CLINICAL TRANSLATION CORE

在目前的形式下，贝勒医学院(BCM)IDDRC的临床翻译核心(CTC) 提供了一个出色的平台，将基础科学研究人员、临床研究人员和临床医生 基础设施和资源，使许多机械发现能够从我们的中心转化为 临床领域。在这项提议中，我们很高兴能在我们反恐中心的成功基础上再接再厉，扩大服务 使用新的管理和新颖的核心结构，使我们能够利用我们的优势来开发 IDD的治疗方法。新的结构和服务源自我们的“不断发展的核心主题”，目的是 容纳和翻译我们中心研究人员的新发现和令人兴奋的发现。 反恐委员会将由三个单位组成：研究设计和实施股、生物质司和数据股。 和中央导航股，将与BCM IDDRC、国家和 国际网络、患者登记、患者和利益相关者，以促进过渡的方方面面 对缺铁性贫血的研究。我们对CTC的愿景是，它应该产生超出我们当地中心和 我们的流程和活动应该对美国各地的其他中心和IDD研究人员有所帮助。 根据这一愿景，反恐委员会将设立一个临床成果评估小组，该小组将利用 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 对临床试验的准备情况至关重要。此外，反恐委员会将致力于研制一种生物检疫剂。 可与其他中心和研究人员共享珍贵样本的储存库。最后，作为未来 IDD的研究依赖于年轻的临床医生和科学家，CTC将专注于培训下一代- 一代IDD临床和转化型研究人员。 我们坚信，反恐委员会将成为核心：1)基础研究人员将有机会获得 临床信息和人体样本，从而整合到临床企业中，2)基础研究 问题将在临床领域得到验证，并将促进更新的诊断模式和治疗方法的发展。 反恐委员会的结构和服务不仅将便利将调查结果从“审判庭到审判庭”的翻译工作。 床边“，但也推动实验室的研究，基于在诊所进行的表型观察。是这样的 双向翻译不仅将促进基础科学研究，而且将对 碘缺乏病患者的护理。

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.