Muscle Cell Biology, Pathophysiology, and Therapeutics

摘要 这项提案是对基础和专业培训计划的竞争性续签申请。 加州大学洛杉矶分校一群高度投入的教师导师在肌肉细胞生物学方面的转化研究。这个 肌肉细胞生物学、病理生理学和治疗学课程的重点是在实地进行衔接训练 肌肉和众多互补领域，是加州大学洛杉矶分校唯一整合基础 以实验室为基础的肌肉相关领域的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以肌肉为重点的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社区得到了两个主要中心的资助，其中包括 NIH P30中心赠款和NIH U54参议员保罗·D·威尔斯通卓越中心赠款 对于从不同领域招聘新的教员将他们的专业知识应用于骨骼肌至关重要 疾病。我们的受训人员通过NIH资助的高分临床科学活动得到进一步发展 翻译学院(CTSI)。多种独特的、互动的、高度整合的肌肉活动 研究对我们的学员来说是一种杰出的资源。该培训计划强调的是在 肌肉基本功能和疾病的细胞、分子和遗传机制。在预赛和预赛中 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专注的优秀学员，2)培训 科学家将在基础和临床肌肉生物学方面进行尖端研究，3)为学员提供 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，促进了职业发展 通过帮助受训人员获得校外支持，并指导受训人员在学术界和 行业和其他适合他们的兴趣和技能的领域，5)为其引入新的和重要的项目 将产生有益的结果，6)通过培训补助金让学员熟悉最先进的研究- 支持基础和肌肉相关的系列研讨会和相关的期刊俱乐部，并提供学员 定期有机会以研讨会的形式介绍他们自己的研究，并从 加州大学洛杉矶分校以外的肌肉和肌营养不良相关国家的培训补助金教职员工和科学界 和国际会议，以及8)实施评估，作为开发工具的更大校园努力的一部分 T32培训计划。加州大学洛杉矶分校已经成为肌肉疾病临床前和临床试验的主要地点，使用 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兴氏肌营养不良症(CDMD)已经支持了20多项神经肌肉临床试验 精神错乱。因此，受训者接触到肌肉疾病研究的基础和临床方面，提供 全方位的教育体验。

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.