Systemic synthesis and regulation of l-dopa
AssigneeMyodopa Limited
InventorMichael McDonald
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Abstract
The present invention relates to an expression system for enzyme replacement therapy with the aim of obtaining or maintaining a steady level of L-DOPA in the blood of an individual, achieved through systemic administration of the expression system. The invention is thus useful in the treatment of catecholamine deficient disorders, such as dopamine deficient disorders including Parkinson's Disease.
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An expression system comprising:
- a first polynucleotide (N1) which upon expression encodes a GTP-cyclohydrolase 1 (GCH1;
- EC 3.5.4.16) polypeptide or a biologically active fragment or variant thereof, wherein said polynucleotide is operably linked to a first promoter, and wherein the biological activity is enzymatic activity of GCH1;
- and
- a second polynucleotide (N2) which upon expression encodes a tyrosine hydroxylase (TH;
- EC 1.14.16.2) polypeptide or a biologically active fragment or variant thereof, wherein said polynucleotide is operably linked to a second promoter, and wherein the biological activity is enzymatic activity of TH;
- and
- a third polynucleotide (N3) which upon expression encodes a 6-pyruvoyltetrahydropterin synthase (PTPS, EC 4.2.3.12) polypeptide or a biologically active fragment or variant thereof, wherein said polynucleotide is operably linked to a third promoter, and wherein the biological activity is enzymatic activity of PTPS.
Dependencies are inferred from the claim text.