The genetics of neurodegenerative diseases

Abstract

In the last 50 years, an enormous amount of progress has been made in dissecting the etiology of hereditary neurodegenerative diseases, including the dementias, the parkinsonisms, the ataxias and the motor-neuron diseases. In addition, these genetic findings are beginning to provide insights into the pathogeneses of the sporadic forms of the diseases. Through animal and cellular modeling studies we are beginning to gain insights into the pathogenic pathways to disease. This mechanistic understanding is now leading to therapeutic strategies based on this new understanding. As yet, however, no mechanistic therapies are in use in the clinic.