直接转基因技术应用于神经系统基因治疗的研究进展

Advance of Gen Therapy for Neurological Diseases by Direct in vivo Gene Transfer

神经系统疾病的基因治疗是目前神经科学中发展比较迅速的一个领域。近年来研究发现，一些来源于单纯疱疹病毒、腺病毒和腺病毒相关病毒等的重组病毒表达载体能够将外源基因直接导入在体或离体培养的神经细胞。已有实验证明这类病毒载体可以感染很多种不分裂细胞（包括神经元）。此外，直接转基因技术还包括物理化学方法。直接转基因技术应用于神经系统对于在分子水平研究神经生理功能、神经系统疾病的发病机理和病理变化以及在分子水平对其进行治疗提供了直接的途径，有希望使神经系统疾病的基因治疗获得突破性进展。

Gene therapy for neurological diseases is a rapidly expanding field in neurosciences.It has been demonstrated that some viral vectors from HSV-1(herpos simplex virus type 1),Ad(adenovirus)and AAV(adeno-associated virus)can transfer foreign genes into nondividing cell types(including neurons).In addition,physical/chemical methods are also used in direct in vivo gene transfer.The direct gene transfer techniques will open a new way to study neurophysiology,neuropathology and therapy for neurological diseases at molecular level.They will hopefully lead to surprising progress in gene therapy for neurological diseases.