Tuszynski Mark H
Department of Neurosciences, University of California at San Diego, and the Veterans Administration Medical Center-San Diego, La Jolla 92093, USA.
Lancet Neurol. 2002 May;1(1):51-7. doi: 10.1016/s1474-4422(02)00006-6.
Preclinical neuroscience has advanced rapidly over the past two decades. New approaches for treating neurological disease, including gene-based therapies, nervous-system growth factors, stem cells, novel vaccines, and modulation of the immune system, offer the potential to prevent cell loss and degeneration in the brain, rather than attempting to compensate for loss after it has occurred. I will review one of these prospective therapies: growth-factor gene therapy for Alzheimer's disease, an approach that is currently the subject of a phase I clinical trial. Other disease targets for gene therapy will also be discussed, including Parkinson's disease, Huntington's disease, inborn errors of metabolism, and cancer. The progress of gene-therapy clinical trials is aiding the transition to molecular and gene-targeted therapeutic approaches which have the potential to improve dramatically the prognosis of neurological disease.
在过去二十年中，临床前神经科学发展迅速。治疗神经疾病的新方法，包括基于基因的疗法、神经系统生长因子、干细胞、新型疫苗以及免疫系统调节，提供了预防大脑细胞损失和退化的潜力，而不是在损失发生后试图进行补偿。我将回顾这些前瞻性疗法之一：用于阿尔茨海默病的生长因子基因疗法，这一方法目前正处于I期临床试验阶段。还将讨论基因疗法的其他疾病靶点，包括帕金森病、亨廷顿病、先天性代谢缺陷和癌症。基因疗法临床试验的进展正在推动向分子和基因靶向治疗方法的转变，这些方法有可能显著改善神经疾病的预后。
