Maria B L, Medina C D, Hoang K B, Phillips M I
Neuro-Oncology Program, University of Florida Brain Institute and Cancer Center, Gainesville, USA.
J Child Neurol. 1997 Feb;12(2):77-84. doi: 10.1177/088307389701200202.
Important advances in basic research have made it possible to examine the safety, toxicity, and efficacy of gene therapy in humans for over 5 years. The development of sophisticated gene delivery systems has resulted in approval by the Recombinant DNA Advisory Committee (RAC) of 125 gene therapy or gene marking studies. One of the primary applications of current retroviral-mediated gene insertion technology has been for malignant brain tumors. Studies are therefore underway to examine the efficacy of "suicide" gene therapy in children with recurrent brain tumors and adults with newly diagnosed or recurrent gliomas. Since a high proportion of genetic disorders produce neurologic dysfunction, gene therapy is likely to impact the management of neurologic disease in the foreseeable future. Patients with human immunodeficiency virus (HIV), Gaucher's disease, and Hunter syndrome are now enrolled in gene therapy trials. It will be challenging for the child neurologist to stay abreast of rapid developments in the field of gene therapy. By participating in the design and implementation of clinical trials in gene therapy, the neurologist may reduce the intense toll that several neurologic diseases take on children and their families.
基础研究的重要进展使得在人体中检测基因治疗的安全性、毒性和有效性成为可能，至今已有五年多时间。先进基因递送系统的开发已使125项基因治疗或基因标记研究获得了重组DNA咨询委员会（RAC）的批准。当前逆转录病毒介导的基因插入技术的主要应用之一是针对恶性脑肿瘤。因此，目前正在开展研究，以检验“自杀”基因疗法对复发性脑肿瘤儿童以及新诊断或复发性神经胶质瘤成人的疗效。由于很大一部分遗传疾病会导致神经功能障碍，基因疗法在可预见的未来可能会影响神经疾病的治疗。感染人类免疫缺陷病毒（HIV）、患有戈谢病和亨特综合征的患者目前已参加基因治疗试验。儿童神经科医生要跟上基因治疗领域的快速发展将具有挑战性。通过参与基因治疗临床试验的设计和实施，神经科医生可以减轻几种神经疾病给儿童及其家庭带来的沉重负担。
