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 virionsInfo
- 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
Links
- 210000000663 muscle cells Anatomy 0.000 title claims abstract 21
- 210000002845 Virion Anatomy 0.000 title claims abstract 15
- 241000432074 Adeno-associated virus Species 0.000 title claims abstract 4
- 229920003013 deoxyribonucleic acid Polymers 0.000 title abstract 2
- 210000003205 Muscles Anatomy 0.000 claims abstract 5
- 210000001519 tissues Anatomy 0.000 claims abstract 5
- 102000004169 proteins and genes Human genes 0.000 claims abstract 3
- 108090000623 proteins and genes Proteins 0.000 claims abstract 3
- 230000001225 therapeutic Effects 0.000 claims abstract 3
- 230000035897 transcription Effects 0.000 claims 6
- 210000000329 Myocytes, Smooth Muscle Anatomy 0.000 claims 4
- 210000002464 Muscle, Smooth, Vascular Anatomy 0.000 claims 3
- 239000002253 acid Substances 0.000 claims 3
- 230000001939 inductive effect Effects 0.000 claims 3
- 210000002027 Muscle, Skeletal Anatomy 0.000 claims 2
- 210000004165 Myocardium Anatomy 0.000 claims 2
- 102000016679 alpha-Glucosidases Human genes 0.000 claims 2
- 108010028144 alpha-Glucosidases Proteins 0.000 claims 2
- 238000004519 manufacturing process Methods 0.000 claims 2
- 230000002463 transducing Effects 0.000 claims 2
- 102000004366 Glucosidases Human genes 0.000 claims 1
- 108010056771 Glucosidases Proteins 0.000 claims 1
- 210000004683 Myoblasts, Skeletal Anatomy 0.000 claims 1
- 210000004413 Myocytes, Cardiac Anatomy 0.000 claims 1
- 210000000587 Skeletal Muscle Fibers Anatomy 0.000 claims 1
- 210000002317 cardiac myocyte Anatomy 0.000 claims 1
- 210000003795 skeletal myoblast Anatomy 0.000 claims 1
- 238000000338 in vitro Methods 0.000 abstract 1
- 238000002347 injection Methods 0.000 abstract 1
- 239000007924 injection Substances 0.000 abstract 1
- 238000010255 intramuscular injection Methods 0.000 abstract 1
- 239000007927 intramuscular injection Substances 0.000 abstract 1
- 230000028327 secretion Effects 0.000 abstract 1
- 230000002459 sustained Effects 0.000 abstract 1
- 230000026683 transduction Effects 0.000 abstract 1
- 238000010361 transduction Methods 0.000 abstract 1
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
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 -
Priority Applications (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
JP52626797A JP2002514896A (en) | 1996-01-18 | 1997-01-17 | Method for delivery of DNA to muscle cells using recombinant adeno-associated virus virions |
EP97904823A EP0874904A1 (en) | 1996-01-18 | 1997-01-17 | Methods for delivering dna to muscle cells using recombinant adenoassociated 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 US5858351A (en) | 1996-01-18 | 1996-01-18 | Methods for delivering DNA to muscle cells using recombinant adeno-associated virus vectors |
US08/588,355 | 1996-01-18 | ||
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
ID=27080262
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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-
1997
- 1997-01-16 US US08/784,757 patent/US5962313A/en not_active Expired - Lifetime
- 1997-01-17 EP EP97904823A patent/EP0874904A1/en not_active Ceased
- 1997-01-17 WO PCT/US1997/000895 patent/WO1997026337A1/en not_active Application Discontinuation
- 1997-01-17 JP JP52626797A patent/JP2002514896A/en not_active Withdrawn
- 1997-01-17 CA CA2243261A patent/CA2243261C/en not_active Expired - Lifetime
-
1999
- 1999-09-28 US US09/406,362 patent/US6335011B1/en not_active Expired - Lifetime
-
2001
- 2001-10-01 US US09/969,204 patent/US6610290B2/en not_active Expired - Lifetime
-
2003
- 2003-05-23 US US10/445,088 patent/US20030219415A1/en not_active Abandoned
-
2005
- 2005-12-15 US US11/303,896 patent/US20060099184A1/en not_active Abandoned
-
2007
- 2007-10-30 US US11/980,214 patent/US20080069803A1/en not_active Abandoned
-
2008
- 2008-07-15 US US12/218,394 patent/US7704492B2/en not_active Expired - Fee Related
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