Przedborski Serge
Departments of Neurology and Pathology, and the Center for Neurobiology and Behavior, Columbia University, New York, NY 10032, USA.
Amyotroph Lateral Scler Other Motor Neuron Disord. 2004 Sep;5 Suppl 1:14-8. doi: 10.1080/1734470410019780.
Amyotrophic lateral sclerosis (ALS) is a fatal paralytic neurodegenerative disorder. Experimental models of ALS such as the transgenic rodents expressing mutant superoxide dimutase-1 are playing a pivotal role in our understanding of ALS pathogenesis, and in our testing of new therapeutic interventions aimed at protecting against neurodegeneration. Apoptosis has emerged as a significant pathogenic factor in several neurodegenerative diseases, including ALS. Constructed of multiple interacting molecules, the apoptosis machinery offers a host of attractive targets for pharmacological and genetic interventions to be tested in experimental models of ALS. Information generated by these pre-clinical studies holds the promise to provide sound scientific basis for the development of effective neuroprotective therapies for ALS.
肌萎缩侧索硬化症(ALS)是一种致命的麻痹性神经退行性疾病。诸如表达突变型超氧化物歧化酶-1的转基因啮齿动物等ALS实验模型,在我们对ALS发病机制的理解以及对旨在预防神经退行性变的新治疗干预措施的测试中发挥着关键作用。细胞凋亡已成为包括ALS在内的几种神经退行性疾病中的一个重要致病因素。细胞凋亡机制由多个相互作用的分子构成,为在ALS实验模型中进行测试的药理和基因干预提供了众多有吸引力的靶点。这些临床前研究产生的信息有望为开发有效的ALS神经保护疗法提供坚实的科学依据。