GENERAL CLINICAL RESEARCH CENTER

本提案支持一个八床的普通临床研究中心，其相关的核心实验室和门诊研究中心在大型医疗中心的儿童附属哈佛医学院。儿童医院有325家，每年约有18000名住院病人和30万名门诊病人。我们在临床研究方面的主要目标与我们改善儿童健康和福利的使命不谋而合，这在1996年David获得临床研究卓越奖时得到了体现。G. Nathan，我们的前首席调查员。我们的中心任务是将基础生物学应用于影响儿童的临床问题，通过利用这些知识来阐明疾病的机制，从而形成新的治疗方法。在过去五年的GCRC资助期间，我们已经开始看到血管生成研究的进展转化为血管和肿瘤疾病的新疗法。我们的研究人员已经确定了几种儿童免疫缺陷疾病的分子基础，以及神经和神经肌肉疾病，如Angelman综合征。我们已经利用氯霉唑调节离子通道的知识来设计一种潜在有效的治疗镰状细胞病的方法。我们继续评估为先天性心脏缺陷接受手术的儿童进行体外循环的最佳方法，并在我们对早产的理解方面取得了进展。我们已经使用移植物抗宿主病的动物模型来实施细胞因子拮抗剂的人体试验。在过去的五年中，该中心支持的协议数量增加了一倍多，未来五年的主要目标是继续刺激我们机构内临床研究的复苏，并促进我们的基础科学家和临床研究人员之间的联系，例如通过利用哈佛基因治疗计划的协议。我们认为，我们的中心代表了儿童医院最重要的使命，并充分利用了其优越的资源和环境。

This proposal of support for an eight bed General Clinical Research Center, its associated Core Laboratory and Outpatient Research Center in large medical center for children affiliated with Harvard Medical School. Children's Hospital has 325, with approximately 18,00 inpatient admissions and 300,000 outpatient visits annually. Our major goal in clinical investigation coincides with our mission to improve the health and welfare of children, which was exemplified when the 1996 Award for Excellence in Clinical Research was bestowed upon David. G. Nathan, our former Principal Investigator. Our central mission to apply basic biology to clinical problems as they affect children, by using this knowledge to elucidate mechanisms of disease which lead to the formation of novel therapies. During the past five year term of our GCRC grant, we have begun to see advances in research on angiogenesis translated into novel therapies for vascular and neoplastic disorders. Our investigators have defined the molecular basis for several immunodeficiency diseases of childhood, as well, as well as neurologic and neuromuscular disorders such as Angelman syndrome. We have used the knowledge that clotrimazole modulates ion channels to devise a potentially effective therapy for sickle cell disease. We continue to evaluate the best means for cardiopulmonary bypass for children undergoing surgery for congenital cardiac defects, and have made progress in our understanding of preterm labor. We have used animal models of graft versus host disease ti implement human trials of cytokine antagonists. During the past five years, the number of protocols supported by the Center has more than doubled, and a major goal of the next five years is to continue to stimulate the resurgence of clinical research within our institution, and to facilitate the links between our basic scientists and clinical investigators, such as via protocols which utilize the Harvard Gene Therapy Initiative. We believe that our Center represents the most important mission of Children's Hospital and makes excellent use of its outstanding resources and environment.