Viral vectors for gene transfer to striated muscle

Current Opinion in Molecular Therapeutics

Volumn 6, Issue 5, 2004, Pages 491-498

  Gregorevic, Paul ^a   Blankinship, Michael J ^a   Chamberlain, Jeffrey S ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

Author keywords

Adeno associated virus; Adenovirus; Gene therapy; Muscular dystrophy; Myocardium; Neuromuscular disease; Retrovirus

Indexed keywords

ADENOVIRUS VECTOR; GENE PRODUCT; HERPESVIRUS VECTOR; LENTIVIRUS VECTOR; PARVOVIRUS VECTOR; RETROVIRUS VECTOR; STRUCTURAL PROTEIN; VIRUS VECTOR;

AGING; CARDIOMYOPATHY; DNA VECTOR; GENE TECHNOLOGY; GENE TRANSFER; GENETIC DISORDER; HUMAN; IMMUNE RESPONSE; MUSCLE ATROPHY; MUSCULAR DYSTROPHY; MYOPATHY; NONHUMAN; REVIEW; SKELETAL MUSCLE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

GENE THERAPY; GENETIC VECTORS; HUMANS; MUSCLE, SKELETAL; MUSCULAR DISEASES; VIRUSES;

ADENO-ASSOCIATED VIRUS; ADENOVIRIDAE; UNIDENTIFIED RETROVIRUS;

EID: 6344228682     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (44)

1. Sampaolesi M, Torrente Y, Innocenzi A, Tonlorenzi R, D'Antona G, Pellegrino MA, Barresi R, Bresolin N, De Angelis MG, Campbell KP, Bottinelli R, Cossu G: Cell therapy of α-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science (2003) 301(5632):487-492.
2. Bachrach E, Li S, Perez AL, Schienda J, Liadaki K, Volinski J, Flint A, Chamberlain J, Kunkel LM: Systemic delivery of human microdystrophin to regenerating mouse dystrophic muscle by muscle progenitor cells. Proc Natl Acad Sci USA (2004) 101 10):3581-3586.
3. Furling D, Doucet G, Langlois MA, Timchenke L, Belanger E, Cossette L, Puymirat J: Viral vector producing antisense RNA restores myotonic dystrophy myoblast functions. Gene Ther (2003) 10 9):795-802.
4. Kang Y, Stein CS, Heth JA, Sinn PL, Penisten AK, Staber PD, Ratliff KL, Shen H, Barker CK, Martins I, Sharkey CM et al: In vivo gene transfer using a nonprimate lentiviral vector pseudotyped with Ross River virus glycoproteins. J Virol (2002) 76 18):9378-9388.
5. Bonci D, Cittadini A, Latronico MV, Berello U, Aycock JK, Drusco A, Innocenzi A, Follenzi A, Lavitrano M, Monti MG, Ross J Jr et al: 'Advanced' generation lentiviruses as efficient vectors for cardiomyocyte gene transduction in vitro and in vivo. Gene Ther (2003) 10(8):630-636.
6. Fleury S, Simeoni E, Zuppinger C, Deglon N, von Segesser LK, Kappenberger L, Vassalli G: Multiply attenuated, self-inactivating lentiviral vectore efficiently deliver and express genes for extended periods of time in adult rat cardiomyocytes in vivo Circulation (2003) 107(18):2375-2382.
7. Ouyang J, Alway SE: Transgene expression in hypertrophied and aged skeletal muscle in vivo by lentivirus delivery. J Gene Med (2004) 6(3):278-287.
8. MacKenzie TC, Kobinger GP, Kootstra NA, Radu A, Sena-Esteves M, Bouchard S, Wilson JM, Verma IM, Flake AW: Efficient transduction of liver and muscle after in utero injection of lentiviral vectors with different pseudotypes. Mol Ther (2002) 6(3 ):349-358.
9. Kobinger GP, Louboutin JP, Barton ER, Sweeney HL, Wilson JM: Correction of the dystrophic phenotype by in vivo targeting of muscle progenitor cells. Hum Gene Ther(2003) 14 15):1441-1449.
10. Gregory LG, Waddington SN, Holder MV, Mitrophanous KA, Buckley SM, Mosley KL, Bigger BW, Ellard FM, Walmsley LE, Lawrence L, Al-Allaf F et al: Highly efficient EIAV-mediated in utero gene transfer and expression in the major muscle groups affected by Duchenne muscular dystrophy. Gene Ther (2004) 11 10):1117-1125.
11. O'Rourke JP, Hiraragi H, Urban K, Patel M, Olsen JC, Bunnell BA: Analysis of gene transfer and expression in skeletel muscle using enhanced EIAV lentivirus vectors. Mol Ther (2003) 7 5):632-639.
12. Emery DW, Yannaki E, Tubb J, Nishino T, Li Q, Stamatoyannopoulos G: Development of virus vectors for gene therapy of β chain hemoglobinopathies: Flanking with a chromatin insulator reduces γ-globin gene silencing in vivo. Blood (2002) 100(6):2012-2019.
13. Roberts ML, Wells D J, Graham IR, Fabb SA, Hill V J, Duisit G, Yuasa K, Takeda S, Cosset FL, Dickson G: Stable micro-dystrophin gene transfer using an integrating adeno-retroviral hybrid vector ameliorates the dystrophic pathology in mdx mouse muscle. Hum Mol Genet (2002) 11(15):1719-1730.
14. Kubo S, Mitani K: A new hybrid system capable of efficient lentiviral vector production and stable gene transfer mediated by a single helper-dependent adenoviral vector. J Virol (2003) 77 5):2964-2971.
15. Hartigan-O'Connor D, Barjot C, Salvatori G, Chamberlain JS: Generation and growth of gutted adenoviral vectors. Methods Enzymol (2002) 346:224-246.
16. Fleury S, Driscoll R, Simeoni E, Dudler J, Von Segesser LK, Kappenberger L, Vassalli G: Helper-dependent adenovirus vectors devoid of all viral genes cause less myocardial inflammation compared with first-generation adenovirus vectors. Basic Res Cardiol (2004) 99(4):247-256.
17. DelloRusso C, Scott JM, Hartigan-O'Connor D, Salvatori G, Barjot C, Robinson AS, Crawford RW, Brooks SV, Chamberlain JS: Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Proc Natl Acad Sci USA (2002) 99(20):12979-12984.
18. Dudley RW, Lu Y, Gilbert R, Matecki S, Nalbantoglu J, Petrof BJ, Karpati G: Sustained improvement of muscle function one year after full-length dystrophin gene transfer into mdx mice by a gutted helper-dependent adenoviral vector. Hum Gene Ther (2004) 15 2):145-156.
19. Jiang Z, Schiedner G, Gilchrist SC, Kochanek S, Clemens PR: CTLA4lg delivered by high-capacity adenoviral vector induces stable expression of dystrophin in mdx mouse muscle. Gene Ther (2004) 11(9):1453-1461.
20. Picard-Maureau M, Kreppel F, Lindemann D, Juretzek T, Herchenroder O, Rethwilm A, Kochanek S, Heinkelein M: Foamy virus - adenovirus hybrid vectors. Gene Ther (2004) 11(8 ):722-728.
21. Ikeda Y, Gu Y, Iwanaga Y, Hoshijima M, Oh SS, Giordano F J, Chen J, Nigro V, Peterson KL, Chien KR, Ross J Jr: Restoration of deficient membrane proteins in the cardiomyopathic hamster by in vivo cardiac gene transfer. Circulation (2002) 105 4):502-508.
22. Cerletti M, Negri T, Cozzi F, Colpo R, Andreetta F, Croci D, Davies KE, Cornelio F, Pozza O, Karpati G, Gilbert R, Mora M: Dystrophic phenotype of canine X-linked muscular dystrophy is mitigated by adenovirus-mediated utrophin gene transfer. Gene Ther (2003) 10(9):750-757.
23. Roy S, Gao G, Clawson DS, Vandenberghe LH, Farina SF, Wilson JM: Complete nucleotide sequences and genome organization of four chimpanzee adenoviruses. Virology(2004) 324(2):361-372.
24. Goncalves MA, van der Velde I, Knaan-Shanzer S, Valerio D, de Vries AA: Stable transduction of large DNA by high-capacity adeno-associated virus/adenovirus hybrid vectors. Virology (2004) 321(2):287-296.
25. Bramson JL, Grinshtein N, Meulenbroek RA, Lunde J, Kottachchi D, Lorimer IA, Jasmin B J, Parks RJ: Helper-dependent adenoviral vectors containing modified fiber for improved transduction of developing and mature muscle cells. Hum Gene Ther (2004) 15 2):179-188.
26. Volpers C, Thirion C, Biermann V, Hussmann S, Kewes H, Dunant P, von der Mark H, Herrmann A, Kochanek S, Lochmuller H: Antibody-mediated targeting of an adenovirus vector modified to contain a synthetic immunoglobulin G-binding domain in the capsid. J Virol (2003) 77(3):2093-2104.
27. Bouchard S, MacKenzie TC, Radu AP, Hayashi S, Peranteau WH, Chirmule N, Flake AW: Long-term transgene expression in cardiac and skeletal muscle following fetal administration of adenoviral or adeno-associated viral vectors in mice. J Gene Med (2003) 5 11):941-950.
28. Bujold M, Caron N, Camiran G, Mukherjee S, Allen PD, Tremblay JP, Wang Y: Autotransplantation in mdx mice of mdx myoblasts genetically corrected by an HSV-1 amplicon vector. Cell Transplant (2002) 11(8):759-767.
29. Cao B, Huard J: Gene transfer to skeletal muscle using herpes simplex virus-based vectors. Methods Mol Biol (2004) 246:301-308.
30. Manno CS, Chew A J, Hutchison S, Larson P J, Herzog RW, Arruda VR, Tai S J, Ragni MV, Thompson A, Ozelo M, Couto LB et al: AAV-mediated Factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood (2003) 101(8 ):2963-2972.
31. Harper SQ, Hauser MA, DelloRusso C, Duan D, Crawford RW, Phelps SF, Harper HA, Robinson AS, Engelhardt JF, Brooks SV, Chamberlain JS: Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nat Med (2002) 8 3):253-261.
32. Watchko J, O'Day T, Wang B, Zhou L, Tang Y, Li J, Xiao X: Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. Hum Gene Ther (2002) 13(12):1451-1460.
33. Gregorevic P, Blankinship M J, Allen JM, Crawford RW, Meuse L, Miller DG, Russell DW, Chamberlain JS: Systemic delivery of genes to striated muscles using adeno-associated viral vectors. Nat Med (2004) 10(8):828-834.
34. Iwanaga Y, Hoshijima M, Gu Y, Iwatate M, Dieterle T, Ikeda Y, Date MO, Chrast J, Matsuzaki M, Peterson KL, Chien KR, Ross J Jr: Chronic phospholamban inhibition prevents progressive cardiac dysfunction and pathological remodeling after infarction in rats. J Clin Invest (2004) 113(5):727-736.
35. Fraites TJ Jr, Schleissing MR, Shanely RA, Walter GA, Cloutier DA, Zolotukhin I, Pauly DF, Raben N, Plotz PH, Powers SK, Kessler PD, Byrne BJ: Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors. Mol Ther (2002) 5(5):571-578.
36. Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi Y, Mingozzi F, Xiao W, Couto LB, High KA: Safety and efficacy of Factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood (2004) 103(1):85-92.
37. Blankinship M J, Gregorevic P, Allen JM, Harper SQ, Harper H, Halbert CL, Miller AD, Chamberlain JS: Efficient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther (2004) 10(4):671-678.
38. Rabinowitz JE, Bowles DE, Faust SM, Ledford JG, Cunningham SE, Samulski R J: Cross-dressing the virion: The transcapsidation of adeno-associated virus serotypes functionally defines subgroups. J Virol (2004) 78(9):4421-4432.
39. Warrington KH Jr, Gorbatyuk OS, Harrison JK, Opie SR, Zolotukhin S, Muzyczka N: Adeno-associated virus type 2 VP2 capsid protein is nonessential and can tolerate large peptide insertions at its N terminus. J Virol (2004) 78(12):6595-6609.
40. Gao G, Vandenberghe LH, Alvira MR, Lu Y, Calcedo R, Zhou X, Wilson JM: Clades of adeno-associated viruses are widely disseminated in human tissues. J Virol (2004) 78(12):6381-6388.
41. Yue Y, Li Z, Harper SQ, Davisson RL, Chamberlain JS, Duan D: Microdystrophin gene therapy of cardiomyopathy restores dystrophin-glycoprotein complex and improves sarcolemma integrity in the mdx mouse heart. Circulation (2003) 108 13):1626-1632.
42. Bridges CR, Burkman JM, Malekan R, Konig SM, Chen H, Yarnall CB, Gardner T J, Stewart AS, Stecker MM, Patterson T, Stedman HH: Global cardiac-specific transgene expression using cardiopulmonary bypass with cardiac isolation. Ann Thorac Surg (2002) 73 6):1939-1946.
43. Halbert CL, Allen JM, Miller AD: Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat Biotechnol (2002) 20(7):697-701.
44. Duan D, Yue Y, Engelhardt JF: Dual vector expansion of the recombinant AAV packaging capacity. Methods Mol Biol (2003) 219:29-51.