Breathing Research and Therapeutics (BREATHE)

摘要 在这个修改后的申请，我们提出了一个新的前和博士后培训计划在大学的 佛罗里达：呼吸研究和治疗学（呼吸）。我们的建议是建立一个培训计划 专注于呼吸神经肌肉系统，强调新知识的发现及其 转化为影响呼吸和气道防御的神经肌肉疾病。呼吸减弱 呼吸能力、呼吸不稳定和/或呼吸道防御不足引起的吸入性肺炎是 许多神经肌肉疾病，呼吸衰竭是最常见的死亡原因。但尽管 呼吸和气道防御对患者生活质量和持续时间的根本重要性 退行性神经肌肉疾病（例如，肌营养不良症、庞贝氏症、ALS）或神经损伤（如 脊髓损伤），我们知道没有其他训练计划与类似的重点呼吸和气道防御， 在这种情况下。我们的提案直接解决了NHLBI资助的研究培训中的这一关键差距。的 BREATHE培训计划的最终目标是培养一批具有强大 呼吸神经肌肉生物学基础研究的基础，以及对 在我们试图开发呼吸受损治疗方法的转化研究中的需求/现实，和/或 气道防御我们的观点是，我们可以加快进展， 通过培训研究者治疗不同神经肌肉疾病中呼吸能力/稳定性受损 具有：1）完善和全面的概念框架，包括相似性和差异性 之间的神经肌肉临床疾病;和2）一个国家的最先进的“工具包”，提供技术知识， 如何在动物模型和患有自发性疾病的人类中进行有意义的研究。我们将 培养具有不同学术背景的个人，包括神经科学，肌肉生物学，工程学 和/或临床培训（医生，物理或语言治疗师和兽医;包括多个 各专业）。我们计划的一个标志是“交叉培训”，基础科学家将 接触有意义的临床经验，临床科学家建立强大的基础研究 层.我们将利用现有的，并在佛罗里达大学建设优势，提供一个 呼吸神经肌肉生物学和转化研究的综合研究培训计划。的UF 健康科学中心通过创建一个新的中心， 呼吸研究和康复。这个中心，以及在UF健康科学中心的其他优势， 将为呼吸培训计划提供一个特殊的学术环境。

ABSTRACT In this revised application, we propose a new pre- and post-doctoral training program at the University of Florida: Breathing REsearch And THErapeutics (BREATHE). Our proposal is to create a training program focused on the respiratory neuromuscular system, emphasizing the discovery of new knowledge and its translation to neuromuscular disorders that compromise breathing and airway defense. Diminished breathing capacity, unstable breathing and/or aspiration pneumonia from inadequate airway defense are hallmarks of many neuromuscular disorders, and respiratory failure is the most common cause of death. However, despite the fundamental importance of breathing and airway defense to the quality and duration of life in patients with degenerative neuromuscular diseases (eg. Muscular Dystrophy, Pompe Disease, ALS) or neural injury (eg. spinal cord injury), we know of no other training program with similar focus on breathing and airway defense in in these conditions. Our proposal directly addresses this critical gap in NHLBI funded research training. The ultimate goal of the BREATHE Training Program is to develop a unique cohort of researchers with strong foundations in basic research on respiratory neuromuscular biology, and a strong appreciation for the needs/realities of translational research in our attempts to develop treatments for impaired breathing and/or airway defense. Our perspective is that we can accelerate progress towards development of effective treatments for impaired breathing capacity/stability in diverse neuromuscular disorders by training investigators with: 1) a well-developed and comprehensive conceptual framework embracing similarities and differences between neuromuscular clinical disorders; and 2) a state-of-the-art “tool kit,” providing the technological know- how to perform meaningful investigations in animal models and humans with spontaneous disease. We will train individuals with diverse academic backgrounds, including neuroscience, muscle biology, engineering and/or clinical training (physicians, physical or speech therapists and veterinarians; including multiple specialties in each group). A hallmark of our program will be “cross-training,” where basic scientists are exposed to meaningful clinical experiences, and clinician scientists establish strong basic research foundations. We will capitalize on existing, and building strengths at the University of Florida to provide an integrated research-training program in respiratory neuromuscular biology and translational research. The UF Health Science Center has demonstrated its commitment to this area of research by creating a new Center for Respiratory Research and Rehabilitation. This Center, and other strengths at the UF Health Science Center, will provide an exceptional academic environment for the BREATHE Training Program.