NCMRR-DC Core Molecular & Functional Outcome Measures in Rehabilitation Medicine

NCMRR-DC核心分子

• 批准号: 8675744
• 负责人: SUSAN M KNOBLACH
• 金额: $ 94.99万
• 依托单位: CHILDREN'S RESEARCH INSTITUTE
• 依托单位国家: 美国
• 财政年份: 2005
• 资助国家: 美国
• 起止时间: 2005-09-15 至 2016-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8675744
• 关键词: Adult; Age; Ancillary Study; Appointment; Award; Back; Bioinformatics; Biological Markers; Brain; Cerebral Palsy; Charge; Child; Clinical; Communities; Computer software; Data; District of Columbia; Educational workshop; Ensure; Environment; Equipment; Evaluation; Faculty; Feedback; Functional disorder; Funding; Funding Applicant; Genes; Genetic; Genomics; Genotype; Goals; Health Sciences; Healthcare Systems; Housing; Human; Human Resources; Institution; International; Investments; Limb structure; Medical; Medical center; Medicine; Messenger RNA; Methods; MicroRNAs; Modeling; Molecular; Monitor; Mus; Occupational; Outcome; Outcome Measure; Outcomes Research; Patients; Pharmaceutical Preparations; Phenotype; Physical Medicine; Pilot Projects; Preclinical Drug Evaluation; Premature Infant; Principal Investigator; Procedures; Process; Proteins; Proteome; Proteomics; Quality of life; Reagent; Rehabilitation Research; Rehabilitation therapy; Research; Research Personnel; Resources; Sampling; Services; Site; Software Tools; Spinal Cord; Spinal Dysraphism; Surveys; Systems Biology; Technology; Testing; Training; Trauma; United States National Institutes of Health; Universities; Washington; Writing; cohort; cost; design; disability; functional outcomes; genome wide association study; improved; lectures; medical schools; meetings; neuromuscular; new technology; outreach; pre-clinical; programs; protein protein interaction; public health relevance; respiratory; success; symposium

DESCRIPTION (provided by applicant): Medical rehabilitation is a research and clinical concern across the age span. Premature infants require rehabilitation of multiple types (physical, respiratory, occupational), children with cerebral palsy and spina bifida have extensive rehabilitation needs. Adults with spinal cord, brain, and limb trauma all require extensive rehabilitation support. Research to improve functional outcomes, and understand the pathophysiology of specific disabilities is important, both for patient quality of life, as well as burden on the health care system. The goal of our NCMRR-DC Core is to provide technical services and training across both molecular and functional outcome measures in rehabilitation research. During the previous award period, our NCMRR Core provided services and training to 108 principal investigators in 32 institutions, with processing and analysis of >4,500 samples. Didactic and outreach efforts included >3,500 hrs of on-site training of external investigators, 95 invited lectures, 29 presentations at national and international conferences, and organization of workshops to write and disseminate standard operating procedures for functional outcome measures. In this competitive renewal application, the PIs have extended their molecular outcomes services and training to include murine and human functional outcomes. These include clinical evaluator services and training, a murine functional testing facility, and facilitation of ancillary studies in pre-existing human cohorts emphasizing functional outcomes. These new resources have been established over the last 4 years, have been heavily utilized by the international rehabilitation research community, and are now proposed as a part of the NCMRR-DC Core.

描述（由申请人提供）：医疗康复是一个跨年龄段的研究和临床问题。早产儿需要多种类型的康复（躯体、呼吸、职业），脑瘫、脊柱裂儿童的康复需求广泛。患有脊髓、大脑和四肢创伤的成年人都需要广泛的康复支持。改善功能结果并了解特定残疾的病理生理学的研究对于患者的生活质量以及医疗保健系统的负担都很重要。我们的 NCMRR-DC 核心的目标是提供康复研究中分子和功能结果测量的技术服务和培训。在上一奖励期间，我们的 NCMRR 核心为 32 个机构的 108 名主要研究人员提供了服务和培训，处理和分析了超过 4,500 个样本。教学和外展工作包括超过 3,500 小时的外部调查员现场培训、95 场邀请讲座、在国内和国际会议上的 29 场演讲，以及组织研讨会以编写和传播功能性成果衡量标准操作程序。在这一竞争性更新应用中，PI 扩展了其分子结果服务和培训，以包括小鼠和人类功能结果。其中包括临床评估服务和培训、小鼠功能测试设施以及促进现有人类队列中强调功能结果的辅助研究。这些新资源是在过去 4 年中建立的，已被国际康复研究界大量利用，现在被提议作为 NCMRR-DC 核心的一部分。

项目成果

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery.

通过全身反义传递，营养不良 mdx52 小鼠体内外显子 45-55 的全身跳跃。

• 发表时间: 2012-08-21
• 影响因子: 11.1
• 作者: Aoki, Yoshitsugu;Yokota, Toshifumi;Nagata, Tetsuya;Nakamura, Akinori;Tanihata, Jun;Saito, Takashi;Duguez, Stephanie M R;Nagaraju, Kanneboyina;Hoffman, Eric P;Partridge, Terence;Takeda, Shin'ichi
• 通讯作者: Takeda, Shin'ichi

VBP15, a glucocorticoid analogue, is effective at reducing allergic lung inflammation in mice.

VBP15 是一种糖皮质激素类似物，可有效减少小鼠过敏性肺部炎症。

• 发表时间: 2013
• 影响因子: 3.7
• 作者: Damsker, Jesse M;Dillingham, Blythe C;Rose, Mary C;Balsley, Molly A;Heier, Christopher R;Watson, Alan M;Stemmy, Erik J;Jurjus, Roslyn A;Huynh, Tony;Tatem, Kathleen;Uaesoontrachoon, Kitipong;Berry, Dana M;Benton, Angela S;Freishtat, Robert J
• 通讯作者: Freishtat, Robert J

Gene sequences regulating the production of apoE and cerebral palsy of variable severity.

调节 apoE 产生和不同严重程度的脑瘫的基因序列。

• 发表时间: 2014-09
• 作者: Lien, Espen;Andersen, Guro L;Bao, Yongde;Gordish;Skranes, Jon;Blackman, James A;Vik, Torstein
• 通讯作者: Vik, Torstein

Integrated next-generation sequencing of 16S rDNA and metaproteomics differentiate the healthy urine microbiome from asymptomatic bacteriuria in neuropathic bladder associated with spinal cord injury.

16S rDNA 和宏蛋白质组学的集成下一代测序可区分健康尿液微生物组和与脊髓损伤相关的神经性膀胱中的无症状菌尿。

• 发表时间: 2012-08-28
• 作者: Fouts, Derrick E;Pieper, Rembert;Szpakowski, Sebastian;Pohl, Hans;Knoblach, Susan;Suh, Moo;Huang, Shih;Ljungberg, Inger;Sprague, Bruce M;Lucas, Sarah K;Torralba, Manolito;Nelson, Karen E;Groah, Suzanne L
• 通讯作者: Groah, Suzanne L

Long-term efficacy of systemic multiexon skipping targeting dystrophin exons 45-55 with a cocktail of vivo-morpholinos in mdx52 mice.

在 mdx52 小鼠中，使用体内吗啡啉混合物靶向肌营养不良蛋白外显子 45-55 的全身多外显子跳跃的长期疗效。

• 发表时间: 2015-02-03
• 作者: Echigoya, Yusuke;Aoki, Yoshitsugu;Miskew, Bailey;Panesar, Dharminder;Touznik, Aleksander;Nagata, Tetsuya;Tanihata, Jun;Nakamura, Akinori;Nagaraju, Kanneboyina;Yokota, Toshifumi
• 通讯作者: Yokota, Toshifumi