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Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat - Administrative Supplement

肌营养不良症的再生失败：炎症、纤维化和脂肪 - 行政补充

基本信息

批准号：
10212504
负责人：
Elizabeth M McNally
金额：
$ 40.39万
依托单位：
UNIVERSITY OF FLORIDA
依托单位国家：
美国
项目类别：
财政年份：
2020
资助国家：
美国
起止时间：
2020-09-01 至 2021-07-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/10212504
关键词：
Administrative Supplement Adrenal Cortex Hormones Animals Basic Science Biological Assay Cell Therapy Clinical Clinical Research Communication Communities Community Outreach Complement Disease Progression Duchenne muscular dystrophy Educational workshop Equipment Ethnic Origin Evaluation Fatty acid glycerol esters Fibrosis Freezing Funding Gender Goals Hindlimb Suspension Imaging Device In Situ Inflammation Injury International Laboratories Laboratory Research Leadership Liquid substance Measurement Mentors Mission Monitor Mus Muscle Muscle function Muscular Dystrophies Natural regeneration Nitrogen Patient advocacy Patients Pharmacology Phase Physicians Physiological Race Research Research Personnel Research Support Resource Sharing Resources Respiratory Diaphragm Respiratory physiology Running Sampling Scientist Services Site Socioeconomic Status Testing Therapeutic Training Training and Education Translational Research Treatment Efficacy Underrepresented Groups United States National Institutes of Health career development cost disability experience experimental study gene therapy improved instrumentation interest meetings mouse model patient outreach pre-doctoral preservation programs recruit side effect targeted treatment therapeutic target training opportunity treadmill web site

项目摘要

Abstract The purpose of this Administrative Supplement is to maintain the three cores of the UF Wellston Center during its one-year no cost extension in order to serve the administrative, training, and resource functions of the Center, as well as the national muscular dystrophy community. The Administrative Core provides integration and management of activities for the Center. It promotes interactions and communications between the research and patient/advocacy communities on both a national and local level. The Administrative Core maintains our website to communicate the Center mission and the availability of training opportunities and services available through our Shared Scientific Resource Core. The overall objective of the Training Core is to increase the number of rigorously trained and scientifically competent researchers capable of sustaining productive basic science, translational and/or clinical research programs in muscular dystrophy. To meet this central objective, the core will identify and recruit qualified basic science and clinical predoctoral and postdoctoral candidates who demonstrate a genuine interest in, and potential for, independent basic science, translational and/or clinical research in muscular dystrophy. Particular emphasis will be placed on the recruitment of trainees from underrepresented groups with respect to race, ethnicity, gender, socio-economic status and disabilities. Critical to the evaluation of potential therapeutics (pharmacological, gene, or cell therapies) are sensitive and repeatable physiological assessments of muscle function applied to mouse models of dystrophy. Therefore, we are proposing to continue our Shared Scientific Resource Core that performs ex vivo, in situ, and whole animal assessments of muscle integrity and function. This core will not only support the needs of the projects within the center during the no cost extension, but will also serve as a national resource for performing functional evaluation of potential therapies for the muscular dystrophies. The Physiological Assessment Core / Shared Scientific Resource Core is housed in the laboratory space designated to Dr. Barton, the Core Director. Dr. Barton is a muscle physiologist with extensive experience evaluating muscle function in mice. This includes all instrumentation for muscle physiological measurements, specialized equipment for hindlimb suspension experiments, documented free wheel running, and treadmill running, as well as interim storage of samples in both –80ºC and liquid nitrogen freezers. We propose to collaborate with neighboring colleagues to assess whole animal respiratory function, which will complement ex vivo assessment of the diaphragm. We believe that this Core has provided a previously unmet need in the past 15 years of its existence, and will continue to be a valuable resource that will enable the entire muscular dystrophy research community to utilize these assays to assess the potential benefits of a large number of approaches to the treatment of different forms of muscular dystrophy.

摘要本行政副刊的目的是维护UF Wellston中心的三个核心其为期一年的免费延期，以服务于中心，以及国家肌肉营养不良社区。管理核心提供集成和管理中心的活动。它促进了两国之间的互动和沟通国家和地方层面的研究和患者/倡导社区。行政核心维护我们的网站以传达中心的使命和培训机会的可用性，并通过我们的共享科学资源核心提供的服务。培训核心的总体目标是增加经过严格培训和具有科学能力的研究人员数量，使其能够肌营养不良症的生产性基础科学、翻译和/或临床研究计划。为了满足这一点中心目标，核心将确定和招聘合格的基础科学和临床博士前和对独立基础科学表现出真正兴趣和潜力的博士后候选人，肌营养不良症的翻译和/或临床研究。特别强调的将是在种族、族裔、性别、社会经济方面从任职人数不足的群体中征聘受训人员身份和残疾。对潜在疗法(药理学、基因或细胞)的评估至关重要疗法)是对小鼠肌肉功能的敏感和可重复的生理学评估营养不良的模型。因此，我们建议继续我们共享的科学资源核心对肌肉的完整性和功能进行体外、原位和整体动物评估。这个核心不会在无成本延期期间，仅支持中心内项目的需求，但也将作为对肌营养不良症的潜在疗法进行功能评估的国家资源。这个生理评估核心/共享科学资源核心安置在实验室空间指定给核心主管巴顿博士。巴顿博士是一位经验丰富的肌肉生理学家。评估小鼠的肌肉功能。这包括用于肌肉生理测量的所有仪器，用于后肢悬挂实验的专用设备、有记录的自由轮运行和跑步机样品在零下80°C和液氮冰柜中运行和临时存储。我们建议与邻居同事合作评估整个动物的呼吸功能，这将补充EX 横隔膜的活体评估。我们认为，这一核心在过去提供了以前没有得到满足的需求它存在了15年，并将继续成为一种宝贵的资源，使整个肌肉营养不良研究界利用这些检测来评估大量治疗不同形式的肌营养不良症的方法。

项目成果

期刊论文数量（74）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（2）

Nutraceutical and pharmaceutical cocktails did not improve muscle function or reduce histological damage in D2-mdx mice.

营养保健品和药物混合物不能改善 D2-mdx 小鼠的肌肉功能或减少组织学损伤。

DOI：
10.1152/japplphysiol.00162.2019
发表时间：
2019
期刊：
Journal of applied physiology (Bethesda, Md. : 1985)
影响因子：
0
作者：
Spaulding,HannahR;Quindry,Tiffany;Hammer,Kayleen;Quindry,JohnC;Selsby,JoshuaT
通讯作者：
Selsby,JoshuaT

Filopodia powered by class x myosin promote fusion of mammalian myoblasts.

DOI：
10.7554/elife.72419
发表时间：
2021-09-14
期刊：
eLife
影响因子：
7.7
作者：
Hammers DW;Hart CC;Matheny MK;Heimsath EG;Lee YI;Hammer JA 3rd;Cheney RE;Sweeney HL
通讯作者：
Sweeney HL

Myostatin is elevated in congenital heart disease and after mechanical unloading.