CLINICAL TRANSLATION CORE

贝勒医学院（Baylor College of Medicine）IDDRC的临床转化核心（CTC） 为基础科学研究人员、临床研究人员和临床医生提供了一个出色的平台， 基础设施和资源，使许多机械发现从我们的中心转化为 临床领域在这份提案中，我们很高兴能够在CTC成功的基础上扩大服务 使用新的行政和新的核心结构，使我们能够利用我们的优势， 治疗IDD。新的结构和服务诞生于我们的“不断发展的核心主题”， 适应和翻译我们中心研究人员的新的和令人兴奋的发现。 CTC将由三个单位组成：研究设计和实施单位、生物样本和数据 该股和中心导航股将与联合国难民署的其他核心、国家和 国际网络、患者登记处、患者和利益相关者，以促进过渡期的所有方面 IDD研究。我们对CTC的一个愿景是，它应该产生超出我们当地中心的影响， 我们的流程和活动应该对美国其他中心和IDD研究人员有用。 根据这一愿景，CTC将建立一个临床结果评估小组， 我们中心拥有重要的临床前和临床专业知识，以开发和验证临床试验的终点， 对临床试验的准备至关重要此外，反恐委员会将努力获得一份生物标本， 珍贵的样本可以与其他中心和研究人员共享。最后，作为未来 由于缺碘症研究的主要力量是年轻的临床医生和科学家，因此CTC将重点培训下一个- 培养缺碘症的临床和转化研究人员。 我们坚信，CTC将成为一个枢纽：1）基础研究人员将获得 临床信息和人体样本，从而被整合到临床企业，2）基础研究 这些问题将在临床竞技场中得到验证; 3）将培育新的诊断模式和治疗方法。 反恐委员会的结构和服务不仅有助于将调查结果从“法官席”转化为“法官席”， 床旁”，而且还推动基于临床表型观察的实验室研究。等 双向翻译不仅可以促进基础科学研究， IDD患者的护理。

In its current form, the Baylor College of Medicine (BCM) IDDRC’s Clinical Translational Core (CTC) has provided an outstanding platform to integrate basic science researchers, clinical investigators, and clinicians with infrastructure and resources to enable the translation of many mechanistic discoveries from our center into the clinical realm. In this proposal, we are excited to build upon the success of our CTC and expand on the services using a new administrative and novel core structure that will allow us to leverage our strengths for developing therapies for IDD. The new structure and services were born from our “evolving Core theme” in order to accommodate and translate the new and exciting discoveries from our center investigators. The CTC will be composed of three units: Study Design and Implementation Unit, Biospecimen and Data Unit, and Center Navigation Unit that will collaborate closely with other cores of the BCM IDDRC, national and international networks, patient registries, patients, and stakeholders to facilitate all aspects of transitional research in IDD. A vision that we have for our CTC is that it should have an impact beyond our local center and that our processes and activities should be of use to other centers and IDD researchers across the United States. In line with that vision, the CTC will establish a Clinical Outcome Assessment Panel that will leverage the significant preclinical and clinical expertise at our center to develop and validate endpoints for clinical trials which would be critical for clinical trial readiness. Additionally, the CTC will work towards having a biospecimen repository from which precious samples can be shared with other centers and researchers. Finally, as the future of IDD research is dependent on young clinicians and scientists, the CTC will focus on training the next- generation of clinical and translational researchers in IDD. We strongly believe that the CTC will be the nidus by which: 1) basic researchers will have access to clinical information and human samples and thus be integrated into the clinical enterprise, 2) basic research questions will be validated in a clinical arena, and 3) newer diagnostic modalities and therapies will be fostered. The structure and services of the CTC will not only facilitate the translation of findings from the “bench-to-the- bedside”, but also drive research in the laboratory based on phenotypic observations made in the clinics. Such bidirectional translation will not only advance the basic science research but also make a significant impact on the care of the patients with IDD.