Method of controlling L-Dopa production and of treating dopamine deficiency
AssigneeCell Genesys, Inc.
InventorsRonald J. Mandel, Stuart E. Leff
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Abstract
The present invention provides an effective approach to achieve the tightly modulated production of L-DOPA and/or dopamine at a preselected target location in the brain of a mammal by combining gene therapy approaches to supply a key enzyme in the synthesis of L-DOPA, and novel drug delivery modalities to administer a uniform level of a modulator of the activity of such key enzyme. The fine-tuned administration of the modulator establishes continuously uniform levels of modulator which in turn allow the effective modulation of L-DOPA and/or dopamine levels at a preselected target location in the brain of the mammal.
Claims15
A method for increasing brain L-DOPA levels in a mammal, comprising the steps of:
- (a) introducing into a preselected location of the brain of the mammal a polynucleotide encoding tyrosine hydroxylase or a functional derivative of tyrosine hydroxylase that converts tyrosine to L-DOPA;
- and
- (b) peripherally administering a modulator of the activity of the tyrosine hydroxylase or functional derivative of tyrosine hydroxylase at continuously regulated levels,
- wherein the administration of the modulator results in increasing brain L-DOPA levels in the mammal.
Dependencies are inferred from the claim text.