GENERAL CLINICAL RESEARCH CENTER

这项提议是为了继续支持八个床位的将军 临床研究中心及其附属实验室和门诊 哈佛附属一家大型儿童医疗中心的诊所 医学院有329张床位，大约有16,565名学生 以及每年共258,015人次的门诊就诊。我们的核心目标是 临床研究与医院的使命是相称的 这是一个旨在改善儿童健康和福利的机构。我们 努力了解疾病的分子和细胞基础 并将这些知识应用于临床研究， 将评估新疗法的疗效。我们在过去的进步 五年来，我们在基因治疗方面取得了进步 慢性肉芽肿性疾病、库利氏贫血和囊性疾病 纤维化对免疫缺陷状态的影响和对 补充代谢，并建立了治疗川崎综合征的方法。 我们研究所的研究已经为 杜兴氏和贝克勒肌营养不良症。这项工作构成了 提供准确的诊断并提供潜在的新治疗方法 机遇只有在理解 疾病的基础。我们继续进行纵向协议检查 同性性性早熟和I型糖原储存疾病 作为评估慢性肾功能衰竭儿童的生长发育和 研究β细胞休息是否可以预防高血压性I型糖尿病 有风险的个人。在该中心设立一个睡眠单位将 允许开发新的方案，如神经内分泌和 青少年重度抑郁障碍的睡眠功能研究。一个 相当大的努力集中在中枢神经系统的后遗症 婴幼儿心脏手术中的循环骤停。一项重大举措 涉及对艾滋病毒的各种治疗方法的组合进行评估 感染是自然研究的一部分。造血生长的应用 骨髓衰竭综合征相关因素和细胞因子的研究进展 在骨髓移植中的新疗法以及对 α-干扰素治疗高危血管瘤的临床研究 婴儿期，所有这些都是在床边结束的长凳研究中发展起来的。 我们相信，我们的中心代表着 儿童医院和发挥其卓越的潜力 资源环境。

This proposal is for continuation of support for an eight bed General Clinical Research Center and its associated laboratory and ambulatory clinic in a large medical center for children affiliated with Harvard Medical School comprising 329 beds with approximately 16,565 admission and a total of 258,015 outpatient visits per year. Our central goal in clinical investigation is commensurate with the mission of the institution which is to improve the health and welfare of children. We strive to understand the molecular and cellular basis of disorders that affect children and apply that knowledge to clinical investigation that will evaluate the efficacy of novel therapies. Our progress in the last five years indicates that we have improved the treatment in genetic disorders like Chronic Granulomatous Disease, Cooley's Anemia, and Cystic Fibrosis, impacted on immunodeficiency states and disturbances of complement metabolism, and established a treatment for Kawasaki Syndrome. Studies from our institution has established the molecular basis for Duchenne's and Beckler's Muscular Dystrophy. This work forms the basis for accurate diagnosis and provides potentially new therapeutic opportunities made possible only by the progress in understanding the basis of disease. We continued longitudinal protocols examining Isosexual Precocious Puberty, and Type I Glycogen Storage Disease as well as evaluating growth in children with Chronic Renal Failure and investigating whether beta cell rest may prevent type I diabetes in high risk individuals. The establishment of a sleep unit at this Center will allow for the development of novel protocols such as a neuroendocrine and sleep function study in adolescents with major depressive disorders. A considerable effort focuses on Central Nervous System sequelae of circulatory arrest during infant cardiac surgery. A major initiative involves the evaluation of combinations of various therapies in HIV infection as part of a natural study. The use of hematopoietic growth factors and cytokines in bone marrow failure syndromes, the evaluation of novel therapies in bone marrow transplantation as well as the use of alfa interferon in the treatment of life-threatening hemangiomas of infancy, all developed from bench research that ended at the bedside. We believe that our Center represents the most important mission of the Children's Hospital and utilizes the potential of its outstanding resources and environment.