Vector encoding therapeutic polypeptide and safety elements to clear transduced cells
OwnerJeffrey A. Medininventor
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Abstract
A composition comprising: a stably integrating delivery vector; an modified mammalian thymidylate kinase (tmpk) activator polynucleotide wherein the modified mammalian tmpk polynucleotide encodings a modified mammalian tmpk polypeptide that increases phosphorylation of converts a prodrug relative to phosphorylation of the prodrug by wild-type mammalian tmpk polypeptide to a drug; and/or a targeting polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent. The disclosure also relates to use of these compositions in methods of treatment of diseases such as Fabry disease.
Claims34
A suicide gene system comprising:
- a) a stably integrating delivery vector;
- b) an activator polynucleotide encoding a polypeptide that converts a prodrug to a drug;
- and/or
- c) a docking polynucleotide encoding a docking polypeptide that selectively binds a toxic binding agent;
- wherein the suicide gene system induces death in a cell expressing the activator polynucleotide and/or docking polynucleotide when the cell is contacted with the prodrug and/or the toxic binding agent.
4-5. (canceled)
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23-24. (canceled)
A method of killing a mammalian cell expressing an activator polynucleotide and/or a docking polynucleotide comprising contacting the cell with an effective amount of a prodrug and/or a toxic binding agent to kill the cell.
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An actuable cell destruction component of an expression vector construct comprising:
- a) an activator polynucleotide encoding a polypeptide that converts a prodrug to a drug;
- and/or
- b) a docking polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent.
31-34. (canceled)
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41-44. (canceled)
Dependencies are inferred from the claim text.