Vector encoding therapeutic polypeptide and safety elements...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) A61P 3/00 (2006.01)

Patent

CA 2584494

A composition comprising: a stably integrating delivery vector; an modified mammalian thymidylate kinase (tmpk) activator polynucleotide wherein the modified mammalian tmpk polynucleotide encodinges a modified mammalian tmpk polypeptide that increases phosphorylation of converts a prodrug relative to phosophorylation of the prodrug by wild-type mammalian tmpk polypeptideto a drug; and/or a targeting polynucleotide encoding a cell surface polypeptide that selectively binds a toxic binding agent. The invention also relates to use of these compositions in methods of treatment of diseases such as Fabry disease.

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