CORE SUPPORT AS AN NGVL TOXICOLOGY CENTER

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。描述(申请人提供)：单个基因的缺失或突变可能导致特定的疾病。人类基因组的测序导致了巨大的努力来识别和分离与各种疾病的病因学有关的特定基因，包括癌症、艾滋病、糖尿病和囊性纤维化。在过去的十年里，已经开发了许多基因疗法来用正常的、有功能的基因取代有缺陷或缺失的基因。虽然基因疗法在治疗和/或治愈各种疾病方面具有巨大的潜力，但它也不是没有风险，正如1999年所证明的那样，当时一名青少年成为该国第一个已知的直接死于基因疗法的人。

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. DESCRIPTION (provided by applicant): Deletion or mutation of a single gene may result in a specific disease. Sequencing of the human genome has led to huge efforts to identify and isolate specific genes involved in the etiology of various diseases, including cancer, AIDS, diabetes, and cystic fibrosis. During the past decade, a number of gene therapies have been developed to replace defective or deleted genes with normal, functional genes. While gene therapy holds great potential for the treatment and/or cure of various diseases, it is not without risk, as demonstrated in 1999 when a teenager became the first known person in the country to die as a direct result of gene therapy.