The Intellectual and Developmental Disabilities Research Center at CHOP/Penn

CHOP/宾夕法尼亚大学智力与发育障碍研究中心

• 批准号: 9173030
• 负责人: MARC YUDKOFF
• 金额: $ 129.99万
• 依托单位: CHILDREN'S HOSP OF PHILADELPHIA
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-11-01 至 2020-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9173030
• 关键词: Advocacy; Affect; Attenuated; Autistic Disorder; Award; Biochemistry; Bioinformatics; Biological Markers; Brain; Centers of Research Excellence; Chalk; Child; Clinical; Clinical Oncology Supplement (K12); Collaborations; Communities; Complement; Core Facility; Cultural Diversity; Developmental Disabilities; Disease; Dissection; Education; Evolution; Faculty; Faculty Recruitment; Family; Feeling; Fellowship; Fostering; Foundations; Funding; Future; Genes; Genetic; Goals; Grant; Health Policy; Health Services; Individual; Intellectual functioning disability; Interdisciplinary Study; Investigation; Knowledge; Language Disorders; Leadership; Magnetoencephalography; Medical Research; Medical center; Mental Retardation and Developmental Disabilities Research Centers; Mentors; Mission; Mitochondrial Diseases; Modification; Mutation; National Institute of Child Health and Human Development; National Institute of Neurological Disorders and Stroke; Neonatal Screening; Neurodevelopmental Disability; Neurosciences; Newsletter; Organizational Objectives; Parents; Periodicity; Philadelphia; Pre-Clinical Model; Private Sector; Privatization; Program Development; Publications; Quality Control; Rare Diseases; Recruitment Activity; Research; Research Personnel; Research Project Grants; Research Support; Resources; Science; Scientist; Series; Services; Severity of illness; Shapes; Source; Stem cells; Technology; Testing; Training; Training Programs; Translational Research; Underrepresented Minority; Vocabulary; Work; authority; autism spectrum disorder; brain behavior; career; career development; cost effective; cost effectiveness; disability; experience; gene therapy; improved; improved outcome; innovation; knowledge translation; lectures; meetings; member; mental health center; neurophysiology; new technology; novel; novel marker; organizational structure; patient advocacy group; prevent; programs; public health relevance; research facility; scientific organization; therapy development; tool; trend; urea cycle

 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.

 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)智力和发育障碍研究中心(IDDRC)和费城儿童福利中心(CHOP)提供资金。我们的IDDRC现在已经是第25个年头了，它是一个跨学科的项目，是CHOP/Penn发布发育障碍研究的主要机构。在下一个周期中，我们将在此基础上提出一个由6个组成部分组成的创新中心：(1)一个研究项目，它使用脑磁图(MEG)为患有自闭症谱系障碍的非语言和最少说话的儿童开发一种新的生物标志物--“电子签名”；(2)一系列6个研究核心设施，将为研究人员提供最先进的设施和专业知识，包括2个新核心(临床前模型和临床翻译)；(3)通过每月举办研讨会，包括IDDRC研究讲座、每月“粉笔讲座”和自闭症杰出讲座系列，培养“IDD社区”的感觉的教育计划；(4)职业发展计划，将使数十名杰出的年轻研究人员受益，他们将获得IDDRC新计划发展奖(由费城基金会资助)、IDDRC管理的神经发育障碍T32培训补助金和当地慈善机构向IDDRC提供的几个私人奖项(5年内总计价值约300万美元)的支持；(5)一个研究宣传任务，其中包括IDDRC-CHOP/Penn合作，以创建“英才中心”，鼓励对IDD进行跨学科的翻译研究；以及(6)参与IDDRC网络，以实现NICHD IDD分会的科学和组织目标。该中心将支持78个联邦资助的项目(2330万美元/年)，其中15个项目来自NICHD(580万美元/年)。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的努力：(A)导致残疾或调节疾病严重程度的遗传原始；(B)伴随基因变化的脑生化和神经生理学异常；以及(C)这些遗传/神经生理学变化的现象学表现，我们认为这是IDD的临床表现。我们采用这种“简化论”方法的理由是，对 将残疾分为孤立和“更简单”的组成部分，为开发预防、减轻甚至扭转这种疾病的破坏性后果的治疗方法提供了一种策略。在接下来的5年里，该中心将通过使用以下工具来实现这一目标：尖端研究核心，专注于转化性研究，CHOP/宾夕法尼亚大学巨大的临床资源，培养未来的领导者干部队伍，以及大力强调与IDDRC网络的创造性合作伙伴关系。