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: 9925057
• 负责人: Rachelle Hope Crosbie
• 金额: $ 30.59万
• 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 2021-05-06
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9925057
• 关键词: Cellular biology; Functional disorder; Muscle Cells; Therapeutic

 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.

 描述（由申请人提供）：该提案是加州大学洛杉矶分校肌肉细胞生物学基础和转化研究多学科培训项目的新申请，该项目由一群高度参与的教师导师组成，该项目涉及肌肉细胞生物学、病理生理学和治疗学。专注于肌肉领域和众多补充领域的桥梁培训，是加州大学洛杉矶分校唯一的培训计划，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。 多年来，加州大学洛杉矶分校杜氏肌营养不良症中心 (CDMD) 在招募来自不同领域的新教员以将其专业知识应用于骨骼肌疾病方面发挥着至关重要的作用。CDMD 利用加州大学洛杉矶分校的国际公认地位，成为杜氏肌营养不良症的主要中心。使用多种创新方法（包括外显子跳跃和 PDE5 抑制剂）的临床前和临床试验，我们已从校内和校外来源获得了大量配套资金，以进一步推进我们的培训任务。此外，我们的学员通过 NIH 资助的高分临床科学转化研究所 (CTSI) 的活动和发展得到进一步发展。因此，培训计划为肌肉分子机制的基础、转化和临床研究提供了强有力的补充。加州大学洛杉矶分校的肌营养不良症研究中心有大量独特、互动且高度综合的肌肉研究活动，为我们的学员提供了出色的资源。该培训计划强调肌营养不良症的细胞、分子和遗传机制方面的培训，重点是杜氏肌营养不良症。肌肉发达的在博士前和博士后水平上，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有高度兴趣和关注的优秀学员，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，现在还干细胞生物学和基因组学，4）通过帮助学员获得校外支持，并指导学员在学术界、工业界以及其他适合其兴趣和技能的领域获得职位，促进职业发展，5）介绍新颖且重要的项目，并带来有益的成果将得出，6）让受训者熟悉状态 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，7）为学员提供定期机会以研讨会的形式展示自己的研究成果，并从培训补助金教师和科学人员那里获得重要反馈加州大学洛杉矶分校以外的社区参加与肌肉和肌营养不良症相关的国家和国际会议，8) 对培训计划和受训人员进行评估，以便向整个科学和教育界传播。