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Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific M

先天性肌营养不良症：从临床病理学到基础科学 M

基本信息

批准号：
8319246
负责人：
DEAN J. BURKIN
金额：
$ 2.85万
依托单位：
UNIVERSITY OF NEVADA RENO
依托单位国家：
美国
项目类别：
财政年份：
2012
资助国家：
美国
起止时间：
2012-04-01 至 2013-03-31
项目状态：
已结题

来源：
https://reporter.nih.gov/project-details/8319246
关键词：
Address Advocacy Animal Model Apoptosis Area Basic Science Biochemical Biological Assay Biomechanics Cell Communication Cell Differentiation process Clinical Clinical Pathology Collaborations Collagen Type VI Connective Tissue Consensus Contracture Cytoskeleton Development Disease Disease model Drug Industry Educational workshop Extracellular Matrix Extracellular Matrix Proteins Fibrosis Funding Future Generations Genetic Genotype Goals Inflammation International Knowledge Laminin Lead Life Longevity Mediator of activation protein Membrane Modeling Modification Molecular Muscle Muscular Dystrophies Mutation Myocardium Myopathy Natural regeneration Neuraxis Neuromuscular Junction Nevada Organelles Outcome Pathogenesis Pathology Pathway interactions Patients Pharmacologic Substance Phenotype Play Process Quality of life Receptor Signaling Relative (related person)Research Research Personnel Role Scientist Screening procedure Signal Transduction Skeletal Muscle Therapeutics for Rare and Neglected Diseases Translational Research United States National Institutes of Health Universities alpha Dystroglycan biobank comparative congenital muscular dystrophy design drug discovery high throughput screening interest lens link protein medical schools mitochondrial dysfunction muscle regeneration neuromuscular novel patient registry physical property research and development satellite cell stem cell niche symposium therapeutic development therapeutic target therapy development

项目摘要

DESCRIPTION (provided by applicant): This application seeks NIH funding to support a conference titled "Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific Mechanisms, Exploring the Role of the Myomatrix" which will be held at the University of Nevada School of Medicine from Sunday April 22 to Tuesday April 24, 2012. This conference will bring together basic scientists, clinicians, advocacy groups and pharmaceutical industry representatives with expertise in the matrix and muscular dystrophies in general, those with expertise in forms of CMD more specifically, and those from outside the immediate neuromuscular field but with expertise that is relevant to the CMDs. This is a unique conference dedicated to the intersection of muscle and extracellular matrix both in development and disease states, specifically muscular dystrophy and CMD. The conference will combine two keynote addresses with seven core sessions: matrix in development, matrix and regeneration, matrix and intracellular organelles, matrix biomechanical and physical properties, matrix and signaling receptors, matrix and the neuromuscular junction. The conference will end with a panel discussion to build a myomatrix roadmap, unifying key themes from prior sessions, providing opportunities for productive collaborations and identifying pathways towards translational research for the congenital muscular dystrophies. PUBLIC HEALTH RELEVANCE: This application seeks funding to support a conference titled "Congenital Muscular Dystrophy: From Clinical Pathology to Underlying Scientific Mechanisms, Exploring the Role of the Myomatrix". The primary conference goal is to address, evaluate and achieve consensus on contributing mechanisms of the myomatrix that drive key clinical pathology relevant to the quality of life and life span of patients with congenital muscular dystrophies. Conference deliverables include defining new focus areas, bringing together researchers that may otherwise not have a chance to communicate or collaborate and using the congenital muscular dystrophy clinical context to frame research queries into translational targets.

描述（由申请人提供）：本申请寻求NIH资助，以支持一个名为“先天性肌肉萎缩症：从临床病理学到潜在的科学机制，探索肌基质的作用”的会议，该会议将于2012年4月22日星期日至4月24日星期二在内华达大学医学院举行。本次会议将汇集基础科学家、临床医生、倡导团体和制药行业代表，他们一般具有基质和肌肉萎缩症方面的专业知识，他们具有更具体的CMD形式的专业知识，以及来自直接神经肌肉领域以外但具有与CMD相关的专业知识。这是一个独特的会议，致力于肌肉和细胞外基质在发育和疾病状态的交集，特别是肌肉萎缩症和CMD。会议将结合两个主题演讲和七个核心会议：基质发育，基质和再生，基质和细胞内细胞器，基质生物力学和物理特性，基质和信号受体，基质和神经肌肉连接。会议将以小组讨论结束，以建立肌基质路线图，统一之前会议的关键主题，为富有成效的合作提供机会，并确定先天性肌营养不良症的转化研究途径。