Latchman D S, Coffin R S
Institute of Child Health, University College London, UK.
Rev Neurosci. 2001;12(1):69-78. doi: 10.1515/revneuro.2001.12.1.69.
The ability of transplanted neurons from aborted foetuses to produce some therapeutic benefit in Parkinson's disease makes this disease an obvious target for the development of gene therapy procedures which involve delivering the same factors as are provided by the foetal neurons but using a reagent which could be produced in large amounts in a standardised manner. This approach could involve both the delivery of the gene encoding tyrosine hydroxylase to boost dopamine production or the delivery of genes encoding neurotrophic factors such as GDNF to promote the survival of dopaminergic neurons. A variety of different viral and non-viral methods for achieving such gene delivery has been described. These are discussed together with the particular advantages of herpes simplex virus-based vectors which have the potential to deliver multiple therapeutic genes in a single virus vector.
来自流产胎儿的移植神经元能够在帕金森病中产生一定治疗效果,这使得该疾病成为基因治疗程序开发的一个明显目标。基因治疗程序涉及传递与胎儿神经元所提供的相同因子,但使用能够以标准化方式大量生产的试剂。这种方法可能包括递送编码酪氨酸羟化酶的基因以促进多巴胺生成,或者递送编码神经营养因子(如胶质细胞源性神经营养因子)的基因以促进多巴胺能神经元的存活。已经描述了多种实现这种基因递送的不同病毒和非病毒方法。这些方法将与基于单纯疱疹病毒的载体的特殊优势一起进行讨论,这种载体有可能在单个病毒载体中递送多个治疗基因。