Latchman D S
Institute of Child Health, University College London.
Neuroscientist. 2001 Dec;7(6):528-37. doi: 10.1177/107385840100700609.
Gene delivery to the nervous system represents perhaps the ultimate challenge of gene therapy in view of the complexity of this system, the wide variety of intractable neurological diseases, and the need to deliver the gene to nondividing cells. Although a variety of systems for such gene delivery are under development, herpes simplex virus has unique advantages in terms of its large genome size and its ability to naturally enter a latent state in neuronal cells. Considerable progress has been made in the effective disablement of this virus while retaining its ability to deliver genes and in producing long-term expression of the foreign gene. It is likely that these viruses may ultimately be of use in human gene therapy procedures for otherwise intractable neurological diseases such as Parkinson's disease.
鉴于神经系统的复杂性、各种难治性神经疾病以及将基因传递至非分裂细胞的需求,向神经系统进行基因传递或许代表了基因治疗的终极挑战。尽管多种用于此类基因传递的系统正在研发中,但单纯疱疹病毒在其基因组较大以及能够自然进入神经元细胞潜伏状态方面具有独特优势。在有效使这种病毒失活同时保留其传递基因的能力以及实现外源基因的长期表达方面已取得了相当大的进展。这些病毒最终有可能用于治疗诸如帕金森病等原本难治的神经疾病的人类基因治疗程序中。