Vector encoding therapeutic polypeptide and safety elements to clear transduced cells
AssigneeUniversity Health Network
InventorJeffrey A. Medin
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Abstract
A composition comprising: a stably integrating delivery vector; a modified mammalian thymidylate kinase (tmpk) activator polynucleotide wherein the modified mammalian tmpk polynucleotide encodes a modified mammalian tmpk polypeptide that increases phosphorylation of 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.
Claims35
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;
- c. a docking polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent;
- and
- d. a therapeutic α-galactosidase A polynucleotide;
- 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.
A composition comprising:
- a. a stably integrating delivery vector;
- b. an activator polynucleotide encoding a polypeptide that converts a prodrug to a drug;
- c. a docking polynucleotide encoding a docking polypeptide that selectively binds an antibody or an antibody conjugated with a toxic agent;
- and
- d. a therapeutic α-galactosidase A polynucleotide.
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;
- b. a docking polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent;
- and
- c. a therapeutic α-galactosidase A polynucleotide.
Dependencies are inferred from the claim text.