RFA-HD-13-002 requires the inclusion of a Clinical Translational Core within each IDDRC, addressing a combination of activities that includes: (a) recruitment of research participants, (b) subject assessment, phenotyping, or treatment, (c) development of biomarkers, (d) manufacturing services, (e) highthroughput screening, and/or (f) collection of samples from individuals with IDD for biobanking. As described in our Introductory Overview and the following Core descriptions, many of these activities are supported by other Cores and existing resources within the large and multidisciplinary research environment developed within our Institutions over the years. Therefore, the overall objective of the Clinical Translational Core is to complement those resources and coordinate access for IDDRC-supported investigators. This will contribute to our primary goal of enhancing the conversion of basic science discoveries into effective, commercially available disease-altering therapies for children and adults with genetic and acquired intellectual and developmental disabilities. The Core will be structured to complement our Clinical Trials Unit (CTU), a program established three years ago in response to the increasing emphasis on translational research within our IDDRC and the broader research program it supports. The CTU is focused on the development of treatments targeting developmental disorders, including conducting investigational dmg trials at all phases. The Clinical Translational Core will expand upon CTU capabilities to provide expert resources including consultation on biostatistics and protocol design, pre-review prior to submission of protocols to the institutional review board (IRB) and hands-on advice and help with carrying out investigational protocols by experienced clinical trials faculty, research coordinators and administrative staff.

National Institute of Health (NIH)
Eunice Kennedy Shriver National Institute of Child Health & Human Development (NICHD)
Specialized Center--Cooperative Agreements (U54)
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Special Emphasis Panel (ZHD1-DSR-H (ID))
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Hugo W. Moser Research Institute Kennedy Krieger
United States
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Kim, Sangjune; Yun, Seung Pil; Lee, Saebom et al. (2018) GBA1 deficiency negatively affects physiological ?-synuclein tetramers and related multimers. Proc Natl Acad Sci U S A 115:798-803
Pilarowski, Genay O; Vernon, Hilary J; Applegate, Carolyn D et al. (2018) Missense variants in the chromatin remodeler CHD1 are associated with neurodevelopmental disability. J Med Genet 55:561-566
Statton, Matthew A; Vazquez, Alejandro; Morton, Susanne M et al. (2018) Making Sense of Cerebellar Contributions to Perceptual and Motor Adaptation. Cerebellum 17:111-121
Jacobson, Lisa A; Geist, Megan; Mahone, E Mark (2018) Sluggish Cognitive Tempo, Processing Speed, and Internalizing Symptoms: the Moderating Effect of Age. J Abnorm Child Psychol 46:127-135
Newcomb, Eli T; Hagopian, Louis P (2018) Treatment of severe problem behaviour in children with autism spectrum disorder and intellectual disabilities. Int Rev Psychiatry 30:96-109
Wang, Shuyan; Shi, Xiaohai; Wei, Shuang et al. (2018) Kr├╝ppel-like factor 4 (KLF4) induces mitochondrial fusion and increases spare respiratory capacity of human glioblastoma cells. J Biol Chem 293:6544-6555
Ruan, Linhao; Zhang, Xi; Li, Rong (2018) Recent insights into the cellular and molecular determinants of aging. J Cell Sci 131:
Wu, Dan; Lei, Jun; Jia, Bei et al. (2018) In vivo assessment of the placental anatomy and perfusion in a mouse model of intrauterine inflammation. J Magn Reson Imaging 47:1260-1267
Chang, Andrew D; Vaidya, Punit V; Retzbach, Edward P et al. (2018) Narp Mediates Antidepressant-Like Effects of Electroconvulsive Seizures. Neuropsychopharmacology 43:1088-1098
Hagopian, L P; Frank-Crawford, M A (2018) Classification of self-injurious behaviour across the continuum of relative environmental-biological influence. J Intellect Disabil Res 62:1108-1113

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