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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.

摘要本行政补充的目的是保持UF韦尔斯顿中心的三个核心，它的一年无费用延长，以服务于行政，培训和资源职能，中心，以及国家肌肉萎缩症社区。行政核心提供整合管理中心的活动。它促进了在国家和地方一级的研究和患者/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，通过我们的共享科学资源核心提供服务。培训核心的总体目标是增加受过严格训练和有科学能力的研究人员的数量，生产性基础科学，转化和/或临床研究计划在肌营养不良症。满足这一中心目标，核心将确定和招募合格的基础科学和临床博士前，博士后候选人谁表现出真正的兴趣，和潜力，独立的基础科学，用于肌营养不良症的转化和/或临床研究。将特别强调从在种族、族裔、性别、社会经济和文化方面代表性不足的群体中招聘受训人员地位和残疾。对潜在治疗方法（药理学、基因或细胞）的评价至关重要治疗）是应用于小鼠的肌肉功能的灵敏的和可重复的生理评估。营养不良的模型因此，我们建议继续我们的共享科学资源核心，进行肌肉完整性和功能的离体、原位和整体动物评估。这个核心不会只支持中心内项目的需要，在没有成本的扩展，但也将作为一个对肌营养不良症的潜在疗法进行功能评估的国家资源。的生理评估核心/共享科学资源核心位于实验室空间指定给核心主管巴顿博士巴顿博士是一位肌肉生理学家，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，专门的设备，用于后肢悬吊实验、记录的自由轮跑步和跑步机运行，以及样品在-80 ° C和液氮冷冻机中的临时储存。我们建议与邻近的同事合作，评估整个动物的呼吸功能，这将补充前隔膜的体内评估。我们认为，这一核心已满足了过去未得到满足的需求 15年的存在，并将继续成为一个宝贵的资源，将使整个肌肉营养不良研究社区利用这些测定来评估大量的潜在益处，不同形式的肌营养不良症的治疗方法。

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）

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

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

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