Patent ID: 6506379
Filing Date: 2003-01-14
Classification: A61K,C07K,C12N

Abstract:
A method of expressing a selected gene in muscle cells present in a mammalian subject, said method comprising:(a) providing recombinant adeno-associated virus (AAV) particles which comprise a recombinant DNA construct comprising said selected gene operably linked to elements for controlling the expression of said selected gene; and (b) delivering said recombinant AAV particles intramuscularly to permit expression of said selected gene at a level which provides a therapeutic effect in the mammalian subject wherein the recombinant AAV particles also contain additional recombinant DNA constructs which encodes and directs the expression in said muscle cells of: (a) a first fusion protein containing one or more FK506-binding protein (FKBP) domains and a transcription activation domain, and (b) a second fusion protein containing an FKBP-rapamycin-binding (FRB) domain and a DNA binding domain which binds to a control element operably linked to said selected gene, wherein said fusion proteins permit rapamycin-dependent expression of the selected gene.