LONG TERM FOLLOW-UP OF RECIPIENTS OF GENE TRANSFER

本子项目是利用由NIH/NCRR资助的中心赠款提供的资源的众多研究子项目之一。子项目和研究者（PI）可能已经从另一个NIH来源获得了主要资金，因此可以在其他CRISP条目中表示。列出的机构是中心的，不一定是研究者的机构。已参加过先前基因转移研究的受试者将被要求参加本研究。本研究的目的是完成随访并了解基因治疗的长期副作用。参与这项研究预计将持续至少15年。后续随访时间可能会延长。由于使用基因转移进行治疗的经验太少，所有参与基因转移研究的受试者都应参与长期随访。这样做是为了评估任何可能与基因治疗相关的晚期问题。作为后续工作的一部分，研究人员希望每年收集一份血液样本，以便以后检测具有复制能力的逆转录病毒（RCR，来自原始基因治疗的病毒，可能会在以后分裂并自行感染新细胞）。如果有理由怀疑RCR，将对这些样本进行检测。基因疗法只使用了几年，而且由于这种治疗形式的经验太少，关于长期安全性的信息也不完整。在一项使用逆转录病毒基因转移方法的研究中，癌症发生在治疗后大约3年。这与使用的逆转录病毒有关。目前尚不清楚基因治疗是否会导致其他晚期副作用。

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.