The Intellectual and Developmental Disabilities Research Center at CHOP/Penn

 描述（由申请人提供）：本申请旨在为宾夕法尼亚大学（宾夕法尼亚大学）和费城儿童医院（CHOP）的智力和发育障碍研究中心（IDDRC）提供资金。我们的IDDRC，现在在其第25个年头，是一个跨学科的计划，是在CHOP/宾夕法尼亚州的首席机构颁布的研究发展障碍。在下一个周期中，我们将在此基础上建立一个创新中心，由6个组成部分组成：（1）一个研究项目，使用脑磁图（MEG）开发一种新的生物标志物-“电子签名”-用于患有自闭症谱系障碍的非语言和最低语言儿童;（2）一系列6个研究核心设施，为研究人员提供最先进的设施和专业知识，包括2个新的核心（3）教育计划，透过每月举办的研讨会，包括IDDRC研究讲座、每月的“粉笔讲座”及自闭症杰出人士讲座系列，培养“IDD社区”的感觉;（4）一项职业发展倡议，将使数十名杰出的年轻调查人员受益，他们将获得IDDRC新项目发展奖的支持（由费城基金会资助），IDDRC管理的T32神经发育障碍培训补助金和几个私人奖项（5）一项研究宣传使命，涉及IDDRC- CHOP/Penn合作创建“卓越中心”，鼓励对IDD进行跨学科转化研究;（6）参加更大的缺碘症防治中心网络，以实现NICHD缺碘症分支的科学和组织目标。该中心将支持78个联邦资助的项目（每年2 330万美元），其中15个来自NICHD（每年580万美元）。我们IDDRC的主题是“基因、大脑、行为”，这个名称反映了我们在三个相互关联的领域中不断努力了解IDD：（a）导致残疾或调节疾病严重程度的遗传原基;（B）伴随遗传变化的大脑生物化学和神经生理学异常;和（c）这些遗传/神经生理学改变的现象学表现，我们认为这些改变是IDD的临床表现。我们对这种“还原论”方法的基本原理是， 将残疾分为孤立和“较简单”的组成部分，提供了一种战略，可据此开发预防、减轻甚至扭转这种疾病的破坏性后果的治疗方法。在未来5年，该中心将通过部署这些工具来实现这一目标：尖端研究核心，专注于转化研究，CHOP/Penn的巨大临床资源，培养未来领导者的干部，并强调与IDDRC网络的创造性合作伙伴关系。

 DESCRIPTION (provided by applicant): This application seeks funding for the Intellectual and Developmental Disabilities Research Center (IDDRC) at the U of PA (Penn) and the Children's Hosp of Philadelphia (CHOP). Our IDDRC, now in its 25th year, is an inter- disciplinary program that is the chief agency at CHOP/Penn for the promulgation of research into the developmental disabilities. In the next cycle we build upon this foundation by proposing an innovative Center with 6 components: (1) A research project which uses magnetoencephalography (MEG) to develop a novel biomarker - an "electronic signature" - for non-verbal and minimally verbal children with autistic spectrum disorders; (2) A series of 6 research core facilities which will provide investigators with state-of-the-art facilities and expertise, including 2 new cores (Preclinical Models and Clinical Translational); (3) An educational program which will nurture a feeling of "IDD Community" by featuring monthly seminars, including the IDDRC Research Lecture, the Monthly "Chalk Talks" and the Autism Distinguished Lecture Series; (4) A career development initiative which will benefit dozens of exceptional young investigators who will receive support from the IDDRC New Program Development award (to be funded by the Philadelphia Foundation), the IDDRC-administered T32 Training Grant in Neurodevelopmental Disabilities and several private awards (aggregate value ~ $3 million over 5 years) given by local philanthropies to the IDDRC; (5) A research advocacy mission which involves an IDDRC- CHOP/Penn collaboration to create "Centers of Excellence" that encourage inter-disciplinary translational research into IDD; and (6) Participation in the larger IDDRC Network in order to realize the scientific and organizational goals of the IDD Branch of NICHD. The Center will support 78 federally-funded projects ($23.3 million/yr), of which 15 are from NICHD ($5.8 million/yr). The theme of our IDDRC is "Genes, Brain, Behavior", a designation which reflects our ongoing effort to understand IDD in 3 inter-related domains: (a) The genetic anlage which causes a disability or modulates disease severity; (b) The anomalies of brain biochemistry and neurophysiology which accompany genetic changes; and (c) The phenomenological manifestations of these genetic/neurophysiologic alterations which we recognize as clinical manifestations of the IDD. Our rationale for this "reductionist" approach is that the dissection of a disability into isolated and "simpler" components affords a strategy with which to develop therapies that will prevent, attenuate or even reverse the devastating consequences of the disorder. Over the coming 5 years the Center will pursue this aim by deploying these tools: cutting-edge research cores, a focus on translational research, the enormous clinical resources of CHOP/Penn, nurturing a cadre of future leaders, and a strong emphasis on creative partnership with the IDDRC Network.