Muscle Cell Biology, Pathophysiology, and Therapeutics

肌肉细胞生物学、病理生理学和治疗学

基本信息

批准号：
10614630
负责人：
Rachelle Hope Crosbie
金额：
$ 46.48万
依托单位：
UNIVERSITY OF CALIFORNIA LOS ANGELES
依托单位国家：
美国
项目类别：
财政年份：
2016
资助国家：
美国
起止时间：
2016-05-01 至 2026-04-30
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10614630
关键词：
Cellular biology Functional disorder Muscle Cells Therapeutic

项目摘要

Abstract This proposal is a competitive renewal application for a multidisciplinary training program in basic and translational research in muscle cell biology at UCLA from a group of highly engaged faculty mentors. The program in Muscle Cell Biology, Pathophysiology, and Therapeutics is focused on bridged training in the fields of muscle and numerous complementary fields and is the sole UCLA training program integrating fundamental laboratory-based research in muscle relevant fields with preclinical and clinical applications. The Muscle Research Center @UCLA is the hub of muscle focused research and educational activities. For many years, the muscle research community at UCLA has been galvanized by funding from two major center grants including the NIH P30 center grant and the NIH U54 Senator Paul D. Wellstone Center of Excellence grant that have been vital to the recruitment of new faculty members from different fields to apply their expertise to skeletal muscle diseases. Our trainees are further developed through activities of a top scoring, NIH-funded Clinical Science Translational Institute (CTSI). A multitude of unique, interactive, and highly integrated activities in muscle research serve as an outstanding resource for our trainees. The training program emphasizes training in the cellular, molecular, and genetic mechanisms of basic muscle function and disease. At both the pre- and postdoctoral levels, the Muscle Cell Biology, Pathophysiology, and Therapeutics main goals are to: 1) identify and support outstanding trainees with a high level of interest and focus in muscle cell biology research, 2) train scientists to conduct cutting-edge research in fundamental and clinical muscle biology, 3) provide trainees with a solid background in the biological sciences with an emphasis in fundamental physiology, immunology, biochemistry, biology, genetics, and now also stem cell biology and genomics, 4) facilitate career development by helping trainees obtain extramural support, and by guiding trainees in obtaining positions in academia and industry and other areas that suit their interests and skills, 5) introduce novel and significant projects for which beneficial outcomes will be derived, 6) acquaint trainees with state-of-the-art research through the training grant- supported fundamental and muscle relevant seminar series and associated journal clubs, and 7) provide trainees with regular opportunities to present their own research in seminar form and receive critical feedback from the training grant faculty and scientific community beyond UCLA at muscle and muscular dystrophy-related national and international meetings, and 8) implement assessments as part of a larger campus effort to develop tools for T32 training programs. UCLA has become a major site for preclinical and clinical trials in muscle disorders, using a variety of innovative approaches. The Neuromuscular Program at UCLA in partnership with the Center for Duchenne Muscular Dystrophy (CDMD) at UCLA has supported over twenty clinical trials in neuromuscular disorders. Thus, trainees are exposed to both basic and clinical aspects of muscle disease research, providing a well-rounded educational experience.

抽象的该提案是基础和多学科培训计划的竞争性更新申请。加州大学洛杉矶分校 (UCLA) 一群高度敬业的教师导师开展肌肉细胞生物学转化研究。这肌肉细胞生物学、病理生理学和治疗学项目专注于这些领域的桥梁培训肌肉和众多互补领域的训练项目，是加州大学洛杉矶分校唯一整合基础训练的项目肌肉相关领域的实验室研究及其临床前和临床应用。肌肉加州大学洛杉矶分校研究中心是专注于肌肉研究和教育活动的中心。多年来，加州大学洛杉矶分校的肌肉研究界受到两个主要中心拨款的资助，包括 NIH P30 中心拨款和 NIH U54 参议员 Paul D. Wellstone 卓越中心拨款对于招募来自不同领域的新教员以将他们的专业知识应用于骨骼肌至关重要疾病。我们的学员通过 NIH 资助的高分临床科学活动得到进一步发展转化研究所（CTSI）。大量独特、互动且高度集成的肌肉活动研究是我们学员的优秀资源。培训计划强调培训基本肌肉功能和疾病的细胞、分子和遗传机制。在前期和后期博士后水平，肌肉细胞生物学、病理生理学和治疗学的主要目标是：1）确定并支持对肌肉细胞生物学研究具有高度兴趣和关注的优秀学员，2）培训科学家在基础和临床肌肉生物学方面进行前沿研究，3）为学员提供具有扎实的生物科学背景，重点是基础生理学、免疫学、生物化学、生物学、遗传学，现在还有干细胞生物学和基因组学，4) 促进职业发展通过帮助学员获得校外支持，并指导学员在学术界和学术界获得职位行业和其他适合他们兴趣和技能的领域，5）介绍新颖且重要的项目将产生有益的成果，6）通过培训补助金让受训者了解最先进的研究- 支持基础和肌肉相关研讨会系列和相关期刊俱乐部，以及 7) 为学员提供定期有机会以研讨会的形式展示自己的研究成果，并获得来自学术界的批评性反馈加州大学洛杉矶分校以外的教师和科学界在肌肉和肌营养不良相关的国家培训补助金和国际会议，以及 8) 实施评估，作为校园开发工具的更大努力的一部分 T32 培训计划。加州大学洛杉矶分校已成为肌肉疾病临床前和临床试验的主要场所，利用各种创新方法。加州大学洛杉矶分校的神经肌肉项目与该中心合作加州大学洛杉矶分校的杜氏肌营养不良症 (CDMD) 已支持二十多项神经肌肉临床试验失调。因此，学员可以接触到肌肉疾病研究的基础和临床方面，从而提供全面的教育经历。

项目成果

期刊论文数量（5）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

Repurposing Dantrolene for Long-Term Combination Therapy to Potentiate Antisense-Mediated DMD Exon Skipping in the mdx Mouse.

DOI：
10.1016/j.omtn.2018.02.002
发表时间：
2018-06-01
期刊：
Molecular therapy. Nucleic acids
影响因子：
0
作者：
Wang DW;Mokhonova EI;Kendall GC;Becerra D;Naeini YB;Cantor RM;Spencer MJ;Nelson SF;Miceli MC
通讯作者：
Miceli MC

High-Throughput Screening to Identify Modulators of Sarcospan.

高通量筛选以鉴定 Sarcospan 调节剂。

DOI：
10.1007/978-1-0716-2772-3_25
发表时间：
2023
期刊：
Methods in molecular biology (Clifton, N.J.)
影响因子：
0
作者：
Shu,Cynthia;Mokhonova,Ekaterina;Crosbie,RachelleH
通讯作者：
Crosbie,RachelleH

Design and implementation of suspended drop crystallization.

DOI：
10.1107/s2052252523004141
发表时间：
2023-07-01
期刊：
IUCrJ
影响因子：
3.9
作者：
通讯作者：

Pacemaker translocations and power laws in 2D stem cell-derived cardiomyocyte cultures.

DOI：
10.1371/journal.pone.0263976
发表时间：
2022
期刊：
PloS one
影响因子：
3.7
作者：
Dunham CS;Mackenzie ME;Nakano H;Kim AR;Juda MB;Nakano A;Stieg AZ;Gimzewski JK
通讯作者：
Gimzewski JK

Development of a high-throughput screen to identify small molecule enhancers of sarcospan for the treatment of Duchenne muscular dystrophy.

开发高通量筛选来鉴定用于治疗杜氏肌营养不良症的肌跨小分子增强剂。

DOI：
10.1186/s13395-019-0218-x
发表时间：
2019
期刊：
Skeletal muscle
影响因子：
4.9
作者：
Shu,Cynthia;Kaxon-Rupp,ArianaN;Collado,JuddR;Damoiseaux,Robert;Crosbie,RachelleH
通讯作者：
Crosbie,RachelleH