Strategies of Gene Therapy

Ongoing research in gene therapy has segregated itself into several specific strategies of disease treatment. These include the viral transmission of enzymes in the dopamine synthesis pathway to restore local dopamine concentrations; the transmission of genes for neurotrophic factors such as GDNF and neurturin to support and protect remaining dopaminergic neurons; the delivery of genes like parkin designed to restore underlying genetic deficits; and the delivery of genes that alter the phenotypic function of local cells to perpetually balance the aberrant function of the basal ganglia. Each of these methods has benefits and it is possible that a combination will be most effective. All share the advantage that, if they are effective, they could provide a single intervention for PD without the need for pump refilling or battery changes and without the potential ethical complications of cellular transplantation therapies.

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