core (CTC) of the Hawk-IDDRC seeks to address the challenges faced by those living with IDD at all life stages by providing investigators the resources to transition discoveries from the laboratory into clinical practice, and enable an understanding of the biology underlying clinical observations, in order to advance translational research. The CTC differentiates itself by providing infrastructure for recruiting rural populations as well as participants at key transition points across the lifespan, including: the perinatal period, puberty and adolescence, adulthood at varying levels of independence, and later adulthood as professional caregivers assume a primary role. These transition points represent critical periods that influence the long-term wellbeing of those living with IDD, yet our understanding of each is limited. Importantly, rural populations 1) have decreased access to appropriate care, 2) have different potential IDD exposures that are not well studied, and 3) lack research infrastructure. The Hawk-IDDRC CTC is poised to fill these needs and will thus provide access to unique clinical data and biosamples from this understudied population by carrying out the following aims: 1) Facilitate and accelerate the recruitment and clinical evaluation of IDD-affected patients at all stages of life and from diverse areas to connect them and their families with clinical studies and resources; 2) Provide IDD researchers with biobanking and bioinformatics services; 3) Facilitate and support the design, development, and testing of novel IDD therapies by providing a successful architecture for clinical trials and drug manufacturing services to IDD researchers. The expertise and resources of the CTC Co- Directors (Mark Santillan and Alexander Bassuk) covers a broad spectrum of capabilities, ranging from prenatal risk assessment, to human subject recruitment, and banking of clinical data and biosamples. Specific assets of the University of Iowa (UI) that cover the lifespan include CoLab (Global Pregnancy Collaboration), an international collaboration of 40 sites focused on pregnancy outcome research, sharing clinical data and biosamples; a clinical recruitment center for SPARK, a nationwide genetic study of autism; UI-led statewide networks of collaborating clinics, schools, and providers in rural and urban areas; and the Intellectual Disability- Mental Illness (ID-MI) clinic, one of only a handful of inpatient units in the country that focuses on patients with intellectual disability and a co-occurring mental illness. The CTC will serve as a central focal point for these resources, streamlining access for investigators, and enabling its support of 31 federally funded projects totaling $12.6 million per year. The access to a unified, harmonized source of clinical data, biosamples, and clinical cohorts allows the CTC to work with all Cores to perform retrospective and prospective IDD studies. The resources of the CTC will support the mission of the Hawk-IDDRC to provide an organizational structure that integrates basic and clinical research across the lifespan—from conception to adulthood—focused on the prevention, diagnosis, treatment and amelioration of IDDs, and tailored to a rural, underserved population.