LONG TERM FOLLOW-UP OF RECIPIENTS OF GENE TRANSFER

基因转移接受者的长期随访

• 批准号: 7716637
• 负责人: John A Zaia
• 金额: $ 0.48万
• 依托单位: UNIVERSITY OF SOUTHERN CALIFORNIA
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-04-20 至 2008-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7716637
• 关键词: Adverse effects; Aftercare; Blood specimen; Cells; Computer Retrieval of Information on Scientific Projects Database; Funding; Future; Gene Transfer; Grant; Institution; Learning; Length; Malignant Neoplasms; Methods; Purpose; Research; Research Personnel; Resources; Retroviridae; Safety; Sampling; Source; Testing; United States National Institutes of Health; Virus; experience; follow-up; gene therapy

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. Subjects who have participated in a prior gene transfer study will be asked to participate in this study. The purpose of this study is to complete follow-up and to learn about the long-term side effects of gene therapy. Participation in this study is expected to last for at least 15 years. The length of follow up may be extended in the future. Because there is so little experience with using gene transfer for treatment, all subjects who participated in gene transfer studies are expected to participate in long-term follow up. This is done so that any late problems that could be related to gene therapy can be evaluated. As part of this follow-up, investigators want to collect a blood sample each year that could be tested later for replication-competent retrovirus (RCR, virus from the original gene therapy that may be able to divide later and infect new cells on its own). These samples would be tested if there were a reason to suspect RCR. Gene therapy has been used for only a few years, and, because there is so little experience with this form of treatment, there is incomplete information about long-term safety. In one study which used a retrovirus gene transfer method, cancers occurred at approximately 3 years after treatment. This was associated with the retrovirus used. It is not known whether gene therapy can cause other late side effects.

该副本是利用众多研究子项目之一 由NIH/NCRR资助的中心赠款提供的资源。子弹和 调查员（PI）可能已经从其他NIH来源获得了主要资金， 因此可以在其他清晰的条目中代表。列出的机构是 对于中心，这不一定是调查员的机构。 参加了先前的基因转移研究的受试者将被要求参加这项研究。 这项研究的目的是完成随访并了解基因治疗的长期副作用。 参与这项研究至少将持续15年。将来可能会延长随访时间。 由于使用基因转移进行治疗的经验很少，因此所有参加基因转移研究的受试者都将参与长期随访。这样做是为了评估可能与基因疗法有关的任何后期问题。作为此随访的一部分，研究人员希望每年收集一个血液样本，以稍后进行复制能力逆转录病毒（RCR，原始基因疗法的病毒，该病毒可能能够以后分裂并独自感染新细胞）。如果有理由怀疑RCR，将对这些样品进行测试。基因疗法仅使用了几年，并且由于这种治疗形式的经验很少，因此关于长期安全的信息不完整。在使用逆转录病毒基因转移方法的一项研究中，癌症发生在治疗后大约3年。这与使用的逆转录病毒有关。尚不清楚基因疗法是否会引起其他晚期副作用。