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

基本信息

批准号：
9054630
负责人：
MARC YUDKOFF
金额：
$ 114.59万
依托单位：
CHILDREN'S HOSP OF PHILADELPHIA
依托单位国家：
美国
项目类别：
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/9054630
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 Electronic Mail Electronics Evolution Faculty Faculty Recruitment Family Feeling Fellowship Fostering Foundations Funding Future Genes Genetic Goals Grant Head 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 Research Newsletter Organizational Objectives Parents Philadelphia Pre-Clinical Model Private Sector Program Development Psyche structure Publications Quality Control Rare Diseases 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 neurophysiology new technology novel novel marker organizational structure patient advocacy group prevent programs public health relevance research facility therapy development tool trend urea cycle

项目摘要

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) 和费城儿童医院 (CHOP) 寻求资助。我们的 IDDRC 现已成立 25 年。一个跨学科项目，是 CHOP/宾夕法尼亚大学推广发育障碍研究的主要机构。在下一个周期中，我们在此基础上提出了一个创新中心的建议。包含 6 个组成部分：(1) 一项研究项目，利用脑磁图 (MEG) 为患有自闭症谱系障碍的非语言和最低限度语言儿童开发一种新型生物标记物——“电子签名”；(2) 一系列 6 个研究核心；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（临床前模型和临床转化）； (3) 一项教育计划，通过以下方式培养“IDD 社区”的感觉；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； (4) 一项职业发展计划，将使数十名杰出的年轻研究人员受益，他们将获得 IDDRC 新项目开发奖的支持（由费城基金会资助）、IDDRC 管理的神经发育障碍 T32 培训补助金以及当地慈善机构颁发的多项私人奖项（5 年内总价值约为 300 万美元） (5) 一项研究倡导任务，其中涉及 IDDRC-CHOP/宾夕法尼亚大学合作创建“卓越中心”，鼓励对 IDD 进行跨学科转化研究；以及 (6) 参与更大的 IDDRC 网络，以便实现 NICHD IDD 分部的科学和组织目标该中心将支持 78 个联邦资助项目（每年 2330 万美元），其中 15 个来自 NICHD。（580 万美元/年）我们 IDDRC 的主题是“基因、大脑、行为”，这一名称反映了我们在 3 个相互关联的领域理解 IDD 的持续努力：(a) 导致残疾或调节的遗传原基。疾病严重程度；(b) 伴随遗传变化的大脑生物化学和神经生理学异常；以及 (c) 这些遗传/神经生理学改变的现象学表现，我们将其视为临床表现。我们采用这种“还原论”方法的理由是：剖析将残疾分为孤立的和“简单”的部分提供了一种策略，可用于开发预防、减轻甚至扭转该疾病的破坏性后果的疗法，该中心将通过部署以下工具来实现这一目标：边缘研究核心、对转化研究的关注、CHOP/Penn 的巨大临床资源、培养未来领导者的骨干队伍，以及高度重视与 IDDRC 网络的创造性合作关系。