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US 2003/0143702 A1Published application
Vesicular monoamine transporter gene therapy in parkinson's disease
AssigneeArch Development Corporation
InventorUn Jung Kang
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Abstract
The present invention provides methods and compositions for the therapeutic intervention of Parkinson's disease. More particularly, methods of making and sequestering dopamine are disclosed. Additionally, methods of genetically modifying donor cells by gene transfer for grafting into the central nervous system to treat defective, diseased or damaged cells are disclosed. Methods and compositions for carrying out such gene transfer and grafting are described.
Claims33
A method for producing dopamine in a cell comprising:
- a) transforming a cell with a first polynticleotide encoding aromatic L-amino acid decarboxylase (AADC) and a second polynucleotide encoding vesicular monoamine transporter (VMAT) under conditions suitable for the expression of AADC and VMAT, wherein said polynucleotides each are under the transcriptional control of a promoter active in eukaryotic cells;
- and
- b) contacting said cell with L-3,4-dihydroxyphenylalanine (L-DOPA),
- whereby AADC converts L-DOPA to dopamine and VMAT sequesters said dopamine in endosomes in said cell.
1 more element
A method of treating Parkinson's disease in a subject comprising:
- a) obtaining a cells from said subject;
- b) transforming said cells with a first polynucleotide encoding L-amino acid decarboxylase (AADC) and a second polynucleotide encoding VMAT under conditions suitable for the expression of AADC and VMAT, wherein said polynucleotides each are under the transcriptional control of a promoter;
- c) implanting transformed cells into said subject;
- and
- d) providing L-DOPA to said subject,
- whereby AADC converts L-DOPA in vivo to dopamine and VMAT sequesters said dopamine in endosomes in said cell, which sequestered dopamine over a longer duration of time than from cells without storage of L-DOPA.
3 more elements
A kit comprising:
- a) a polynucleotide encoding aromatic L-amino acid decarboxylase (AADC);
- and
- b) a polynucleotide encoding vesicular monoamine transporter (VMAT),
- said polynucleotides being located in suitable container means therefor.
1 more element
Dependencies are inferred from the claim text.