Gordon Conference on Myogenesis: Advanced mechanisms of growth and repair

摘要 骨骼肌肌发生领域在过去几年中经历了巨大的发展， 包括一些令人兴奋的神经肌肉疾病的新疗法。最近，美国食品和药物管理局 美国政府批准了第一种治疗杜氏肌营养不良症的药物。这一点非常重要，因为 在此之前，骨骼肌被认为是人体内最后一个可药用的器官。 没有针对性的治疗。随着这一突破，其他机会等待治疗肌肉萎缩症 营养不良和其它骨骼肌消耗状况。与杜氏肌营养不良症类似， 突破可能来自基础科学的重大发现。在骨骼肌发生中， 来自于扩展我们对肌肉细胞在胚胎发育和出生后如何发育的知识基础， 生长，肌肉干细胞如何促进骨骼肌修复，肌肉如何与其他细胞类型相互作用 如肌肉微环境中的血管和运动神经元，以及肌肉干细胞和 肌纤维调节它们的新陈代谢和细胞存活。基础研究将导致确定关键的 基因和信号通路在这些领域中的每一个，并最终设计新的疗法。的 关于肌生成的国际会议，戈登研究会议（GRC）历来被视为 这是一个杰出的骨骼肌会议，吸引了来自世界各地的演讲者和与会者。 此R13提案的目的是为即将举行的GRC肌生成会议寻求部分支持： 高级生长和修复机制，将在文艺复兴时期的托斯卡纳伊尔乔科度假村卢卡举行， 意大利，2017年6月11日至16日。本次会议的总体目标是汇集各种专业知识，包括 领导人在我们的领域，教育和激励参与和讨论之间的所有会议与会者。 这一目标将通过实现两个具体目标来实现。目标1将邀请演讲者参加 GRC交换关于骨骼肌稳态的潜在机制的新信息， 在发育、疾病和衰老中的周转。这些主题将通过九个科学 会议侧重于胚胎和出生后发育，再生，细胞融合，肌肉/基质 相互作用，能量调节，重塑，转录和表观基因组学，以及 神经肌肉疾病此外，四个晚上的海报会议将激发所有人之间的互动 与会者将专门为初级科学家提供一个论坛，以展示他们的工作并获得反馈 从其他参与者。目标2将通过戈登研究丰富初级科学家的教育 研讨会（GRS），为期两天的研究生和博士后只有会议，发生在紧接着之前， GRC。GRS将举办四场研讨会和两场研究生海报会议， 博士后这些活动为下一代科学家提供了一个独特的机会， 展示他们对肌生成的研究，与他们的同龄人互动，并形成新的合作。实现这些 aims将使该领域处于更有利的地位，以加速新疗法的开发。

ABSTRACT The field of skeletal muscle myogenesis has undergone a tremendous growth in the last several years, including some exciting new therapies in neuromuscular diseases. Very recently, the US Food and Drug Administration approved the first drug to treat Duchenne muscular dystrophy. This is highly significant, as before this approval, skeletal muscle had been considered the last remaining druggable organ in the human body without a targeted therapy. With this breakthrough, other opportunities await for the treatment of muscular dystrophy and other skeletal muscle wasting conditions. Similar to Duchenne muscular dystrophy, the next breakthroughs will likely derive from fundamental discoveries in basic science. In skeletal myogenesis, this will come from expanding our knowledge base for how muscle cells develop during embryogenesis and postnatal growth, how muscle stem cells contribute to skeletal muscle repair, how muscle interacts with other cell types such as blood vessels and motor neurons in the muscle microenvironment, and how muscle stem cells and myofibers regulate their metabolism and cell survival. Basic research will lead to the identification of critical genes and signaling pathways in each of these areas, and ultimately to the design of new therapies. The international meeting on Myogenesis, a Gordon Research Conference (GRC) has historically been viewed as one of the preeminent skeletal muscle meetings, attracting speakers and attendants from around the world. The purpose of this R13 proposal is to seek partial support for the upcoming GRC conference on Myogenesis: Advanced Mechanisms of Growth and Repair, to be held at the Renaissance Tuscany Il Ciocco Resort Lucca, Italy, on June 11-16, 2017. The overall goal for this conference is to bring together diverse expertise, including leaders in our field, to educate and stimulate participation and discussion among all conference attendees. This goal will be accomplished by performing two specific aims. Aim 1 will convene invited speakers to the GRC to exchange new information on the underlying mechanisms of skeletal muscle homeostasis and turnover in development, disease, and aging. These topics will be communicated through nine scientific sessions focusing on embryonic and postnatal development, regeneration, cell fusion, muscle/stroma interactions, energy regulation, remodelling, transcription and epigenomics, and basic mechanisms in neuromuscular disorders. In addition, four evening poster sessions will stimulate interactions between all attendees will specifically providing a forum for junior scientists to present their work and receive feedback from other participants. Aim 2 will enrich the education of junior scientists through the Gordon Research Seminar (GRS), a two-day graduate and postdoc only conference that takes place immediately preceding the GRC. The GRS will conduct four sessions of talks and two poster sessions by graduate students and postdoctoral fellows. These activities represent a unique opportunity for the next generation of scientists to present their research on myogenesis, interact with their peers, and form new collaborations. Achieving these aims will place the field in a better position to accelerate the development of new therapies.