Gene- and cell-based approaches for neurodegenerative disease

Abstract

Neurodegenerative diseases comprise an important group ofchronic diseases that increase in incidence with rising age. In particular, the two most common neurodegenerative diseases are Alzheimer's disease and Parkinson's disease, both of which will be discussed below. A third, Huntington's disease, occurs infrequently, but has been studied intensely. Each of these diseases shares characteristics which are also generalizeable to other neurodegenerative diseases: accumulation ofproteinaceous substances that leads inexorably to selective neuronal death and decline in neural function. Treatments for these diseases have historically focused on symptomatic relief, but recent advances in molecular research have identified more specific targets. Additionally, stem cell therapy, immunotherapy and trophic-factor delivery provide avenues for neuronal protection that may alter the natural progression of these devastating illnesses. Upcoming clinical trials will evaluate treatment strategies and provide hope that translational research will decrease the onset of debilitating disability associated with neurodegenerative disease.