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WO1997026337B1 - Methods for delivering dna to muscle cells using recombinant adeno-associated virus virions - Google Patents

Methods for delivering dna to muscle cells using recombinant adeno-associated virus virions

Info

Publication number
WO1997026337B1
WO1997026337B1 PCT/US1997/000895 US9700895W WO9726337B1 WO 1997026337 B1 WO1997026337 B1 WO 1997026337B1 US 9700895 W US9700895 W US 9700895W WO 9726337 B1 WO9726337 B1 WO 9726337B1
Authority
WO
WIPO (PCT)
Prior art keywords
muscle cell
muscle
control elements
virion
selected gene
Prior art date
Application number
PCT/US1997/000895
Other languages
French (fr)
Other versions
WO1997026337A1 (en
WO1997026337A9 (en
Filing date
Publication date
Priority claimed from US08/588,355 external-priority patent/US5858351A/en
Priority claimed from US08/784,757 external-priority patent/US5962313A/en
Application filed filed Critical
Priority to EP97904823A priority Critical patent/EP0874904A1/en
Priority to JP52626797A priority patent/JP2002514896A/en
Priority to CA2243261A priority patent/CA2243261C/en
Publication of WO1997026337A1 publication Critical patent/WO1997026337A1/en
Publication of WO1997026337B1 publication Critical patent/WO1997026337B1/en
Publication of WO1997026337A9 publication Critical patent/WO1997026337A9/en

Links

Abstract

The use of recombinant adeno-associated virus (AAV) virions for delivery of DNA molecules to muscle cells and tissue 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 the delivered gene and for in vivo secretion of the therapeutic protein from transduced muscle cells such that systemic delivery is achieved.

Claims

AMENDED CLAIMS[received by the International Bureau on 10 August 1997 (10.08.97); original claims 1-23 replaced by new claims 1-22 (4 pages)]
1. A method for producing a composition useful for delivering a selected gene to a smooth muscle cell or to smooth muscle tissue, said method comprising:
(a) providing a recombinant adeno-associated virus (AAV) virion which comprises an AAV vector having the selected gene operably linked to control elements capable of directing the in vivo transcription and translation thereof; and
(b) combining the recombinant AAV virion with a pharmaceutically acceptable vehicle.
2. The method of claim 1, characterized in that the selected gene encodes a therapeutic protein.
3. The method of claim 1 or 2, characterized in that the control elements comprise a muscle-specific promoter sequence.
4. The method of claim 1 or 2, characterized in that the control elements comprise an inducible promoter sequence.
5. A smooth muscle cell transduced with a recombinant AAV virion, said virion comprising an AAV vector having a selected gene operably linked to control elements capable of directing the in vivo transcription and translation of said selected gene.
6. The transduced muscle cell of claim 5, characterized in that the selected gene encodes a therapeutic protein.
- 63 -
7. The transduced muscle cell of claim 5 or 6, characterized in that the control elements comprise a muscle-specific promoter sequence.
8. The transduced muscle cell of claim 5 or
6, characterized in that the control elements comprise an inducible promoter sequence.
9. A method of transducing a mammalian smooth muscle cell, said method comprising:
(a) providing a recombinant AAV virion which comprises an AAV vector having a selected gene operably linked to control elements capable of directing the in vivo transcription and translation of said selected gene; and
(b) introducing the recombinant AAV virion into a suitable smooth muscle cell to produce a transduced muscle cell.
10. A method for producing a composition useful for delivering to a muscle cell or muscle tissue a gene encoding acid α-glucosidase, said method comprising:
(a) providing a recombinant adeno-associated virus (AAV) virion which comprises an AAV vector having said gene operably linked to control elements capable of directing the in vivo transcription and translation thereof; and
(b) combining the recombinant AAV virion with a pharmaceutically acceptable vehicle.
11. The method of claim 10, characterized in that the control elements comprise a muscle-specific promoter sequence .
- 64 -
12. The method of claim 10, characterized in that the control elements comprise an inducible promoter sequence.
13. The method of claim 10, characterized in that the composition is useful for delivering a selected gene to a muscle cell or tissue derived from skeletal muscle.
14. The method of claim 13, characterized in that the muscle cell is a skeletal myoblast or a skeletal myocyte.
15. The method of claim 10, characterized in that the composition is useful for delivering a selected gene to a muscle cell or tissue derived from smooth muscle.
16. The method of claim 10, characterized in that the composition is useful for delivering a selected gene to a muscle cell or tissue derived from cardiac muscle.
17. The method of claim 16, characterized in that the muscle cell is a cardiomyocyte.
18. A muscle cell transduced with a recombinant AAV virion, said virion comprising an AAV vector having a gene encoding acid α-glucosidase operably linked to control elements capable of directing the in vivo transcription and translation thereof.
- 65 -
19. A method of transducing a mammalian muscle cell, comprising:
(a) providing a recombinant AAV virion which comprises an AAV vector having a gene encoding acid - glucosidase operably linked to control elements capable of directing the in vivo transcription and translation thereof; and
(b) introducing the recombinant AAV virion into a suitable muscle cell to produce a transduced muscle cell.
20. The method of claim 19, characterized in that the muscle cell is derived from skeletal muscle.
21. The method of claim 19, characterized in that the muscle cell is derived from smooth muscle.
22. The method of claim 19, characterized in that the muscle cell is derived from cardiac muscle.
- 66 -
PCT/US1997/000895 1996-01-18 1997-01-17 Methods for delivering dna to muscle cells using recombinant adeno-associated virus virions WO1997026337A1 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
EP97904823A EP0874904A1 (en) 1996-01-18 1997-01-17 Methods for delivering dna to muscle cells using recombinant adenoassociated virus virions
JP52626797A JP2002514896A (en) 1996-01-18 1997-01-17 Method for delivery of DNA to muscle cells using recombinant adeno-associated virus virions
CA2243261A CA2243261C (en) 1996-01-18 1997-01-17 Methods for delivering dna to muscle cells using recombinant adeno-associated virus virions

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US08/588,355 1996-01-18
US08/588,355 US5858351A (en) 1996-01-18 1996-01-18 Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors
US08/784,757 US5962313A (en) 1996-01-18 1997-01-16 Adeno-associated virus vectors comprising a gene encoding a lyosomal enzyme
US08/784,757 1997-01-16

Publications (3)

Publication Number Publication Date
WO1997026337A1 WO1997026337A1 (en) 1997-07-24
WO1997026337B1 true WO1997026337B1 (en) 1997-10-02
WO1997026337A9 WO1997026337A9 (en) 1997-10-30

Family

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Family Applications (1)

Application Number Title Priority Date Filing Date
PCT/US1997/000895 WO1997026337A1 (en) 1996-01-18 1997-01-17 Methods for delivering dna to muscle cells using recombinant adeno-associated virus virions

Country Status (5)

Country Link
US (7) US5962313A (en)
EP (1) EP0874904A1 (en)
JP (1) JP2002514896A (en)
CA (1) CA2243261C (en)
WO (1) WO1997026337A1 (en)

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