Muscle Cell Biology, Pathophysiology, and Therapeutics

 描述（由申请人提供）：该提案是加州大学洛杉矶分校肌细胞生物学基础和转化研究多学科培训计划的新申请，由一群高度敬业的教师导师组成。该计划在肌肉细胞生物学，病理生理学和治疗学的重点是在肌肉和许多互补领域的桥接训练，是唯一的UCLA培训计划整合基础实验室研究在肌肉相关领域与临床前和临床应用。为 多年来，加州大学洛杉矶分校杜氏肌营养不良症中心（CDMD）一直是至关重要的招聘新的教员来自不同领域，以应用他们的专业知识，骨骼肌疾病。CDMD利用加州大学洛杉矶分校的国际公认的地位，成为DMD临床前和临床试验的主要中心，这些试验使用各种创新方法，包括外显子跳跃和PDE 5抑制剂。我们已经从校内和校外获得了大量的配套资金，以进一步推进我们的培训使命。此外，我们的学员通过活动和得分最高的NIH资助的临床科学转化研究所（CTSI）的发展得到进一步发展。因此，培训计划提供了一个强大的补充基本，翻译和临床研究的分子机制肌营养不良症在加州大学洛杉矶分校。在肌肉研究中，许多独特的，互动的和高度集成的活动是我们学员的优秀资源。该培训计划强调肌营养不良症的细胞，分子和遗传机制的培训，重点是杜氏肌营养不良症作为模型。在前-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）识别和支持对肌细胞生物学研究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）为学员提供生物科学的坚实背景，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）通过帮助受训者获得校外支持，并指导受训者在学术界、工业界和其他适合其兴趣和技能的领域获得职位，促进职业发展，5）引入能够产生有益成果的新颖和重要项目，6）使受训者了解国家 通过培训补助金支持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，以及7）为学员提供定期机会，以研讨会形式展示他们自己的研究，并在肌肉和肌肉营养不良相关的国家和国际会议上接受来自加州大学洛杉矶分校以外的培训补助金教师和科学界的关键反馈，对培训方案和受训人员进行评估，以便向广大科学和教育界宣传。

 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.