Chemistry: molecular biology and microbiology – Vector – per se
Patent
1997-01-16
1999-10-05
Crouch, Deborah
Chemistry: molecular biology and microbiology
Vector, per se
435455, 514 44, C12N 1500, A61K 4800
Patent
active
059623136
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: 5252479 (1993-10-01), Srivastava
patent: 5589362 (1996-12-01), Bujard et al.
patent: 5658565 (1997-08-01), Billiar et al.
Tzail et al (1991) Biochem. Biophys. Res. Comm. 176, 1509-15-15.
Stratford-Pemcaudet et al (1992) J. Clin. Invest. 90, 626-630.
Blau et al., "Myoblast in Pattern Formation and Gene Therapy," Trends In Genetics 9(8):269-274(1993).
Clark et al., "Cell Lines for the Production of Recombinant Adeno-Associated Virus," Human Gene Therapy 6:1329-1341(1995).
Kourtis et al., "Cardiac Gene Therapy with Adeno-Associated Virus as a Means of Achieving Graft-Specific Immunosuppression," Modern Pathology 8(1):33A(178)(1995).
Podsakoff et al., "Long-Term In Vivo Gene Expression in Muscle Using AAV Vectors," J. of The American Society of Hematology 86(10):1004A(4004)(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. Acac. Sci. USA 91:11557-11561(1994).
Kessler et al., "Gene Delivery to Skeletal Muscle Results in Sustained Expression and Systemic Delivery of a Therapeutic Protein," Proc. Natl. Acad. Sci. USA 93:14082-14087 (1996).
Naffakh et al., "Long-Term Secretion of Therapeutic Proteins from Genetically Modified Skeletal Muscles," Human Gene Therapy 7:11-21 (1996).
Podsakoff et al., "AAV Vector-Mediated Gene Delivery to Skeletal Muscle In Vivo Results in Sustained Levels of Systemic Erythropoietin," Blood 88(10):1066(1996).
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 differential 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.
Bartlett et al., Am. J. Human Genetics (1995) 57 (Supp 4) #A235.
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 factor 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.
Einerhand et al., "Regulated high-level human .beta.-globin gene expression in erythroid cells following recombinant adeno-associated virus-mediated gene transfer," Gene Therapy (1995) 2:336-343.
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 transmembrane 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 erythropoietin 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, "Gene therapy-promise, pitfalls and prognosis," New Eng. J. Med. (1995) 333:871-872.
March et al., Clin. Res. (1992) 40(2):358A.
Mendell et al., "Myoblast transfer in the treatment of Duchenne's muscular dystrophy," New Eng. J. Med. (1995) 333:832-838.
Naffakh et al., "Sustained delivery of erythropoietin in mice by genetically modified skin fibroblasts," Proc. Natl. Acad. Sci. (1995) 92:3194-3198.
Osborne et al., "Gene therapy for long-term expression of erythropoietin in rats," Proc. Natl. Acad. Sci. (1995) 92:8055-8058.
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. 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.
Xiao et al., "Adeno-associated virus (AAV) vectors for gene transfer," Advanced Drug Delivery Reviews (1993) 12:201-215.
Xiao et al., "Efficient long-term gene transfer into Muscle tissue of immunocompetent mice by adeno-addociated virus vector," J. of Virol. (1996) 70(11):8098-8108.
Byrne Barry J.
Kessler Paul D.
Kurtzman Gary J.
Podsakoff Gregory M.
Avigen, Inc.
Crouch Deborah
Johns Hopkins University
LandOfFree
Adeno-associated virus vectors comprising a gene encoding a lyos does not yet have a rating. At this time, there are no reviews or comments for this patent.
If you have personal experience with Adeno-associated virus vectors comprising a gene encoding a lyos, we encourage you to share that experience with our LandOfFree.com community. Your opinion is very important and Adeno-associated virus vectors comprising a gene encoding a lyos will most certainly appreciate the feedback.
Profile ID: LFUS-PAI-O-1170702