Childhood Interstitial & Diffuse Lung Disease Scientific Conference

童年插页式

• 批准号: 8319294
• 负责人: James S. Hagood
• 金额: $ 2.3万
• 依托单位: UNIVERSITY OF CALIFORNIA, SAN DIEGO
• 依托单位国家: 美国
• 财政年份: 2012
• 资助国家: 美国
• 起止时间: 2012-06-01 至 2013-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8319294
• 关键词: Address; Adult; Advocate; Affect; Basic Science; Biological; Biological Markers; Biology; Biomedical Engineering; Biotechnology; California; Child; Childhood; Chronic Obstructive Airway Disease; Clinical; Development; Developmental Biology; Diffuse; Disabled Persons; Discipline; Disease; Educational workshop; Emerging Technologies; Epigenetic Process; Family; Functional disorder; Funding; Future; Genomics; Genotype; Glycogen Storage Disease; Growth; Hamman-Rich syndrome; Hyperplasia; Individual; Innovative Therapy; Interstitial Lung Diseases; Knowledge; Life; Lung; Lung diseases; Medicine; Molecular; Morbidity - disease rate; Mutation; Natural regeneration; Neonatology; Neuroendocrine Cell; Parents; Participant; Pathogenesis; Pathway interactions; Patients; Predictive Factor; Public Health; Rare Diseases; Research Personnel; Science; Scientist; Therapeutic; Underrepresented Minority; Work; abstracting; base; career; clinical phenotype; effective therapy; evidence base; infancy; interdisciplinary approach; interstitial; lung development; meetings; molecular phenotype; multidisciplinary; novel; novel strategies; patient advocacy group; patient population; posters; programs; respiratory; stem cell biology; surfactant; symposium; therapeutic target

DESCRIPTION (provided by applicant): Children with childhood diffuse and interstitial lung diseases (chILD) are afflicted with rare, life-threatening, high-morbidity disorders that are poorl understood, under-recognized, and have no known evidence-based therapy. Adult ILD paradigms do not generally apply to chILD entities, which include recently-described disorders such as surfactant dysfunction mutations (SDM), Pulmonary Interstitial Glycogenosis (PIG), and Neuroendocrine Cell Hyperplasia of Infancy (NEHI). However, many of these rare disorders hold clues to understanding normal lung biology, and may unravel aspects of more common diseases. Rare disorders have limited patient populations, requiring novel and interdisciplinary approaches to define clinical phenotypes, genotypes, molecular pathogenesis, predictive factors, biomarkers, and therapeutic targets. Breakthroughs in other rare diseases have recently been catalyzed by the efforts of multidisciplinary teams spurred on by passionate patient advocacy groups. There has not been an interdisciplinary scientific meeting focused on these entities in over five years. This workshop will bring together leaders from clinical, basic science and biotechnology fields, together with chILD family advocates, to promote novel cross-cutting approaches to promote scientific breakthroughs for these clinically vexing disorders. The conference will take place in the summer of 2012 in La Jolla, CA. The objectives of the conference are to: 1) showcase cutting-edge scientific knowledge regarding lung biology relevant to childhood diffuse and interstitial lung disease, including lung development, cellular programming, remodeling and regeneration, 2) promote and encourage young scientists, underrepresented minorities, and persons with disabilities to continue their work in this field and to pursue careers in science, 3) review emerging technologies that will drive breakthroughs for these rare disorders, and 4) promote novel interactions among the fields of developmental biology, stem cell biology, respiratory medicine, neonatology, genomics, epigenetics and bioengineering, that will ultimately facilitate development of innovative and effective therapies. The conference will bring together leaders and future leaders from these disciplines to address core questions surrounding lung growth, molecular pathogenesis of chILD disorders, and application of emerging technologies for molecular phenotyping and therapeutic discovery. The workshop will provide attendees with a look beyond current state of the art and a pathway to the future.

描述(由申请人提供)：患有儿童弥漫性和间质性肺部疾病的儿童(CHILL)患有罕见的、危及生命的、高发病率的疾病，这些疾病缺乏了解、认识不足，也没有已知的循证治疗。成人ILD范例一般不适用于儿童实体，包括最近描述的疾病，如肺表面活性物质功能障碍突变(SDM)、肺间质糖异症(PIG)和婴儿期神经内分泌细胞增生(NEHI)。然而，这些罕见的疾病中有许多掌握了了解正常肺部生物学的线索，并可能揭开更常见疾病的面纱。罕见疾病的患者群体有限，需要新的跨学科方法来定义临床表型、基因类型、分子发病机制、预测因素、生物标记物和治疗靶点。最近，在热情的患者倡导团体的推动下，多学科团队的努力促进了其他罕见疾病的突破。在五年多的时间里，没有举行过以这些实体为重点的跨学科科学会议。这次研讨会将汇集临床、基础科学和生物技术领域的领导人，以及儿童家庭倡导者，以促进新的交叉方法，以促进这些临床上令人烦恼的疾病的科学突破。会议将于2012年夏天在加利福尼亚州的拉荷亚举行。会议的目标是：1)展示与儿童弥漫性和间质性肺部疾病有关的肺生物学的尖端科学知识，包括肺发育、细胞编程、重塑和再生；2)促进和鼓励青年科学家、代表性不足的少数群体和残疾人继续在这一领域和 为了从事科学事业，3)审查将推动这些罕见疾病的突破的新兴技术，以及4)促进发育生物学、干细胞生物学、呼吸医学、新生儿学、基因组学、表观遗传学和生物工程等领域的新互动，最终将促进创新和有效疗法的开发。会议将汇集这些学科的领导者和未来的领导者，讨论围绕肺生长、儿童疾病的分子发病机制以及分子表型和治疗发现的新兴技术的应用等核心问题。研讨会将为与会者提供超越当前技术水平的展望和通往未来的途径。