Muscle Cell Biology, Pathophysiology, and Therapeutics

 描述(由申请人提供)：这份提案是加州大学洛杉矶分校一群高度投入的教师导师提出的关于肌肉细胞生物学基础和翻译研究的多学科培训计划的新申请。肌肉细胞生物学、病理生理学和治疗学课程专注于肌肉和众多互补领域的桥梁培训，是加州大学洛杉矶分校唯一的将肌肉相关领域的基础实验室研究与临床前和临床应用相结合的培训课程。为 多年来，加州大学洛杉矶分校杜兴氏肌营养不良症中心(CDMD)对于从不同领域招募新的教职员工将他们的专业知识应用于骨骼肌疾病至关重要。CDMD利用加州大学洛杉矶分校的国际公认地位，成为DMD临床前和临床试验的主要中心，这些试验使用了各种创新方法，包括外显子跳过和PDE5抑制剂。我们从内部和外部来源筹集了大量配套资金，以进一步推进我们的培训任务。此外，我们的受训人员通过NIH资助的临床科学翻译研究所(CTSI)的活动和发展得到进一步发展。因此，该培训计划作为加州大学洛杉矶分校肌营养不良分子机制的基础、翻译和临床研究的有力补充。众多独特的、互动的和高度整合的肌肉研究活动为我们的受训者提供了出色的资源。该培训计划强调对肌营养不良症的细胞、分子和遗传机制的培训，重点是以Duchenne肌营养不良症为模型。在博士后和博士后阶段，肌肉细胞生物学、病理生理学和治疗学的主要目标是：1)识别和支持对肌肉细胞生物学研究具有高度兴趣和关注的优秀受训人员，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)为受训人员提供坚实的生物科学背景，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)通过帮助受训人员获得校外支持，并通过指导受训人员在学术和工业以及其他适合他们的兴趣和技能的领域获得职位，促进职业发展，5)引入新的、有意义的项目，这些项目将产生有益的结果；6)让学员熟悉国家 通过培训基金资助的基础和肌肉相关研讨会系列和相关期刊俱乐部进行最先进的研究；7)为学员提供定期的机会，以研讨会形式介绍他们自己的研究成果，并在加州大学洛杉矶分校以外的培训拨款教员和科学界收到与肌肉和肌肉营养不良相关的国内和国际会议上的批判性反馈；以及8)对培训计划和学员进行评估，以便向广大科学和教育界传播。

 DESCRIPTION (provided by applicant): This proposal is a new 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. For many years, the UCLA Center for Duchenne Muscular Dystrophy (CDMD) has been vital to the recruitment of new faculty members from different fields to apply their expertise to skeletal muscle diseases. The CDMD has leveraged UCLA's internationally recognized standing to become a major center in DMD preclinical and clinical trials that use a variety of innovative approaches including exon skipping and PDE5 inhibitors. We have garnered significant matching funds from intramural and extramural sources to further advance our training mission. In addition, our trainees are further developed through activities and development of a top scoring, NIH-funded Clinical Science Translational Institute (CTSI). Thus, the training program provides as a strong complement of basic, translational, and clinical investigations in molecular mechanisms of muscular dystrophy at UCLA. 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 muscular dystrophy, with a focus on Duchenne muscular dystrophy as a model. 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 of the training program as well as trainees for dissemination to the science and education community at large.