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[Perspectives on postgenome medicine: Neurodegenerative disease]
1Department of Degenerative Neurological Diseases, National Institute of Neuroscience, National Center of Neurology and Psychiatry.
Abstract:
The genes of the majority neurodegenerative disorders have been identified for the past decade. Diseases of early onset are usually transmitted as autosomal recessive trait and caused by the deficiency of the gene products, becoming good candidates for gene or protein transfers. Late onset diseases are usually transmitted dominantly where the gains of function by the mutant gene accumulate into specific lesion. The pathophysiology following gene mutation need to be clarified for the therapeutic approach. Recent progresses on neurodegenerative mechanism have specified crucial targets for the treatments. Stimulation to intrinsic mechanisms, i.e., chaperon, ubiquitin-proteasome system and stress response in endoplasmic reticulum and/or antagonize the toxic cascade by caspases, mitochondria insufficiency are argued as therapeutic targets, hoping that in this decade we can propose effective therapies for these devastating disorders.