Treating anemia using recombinant adeno-associated virus virions comprising an EPO DNA sequence
AssigneesAvigen, Inc., The Johns Hopkins University
InventorsGregory M. Podsakoff, Gary J. Kurtzman
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Abstract
The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue in the treatment of anemia is disclosed. The invention allows for the direct, in vivo injection of recombinant AAV virions into muscle tissue, e.g., by intramuscular injection, as well as for the in vitro transduction of muscle cells which can subsequently be introduced into a subject for treatment. The invention provides for sustained, high-level expression of a delivered nucleotide sequence encoding erythropoietin, and for in vivo secretion thereof from transduced muscle cells such that systemic delivery is achieved.
Claims34
A composition, comprising:
- (a) recombinant AAV virions which comprise an AAV vector containing a nucleic acid molecule encoding erythropoietin operably linked to control elements that direct the transcription and translation thereof;
- and
- (b) a pharmaceutically acceptable excipient.
A method of treating anemia in a mammalian subject, said method comprising:
- (a) introducing recombinant AAV virions into muscle cells in vitro to produce transduced muscle cells, wherein said recombinant AAV virions comprise an AAV vector having a nucleic acid molecule encoding erythropoietin operably linked to control elements that direct the transcription and translation thereof when present in muscle of said subject;
- and
- (b) administering directly into muscle of said subject a therapeutically effective amount of a composition comprising the transduced muscle cells from step (a) to deliver said transduced cells to muscle of said subject, whereafter said nucleic acid molecule is expressed by said transduced cells at a level sufficient to increase a hematocrit of said subject.
A method for delivering erythropoietin systemically to a mammalian subject, said method comprising:
- (a) introducing recombinant AAV virions into muscle cells in vitro to produce transduced muscle cells, wherein said recombinant AAV virions comprise an AAV vector having a nucleic acid molecule encoding erythropoietin operably linked to control elements that direct the transcription and translation thereof when present in muscle of said subject;
- and
- (b) administering directly into muscle of said subject a therapeutically effective amount of a composition comprising the transduced muscle cells from step (a) to deliver said transduced cells to muscle of said subject, whereafter said nucleic acid molecule is expressed by said transduced cells to produce a systemic level of erythropoietin, thereby providing for an increased hematocrit of said subject.
Dependencies are inferred from the claim text.