Methods for delivering DNA to muscle cells using recombinant...

Drug – bio-affecting and body treating compositions – Designated organic active ingredient containing – Carbohydrate doai

Reexamination Certificate

Rate now

  [ 0.00 ] – not rated yet Voters 0   Comments 0

Details

C435S320100, C435S325000, C435S455000, C424S093200, C424S093100

Reexamination Certificate

active

10092454

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), Bujard et al.
patent: 5658565 (1997-08-01), Billiar et al.
patent: 5846528 (1998-12-01), Podsakoff et al.
patent: 5858351 (1999-01-01), Podsakoff et al.
patent: 5962313 (1999-10-01), Podsakoff et al.
patent: 6211163 (2001-04-01), Podsakoff et al.
patent: 6325998 (2001-12-01), Podsakoff et al.
patent: 6335011 (2002-01-01), Podsakoff et al.
patent: 6391858 (2002-05-01), Podsakoff et al.
patent: 6610290 (2003-08-01), Podsakoff et al.
patent: 2002/0192189 (2002-12-01), Xiao et al.
patent: WO 94/13788 (1994-06-01), None
patent: WO 95/13376 (1995-05-01), None
patent: WO 95/20671 (1995-08-01), None
patent: WO 95/34670 (1995-12-01), None
patent: WO 96/40272 (1996-12-01), None
patent: WO 97/12050 (1997-03-01), None
Acsadi et al., “Human Dystrophin Expression in MDX Mice After Intramuscular Injection of DNA Constructs,” Nature 352:815-818 (1995).
Acsadi et al., “Cultured Human Myoblast and Myotubes Show Markedly Different Transducibility by Replication-Defective Adenovirus Recombinant,”Gene Therapy 1:338-340 (1994).
Acsadi et al., “A Differential Efficiency of Adenovirus-Mediated in vivo Gene Transfer Into Skeletal Muscle Cells of Different Maturity,”Hum. Mol. Genetics 3:579-584 (1994).
Barr and Leiden, “Systemic Delivery of Recombinant Proteins by Genetically Modified Myoblast,”Science 254:1507-1509 (1991).
Bartlett et al.,Am. J. Human Genetics 57Supp. 4 #A235.
Blau et al., “Myoblast in Pattern Formation and Gene Therapy,”Trends in Genetics 9(9):269 (1993).
Blau and Springer, “Molecular Medicine Muscle-Mediated Gene Therapy,”New Eng. J. Med. 333:1554-1556 (1995).
Brockstedt et al.,Clin. Immuno. 82(1):67-75 (1999).
Clark et al., “Cell Lines for the Production Recombinant Adeno-Associated Virus,”Human Gene Therapy 6:1329-1341 (1995).
Culver et al.,Trends Genetics 10(5):174-178 (1994).
Dai et al., “Gene Therapy Via Primary Myoblast: Long-Term Expression of Factor IX Protein Following Transplantation in vitro,”Proc. Natl. Acad. Sci. USA 89:10892-10895 (1992).
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 92:1401-1405 (1995).
Davis et al., “Direct Gene Transfer Into Skeletal Muscle in vivo : Factors Affecting Efficiency of Transfer and Stability of Expression,”Hum. Gene Therapy 4:151-159 (1993).
Descamps et al., “Organoids Direct Systemic Expression of Erythropoietin in Mice,”Gene Therapy 2:411-417 (1995).
Dhawan et al., “Systemic Delivery of Human Growth Hormone by Injection of Genetically Engineered Myoblast,”Science 254:1509-1512 (1991).
Einerhand et al.,Gene Therapy 2(5):336-343 (1995).
Flotte et al., “Gene Expression from Adeno-Associated Virus Vectors in Airway Epithelial Cells,”Am. J. Respir. Cell Mol. Biol. 7:349-356 (1992).
Flotte et al., “Expression of the Cystic Fibrosis Transmembrane Conductance Regulator From a Novel Adeno-Associated Virus Promoter,”J. Biol. Chem. 268:3781-3790 (1993).
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 90:10613-10617 (1993).
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. Biool. 11:517-521 (1994).
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 6:1265-1274 (1995).
Greelish et al., “Stable Restoration of the Sarcoglycan Complex in Dystrophic Muscle Perfused with Histamine and a Recombinant Adeno-Associated Viral Vector,”Nature Medicine 4:439-443 (1999).
Hamamori et al., “Myoblast Transfer of Human Erythropoietin Gene in a Mouse Model of Renal Failure,”J. Clin. Inves. 95:1808-1813 (1995).
Hamamori et al., “Persistent Erythropoietin by Myoblast Transfer of Erythropoietin cDNA,”Human Gene Therapy 5:1349-1356 (1994).
Herzog et al.,Proc. Natl. Acad. Sci. 94:5804-5809 (1997).
Hodgson,Exp. Opin. Ther. Pat. 5(5):459-462 (1995).
Kaplitt et al., “Long-Term Gene Expression and Phenotypic Correction Using Adeno-Associated Virus Vectors in the Mammalian Brain.,”Nature Genetics. 8:148-154 (1994).
Kaplitt et al., “Long-Term Gene Transfer in Porcine Myocardium After Coronary Infusion of an Adeno-Associated Virus Vector,”Ann. Thorac Surgery 62:1669-1676).
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).
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. 333(13):823-831 (1995).
Kourtis et al., “Cardiac Gene Therapy With Adeno-Associated Virus Suppression,”Modern Pathology 8(1):33A (1995).
Lafont,Lancet 346:1442-1443 (1995).
Leiden, J.M., “Gene Therapy-Promise, Pitfalls and Prognosis,”New Eng. J. Med. 333:871-872 (1995).
March et al.,Clin. Res. 40(2):358A (1992).
Marshall et al.,Science 269:1050-1055 (1995).
Mendell et al., “ Myoblast Transfer in the Treatment of Duchenne's Muscular Dystrophy,”New Eng. J. Med. 333:832-838 (1995).
Miller et al.,FASE B J 9:190-199 (1995).
Mimuro et al., “Recombinant Adeno-Associated Virus Vector-Transduced Vascular Entodothelial Cells Express the Thrombomodulin Transgene Under the Regulation of Enhanced Plasminogen Activator Inhibitor-1 Promoter,”Abstracts of Scientific Presentation: The Fourth Annual Meeting of the American Society of Gene Therapy Abstract 743(2001).
Naffakh et al., “Sustained Delivery of Erythropoietin in Mice by Genetically Modified Skin Fibroblast,”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).
Nakai et al.,Blood 91(12):4600-4607 (1998).
Orkin et al.,Report and Recommendation from the Panel to Assess the NIH Investment. . . (1995).
Osborne et al., “Gene Therapy for Long-Term Expression of Erythropoietin in Rats,”Natl. Acad. Sci. USA 92:8055-8058 (1995).
Podsakoff et al., “Efficient Gene Transfer into Nondividing Cells by Adeno-Associated Virus-Based Vectors,”J. Virol. 68:5656-5666 (1994).
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).
Quantin et al., “Adenovirus as an Expression Vector in Muscle Cellsin vivo,” Proc. Natl. Acad. Sci. USA 89:2581-2584 (1992).
Raz et al., &

LandOfFree

Say what you really think

Search LandOfFree.com for the USA inventors and patents. Rate them and share your experience with other people.

Rating

Methods for delivering DNA to muscle cells using recombinant... 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..., 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... will most certainly appreciate the feedback.

Rate now

     

Profile ID: LFUS-PAI-O-3784601

  Search
All data on this website is collected from public sources. Our data reflects the most accurate information available at the time of publication.