Drug – bio-affecting and body treating compositions – Whole live micro-organism – cell – or virus containing – Genetically modified micro-organism – cell – or virus
Patent
1996-01-18
1999-01-12
Crouch, Deborah
Drug, bio-affecting and body treating compositions
Whole live micro-organism, cell, or virus containing
Genetically modified micro-organism, cell, or virus
424 9321, 514 44, 4353201, 935 62, A61K 4800, C12N 500, C12N 1500
Patent
active
058583513
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.
REFERENCES:
patent: 5139941 (1992-08-01), Muzyczka et al.
patent: 5173414 (1992-12-01), Lebrowski et al.
patent: 5252479 (1993-10-01), Srivatava
patent: 5478745 (1995-12-01), Samulski et al.
patent: 5587308 (1996-12-01), Carter et al.
patent: 5589362 (1996-12-01), Bujavd et al.
patent: 5658656 (1997-08-01), Billiar et al.
Miller et al., FASE B J. 9:190-199, 1995.
Marshal, Science 269:1050-1055, 1995.
Culver et al., Trends Genetics 10(5): 174-178, 1994.
Hodgson, Exp. Opin. Ther. Pat. 5(5): 459-468, 1995.
Einerhand et al., Gene Therapy 2(5): 336-343, 1995.
Xiao et al., J. Virol. 70(11):8098-8108, 1996.
Xiao et al., Adv. Drug Del. Rev. 12:201-215, 1993.
Kaplitt et al., Nature Genetics 8:148-153, 1994.
Bartlett et al., Am. J. Human Genetics, 57, Supp. 4 #A235 1995.
March et al., Clin. Res. 40(2): 358A, 1992.
LaFont, Lancet, 346:1442-1443, 1995.
Blau et a., "Myoblast in Pattern Formation and Gene Therapy," Trends in Genetics 9(9):269 (1993).
Clark et al., "Cell Lines for the Production of Recombinant Adeno-Associated Virus," Human Gene Therapy 6:1329-1341 (1995).
Hamamori et al., "Persistent Erythropoiesis by Myoblast Transfer of Erythropoietin cDNA," Human Gene Therapy 5:1349-1356 (1994).
Kessler et al., "Gene Delivery to Skeletal Muscle Results in Sustained Experssion and Systemic Delivery of a Therapeutic Protein," Proc. Natl. Acad. Sci. USA 93: 14082-14087 (1996).
Kourtis et al., "Cardiac Gene Therapy With Adeno-Associated Virus Suppression," Modern Pathology 8(1):33A (1995).
Naffakh et al., "Sustained Delivery of Erythropoietin in Mice by Genetically Modified Skin Fibroblasts," Proc. Natl. Acad. Sci. USA 92:3194-3198 (1995).
Naffakh et al., "Long-Term Secretion of Therapeutic Proteins from Genetically Modified Skeletal Muscles," Human Gene Therapy 7:11-21 (1996).
Osborne et al., "Gene Therapy for Long-Term Expression of Erythropoietin in Rats," Proc. Natl. Acad. Sci. USA 92:8055-8058 (1995).
Podsakoff et al., "AAV Vector-Mediated Gene Delivery to Skeletal Muscle In Vivo Results in Sustained Levels of Systemic Erythropoietin," Gene Therapy Clinic 88(10:1066-(269A) (1996.
Podsakoff et al., "Long-Term In Vivo Gene Expression in Muscle Using AAV Vectors," Blood 86:(10):1004A (1995).
Tripathy et al., "Stable Delivery of Physiologic Levels of Recombinant Erythropoietin to the Systemic Circulation by Intramuscular Injection of Replication-Defective Adenovirus," Proc. Natl. Acad. Sci.USA 91:115.
Herzog et al (1997) Proced. Natl. Acad. Sci. 94,5804-5809.
Synder et al (1997) Nature Gent.16, 270-276.
Acsadi et al. "Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs," Nature (1991) 352:815-818.
Acsadi et al. "Cultured human myoblasts and myotubes show markedly different transducibility by replication-defective adenovirus recombinants," Gene Therapy (1994) 1:338-340.
Acsadi et al. "A differnetial efficiency of adenovirus-mediated in vivo gene transfer into skeletal muscle cells of different maturity," Hum. Mol. Genetics (1994) 3:579-584.
Barr and Leiden "Systemic delivery of recombinant proteins by genetically modified myoblasts," Science (1991) 254:1507-1509.
Blau and Springer "Molecular medicine muscle-mediated gene therapy," New Eng. J. Med. (1995) 333:1204-1207.
Blau and Springer "Molecular medicine gene therapy-a novel form of drug delivery," New Eng. J. Med. (1995) 333:1554-1556.
Dai et al. "Gene therapy via primary myoblasts: long-term expression of factor IX protein following transplantation in vitro," Proc. Natl. Acad. Sci. USA (1992) 89:10892-10895.
Dai et al. "Cellular and humoral immune responses to adenoviral vectors containing ractor IX gene: tolerization of factor IX and vector antigens allows for long-term expression," Proc. Natl. Acad. Sci. USA (1995) 92:1401-1405.
Davis et al. "Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression," Hum. Gene. Therapy (1993) 4:151-159.
Descamps et al. "Organoids direct systemic expression of erythropoietin in mice," Gene Therapy (1995) 2:411-417.
Dhawan et al. "Systemic delivery of human growth hormone by injection of genetically engineered myoblasts," Science (1991) 254:1509-1512.
Flotte et al. "Gene expression from Adeno-associated virus vectors in airway epithelial cells," Am. J. Respir. Cell Mol. Biol. (1992) 7:349-356.
Flotte et al. "Expression of the cystic fibrosis transmenbrane conductance regulator from a novel adeno-associated virus promoter," J. Biol. Chem. (1993) 268:3781-3790.
Flotte et al. "Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector," Proc. Natl. Acad. Sci. USA (1993) 90:10613-10617).
Flotte et al. "Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration," Am. J. Respir. Cell Mol. Biol. (1994) 11:517-521.
Gilgenkrantz et al. "Transient expression of genes transferred in vivo into heart using first-generation adenoviral vectors: role of the immune response," Hum. Gene Therapy (1995) 6:1265-1274.
Hamamori et al. "Persistent erythropoiesis by myoblast transfer of erythropietin cDNA," Hum. Gene Therapy (1994) 5:1349-1356.
Hamamori et al. "Myoblast transfer of human erythropoietin gene in a mouse model of renal failure," J. Clin, Invest. (1995) 95:1808-1813.
Kaplitt et al. "Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain," Nature Genetics (1994)8:148-154.
Knowles et al. "A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis," The New Eng. J. of Med. (1995) 333:(13)823-831.
Leiden, J.M. "Gene therapy-promise, pitfalls and prognosis," New Eng. J. Med. (1995) 333:871-872.
Mendell et al. "Myoblast transfer in the treatment of Duchenne's muscular dystrophy," New. Eng. J. Med. (1995) 333:832-838.
Podsakoff et al. "Efficient gene transfer into nondividing cells by adeno-associated virus-based vectors;" J. Virol. (1994) 68:5656-5666.
Quantin et al. "Adenovirus as an expression vector in muscle cells in vivo," Proc. Natl. Acad. Sci. USA (1992) 89:2581-2584.
Raz et al. "Systemic immunological effects of cytokine genes injected into skeletal muscle," Proc. Natl. Acad. Sci. USA (1993) 90:4523-4527.
Russell et al. "Adeno-associated virus vectors preferentially transduce cells in S phase," Proc. Natl. Acad. Sci. USA (1994) 91:8915-8919.
Villeval et al. "Retrovirus-medicated transfer of the erythropoietin gene in hematopoietic cells improves the erythrocyte phenotype in murine .beta.-thalassemia," Blood (1994) 84(3):928-933.
Wolff et al. "Direct gene transfer into mouse muscle in vivo," Science (1990) 247:1465-1468.
Wolff et al. "Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle," Human Mol. Genet. (1992) 1:363-369.
Byrne Barry J.
Kessler Paul D.
Kurtzman Gary J.
Podsakoff Gregory M.
Avigen, Inc.
Crouch Deborah
Johns Hopkins University
LandOfFree
Methods for delivering DNA to muscle cells using recombinant ade does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Methods for delivering DNA to muscle cells using recombinant ade, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Methods for delivering DNA to muscle cells using recombinant ade will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1511776