Muscle Cell Biology, Pathophysiology, and Therapeutics

肌肉细胞生物学、病理生理学和治疗学

• 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
• 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

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.

摘要 该提案是一个多学科培训计划的竞争性更新申请， 在加州大学洛杉矶分校的肌细胞生物学的转化研究，从一组高度参与教师导师。的 在肌肉细胞生物学，病理生理学和治疗方案的重点是在该领域的桥梁训练 肌肉和许多互补领域，是唯一的加州大学洛杉矶分校的训练计划，结合基本 肌肉相关领域的实验室研究，并具有临床前和临床应用。肌肉 研究中心@UCLA是肌肉为重点的研究和教育活动的枢纽。多年来 加州大学洛杉矶分校的肌肉研究社区受到两个主要中心赠款的资助， 美国国立卫生研究院P30中心拨款和美国国立卫生研究院U 54参议员保罗D. Wellstone卓越中心的赠款， 这对于从不同领域招募新的教师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得分最高，NIH资助的临床科学活动得到进一步发展 翻译研究所（CTSI）。众多独特的，互动的，高度整合的肌肉活动 研究为我们的学员提供了出色的资源。该培训计划强调培训， 基本肌肉功能和疾病的细胞、分子和遗传机制。在预处理和 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）识别 并支持优秀的学员与高水平的兴趣和重点在肌肉细胞生物学研究，2）培训 科学家进行基础和临床肌肉生物学的前沿研究，3）为学员提供 在生物科学与基础生理学，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 通过帮助受训者获得校外支持，并指导受训者在学术界获得职位， 行业和其他领域，适合他们的兴趣和技能，5）介绍新颖的和重要的项目， 6）通过培训补助金使受训人员了解最新的研究成果- 支持基本和肌肉相关的研讨会系列和相关的期刊俱乐部，以及7）为学员提供 定期有机会以研讨会的形式介绍自己的研究，并从 培训补助金教师和科学界超越加州大学洛杉矶分校在肌肉和肌肉萎缩症相关的国家 和国际会议，以及8）实施评估作为更大的校园努力的一部分，以开发工具， T32训练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， 各种创新方法。加州大学洛杉矶分校的神经肌肉项目与 加州大学洛杉矶分校的杜氏肌营养不良症（CDMD）已经支持了20多项神经肌肉 紊乱因此，学员接触到肌肉疾病研究的基础和临床方面， 全面的教育经历。

项目成果

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
• 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.)
• 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

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