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：
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
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/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

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

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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（由应用程序提供）：本申请寻求宾夕法尼亚州U（Penn）和费城儿童医院（CHOP）的智力和发展障碍研究中心（IDDRC）的资金。我们的IDDRC已于25年，是一项跨学科计划，是CHOP/PENN的首席机构，旨在颁发有关发展障碍研究的研究。在下一个周期中，我们通过提出一个具有6个组成部分的创新中心来建立基础：（1）一个研究项目，该项目使用磁脑电图（MEG）开发一种新颖的生物标志物（一种“电子签名”），用于非语言和微型言语儿童，患有令人印象深刻的光谱障碍；（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）一个教育计划，将通过每月下水道（包括IDDRC研究讲座，每月的“粉笔谈话”和自闭症杰出的讲座系列）来为“ IDD社区”的感觉进行护理；（4）一项职业发展计划将使数十名杰出的年轻调查人员受益，他们将获得IDDRC新计划开发奖的支持（由费城基金会资助），IDDRC的T32 T32 T32 T32 T32 T32培训赠款在神经发育障碍和几个私人奖励（在5年内价值300万年）（在500万年内由Philan philan the Philan the IDCROP）授予; （5）一项研究倡导任务，涉及IDDRC-CHOP/PENN合作，以创建“卓越中心”，鼓励跨学科的翻译研究；（6）参与较大的IDDRC网络，以实现NICHD IDD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”，它反映了我们在3个相关领域中理解IDD的持续努力：（a）导致残疾或调节疾病严重程度的遗传性；（b）适应遗传变化的脑生物化学和神经生理学的异常；（c）这些遗传/神经生理学改变的现象学表现，我们认为这是IDD的临床表现。我们采用这种“简化主义者”方法的理由是解剖孤立和“简单”组成部分的残疾提供了一种策略，以制定疗法，以防止，减弱甚至扭转这种疾病的毁灭性后果。在接下来的5年中，该中心将通过部署这些工具来追求这一目标：尖端的研究核心，专注于翻译研究，Chop/Penn的巨大临床资源，培养未来领导者的干部以及与IDDRC网络的创造性合作伙伴关系。