Maria B L, Medina C D, Hoang K B, Phillips M I
Neuro-Oncology Program, University of Florida Brain Institute, Gainesville, USA.
J Child Neurol. 1997 Jan;12(1):1-12. doi: 10.1177/088307389701200101.
The overall goal of this review is to provide the pediatric neurologist with a theoretical foundation in gene therapy. Gene therapy became feasible in the early 1970s and the first transfer of a foreign gene into humans was approved by the NIH in 1989. Adenovirus, adeno-associated virus, herpes-simplex virus, retroviruses, and other vectors have been used to efficiently transduce genes into cells in vitro and in vivo. We discuss laboratory experiments that have provided a strong scientific rationale for implementing human clinical trials of gene therapy for neurologic malignancy. The development of viral and nonviral vectors that mediate efficient gene insertion into human cells has created the prospect of using gene therapy for cancer or brain disease. The NIH has approved more than 100 gene therapy protocols since 1989. However, the field will require more research on gene delivery systems before gene therapy becomes an established therapeutic strategy for an array of central nervous system diseases.
本综述的总体目标是为儿科神经科医生提供基因治疗的理论基础。基因治疗在20世纪70年代初变得可行，1989年美国国立卫生研究院（NIH）批准了首次将外源基因转入人体。腺病毒、腺相关病毒、单纯疱疹病毒、逆转录病毒和其他载体已被用于在体外和体内有效地将基因转导到细胞中。我们讨论了一些实验室实验，这些实验为开展针对神经恶性肿瘤的基因治疗人体临床试验提供了强有力的科学依据。能够介导将基因有效插入人体细胞的病毒和非病毒载体的开发，为使用基因治疗癌症或脑部疾病带来了希望。自1989年以来，NIH已批准了100多个基因治疗方案。然而，在基因治疗成为一系列中枢神经系统疾病的既定治疗策略之前，该领域还需要对基因递送系统进行更多研究。
