Sustained whole-body functional rescue in congestive heart failure and muscular dystrophy hamsters by systemic gene transfer

Circulation

Volumn 112, Issue 17, 2005, Pages 2650-2659

  Zhu, Tong ^a   Zhou, Liqiao ^a   Mori, Satsuki ^a   Wang, Zhong ^a   McTiernan, Charles F ^a   Qiao, Chunping ^a   Chen, Chunlian ^a,b   Wang, Dao Wen ^b   Li, Juan ^a   Xiao, Xiao ^a  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

^b HUAZHONG UNIVERSITY OF SCIENCE AND TECHNOLOGY   (China)

Author keywords

Cardiomyopathy; Gene therapy; Viruses

Indexed keywords

CREATINE KINASE; DELTA SARCOGLYCAN; PARVOVIRUS VECTOR; SARCOGLYCAN; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONGESTIVE HEART FAILURE; CONTROLLED STUDY; CREATINE KINASE BLOOD LEVEL; DELTA SARCOGLYCAN GENE; ECHOCARDIOGRAPHY; FIBROSIS; GENE; GENE EXPRESSION; GENE MUTATION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; HAMSTER; HEART FUNCTION; HEART LEFT VENTRICLE ENDDIASTOLIC PRESSURE; HEART MUSCLE; MUSCLE FIBER MEMBRANE; MUSCULAR DYSTROPHY; NEWBORN; NONHUMAN; PRIORITY JOURNAL; SKELETAL MUSCLE; SURVIVAL TIME; TREADMILL EXERCISE;

ANIMALS; CREATINE KINASE; CRICETINAE; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE DELETION; GENE THERAPY; GENE TRANSFER TECHNIQUES; HEART FAILURE, CONGESTIVE; MESOCRICETUS; MUSCULAR DYSTROPHY, ANIMAL; SARCOGLYCANS;

EID: 27444434461     PISSN: 00097322     EISSN: None     Source Type: Journal    
DOI: 10.1161/CIRCULATIONAHA.105.565598     Document Type: Article

References (46)

1. Gussoni E, Soneoka Y, Strickland CD, Buzney EA, Khan MK, Flint AF, Kunkel LM, Mulligan RC. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature. 1999;401:390-394.
2. 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;8:253-261.
3. Wang Z, Zhu T, Qiao C, Zhou L, Wang B, Zhang J, Chen C, Li J, Xiao X. Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat Biotechnol. 2005;23:321-328.
4. 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 alpha-sarcoglycan null dystrophic mice through intra-arterial delivery of mesoangioblasts. Science. 2003;301:487-492.
5. 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 U S A. 2004;101:3581-3586.
6. Greelish JP, Su LT, Lankford EB, Burkman JM, Chen H, Konig SK, Mercier IM, Desjardins PR, Mitchell MA, Zheng XG, Leferovich J, Gao GP, Balice-Gordon RJ, Wilson JM, Stedman HH. Stable restoration of the sarcoglycan complex in dystrophic muscle perfused with histamine and a recombinant adeno-associated viral vector. Nat Med. 1999;5:439-443.
7. Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol. 1996;70:8098-8108.
8. Fisher KJ, Jooss K, Alston J, Yang Y, Haecker SE, High K, Pathak R, Raper SE, Wilson JM. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med. 1997;3:306-312.
9. Li J, Wang D, Qian S, Chen Z, Zhu T, Xiao X. Efficient and long-term intracardiac gene transfer in delta-sarcoglycan-deficiency hamster by adeno-associated virus-2 vectors. Gene Ther. 2003;10:1807-1813.
10. Hoshijima M, Ikeda Y, Iwanaga Y, Minamisawa S, Date MO, Gu Y, Iwatate M, Li M, Wang L, Wilson JM, Wang Y, Ross J, Chien KR. Chronic suppression of heart-failure progression by a pseudophosphorylated mutant of phospholamban via in vivo cardiac rAAV gene delivery. Nat Med. 2002;8:864-871.
11. Chao H, Liu Y, Rabinowitz J, Li C, Samulski RJ, Walsh CE. Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors. Mol Ther. 2000;2:619-623.
12. Goyenvalle A, Vulin A, Fougerousse F, Leturcq F, Kaplan JC, Garcia L, Danos O. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science. 2004;306:1796-1799.
13. Wang B, Li J, Xiao X. Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc Natl Acad Sci U S A. 2000;97:13714-13719.
14. Xiao X, Li J, Tsao YP, Dressman D, Hoffman EP, Watchko JF. Full functional rescue of a complete muscle (TA) in dystrophic hamsters by adeno-associated virus vector-directed gene therapy. J Virol. 2000;74:1436-1442.
15. Cordier L, Hack AA, Scott MO, Barton-Davis ER, Gao G, Wilson JM, McNally EM, Sweeney HL. Rescue of skeletal muscles of gamma-sarcoglycan-deficient mice with adeno-associated virus-mediated gene transfer. Mol Ther. 2000;1:119-129.
16. 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;13:1451-1460.
17. Budker V, Zhang G, Danko I, Williams P, Wolff J. The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther. 1998;5:272-276.
18. Hagstrom JE, Hegge J, Zhang G, Noble M, Budker V, Lewis DL, Herweijer H, Wolff JA. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol Ther. 2004;10:386-398.
19. Gregorevic P, Blankinship MJ, 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;10:828-834.
20. Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA. Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model. Blood. 2005;109:3458-3464.
21. Lee CG, Link H, Baluk P, Homer RJ, Chapoval S, Bhandari V, Kang MJ, Cohn L, Kim YK, McDonald DM, Elias JA. Vascular endothelial growth factor (VEGF) induces remodeling and enhances T(H)2-mediated sensitization and inflammation in the lung. Nat Med. 2004;10:1095-1103.
22. Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A. 2002;99:11854-11859.
23. Xiao W, Chirmule N, Berta SC, McCullough B, Gao G, Wilson JM. Gene therapy vectors based on adeno-associated virus type 1. J Virol. 1999;73:3994-4003.
24. 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;78:6381-6388.
25. Rutledge EA, Halbert CL, Russell DW. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol. 1998;72:309-319.
26. Chiorini JA, Kim F, Yang L, Kotin RM. Cloning and characterization of adeno-associated virus type 5. J Virol. 1999;73:1309-1319.
27. Chiorini JA, Yang L, Liu Y, Safer B, Kotin RM. Cloning of adeno-associated virus type 4 (AAV4) and generation of recombinant AAV4 particles. J Virol. 1997;71:6823-6833.
28. Homburger F, Nixon CW, Eppenberger M, Baker JR. Hereditary myopathy in the Syrian hamster: studies on pathogenesis. Ann N Y Acad Sci. 1966;138:14-27.
29. Holt KH, Lim LE, Straub V, Venzke DP, Duclos F, Anderson RD, Davidson BL, Campbell KP. Functional rescue of the sarcoglycan complex in the BIO 14.6 hamster using delta-sarcoglycan gene transfer. Mol Cell. 1998;1:841-848.
30. Li J, Dressman D, Tsao YP, Sakamoto A, Hoffman EP, Xiao X. rAAV vector-mediated sarcogylcan gene transfer in a hamster model for limb girdle muscular dystrophy. Gene Ther. 1999;6:74-82.
31. Ikeda Y, Gu Y, Iwanaga Y, Hoshijima M, Oh SS, Giordano FJ, 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;105:502-508.
32. 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;108:1626-1632.
33. Nigro V, Okazaki Y, Belsito A, Piluso G, Matsuda Y, Politano L, Nigro G, Ventura C, Abbondanza C, Molinari AM, Acampora D, Nishimura M, Hayashizaki Y, Puca GA. Identification of the Syrian hamster cardiomyopathy gene. Hum Mol Genet. 1997;6:601-607.
34. Sakamoto A, Ono K, Abe M, Jasmin G, Eki T, Murakami Y, Masaki T, Toyo-oka T, Hanaoka F. Both hypertrophic and dilated cardiomyopathies are caused by mutation of the same gene, delta-sarcoglycan, in hamster: an animal model of disrupted dystrophin-associated glycoprotein complex. Proc Natl Acad Sci U S A. 1997;94:13873-13878.
35. Hoffman EP. Counting muscular dystrophies in the post-molecular census. J Neurol Sci. 1999;164:3-6.
36. Lim LE, Campbell KP. The sarcoglycan complex in limb-girdle muscular dystrophy. Curr Opin Neurol. 1998;11:443-452.
37. Li X, Eastman EM, Schwartz RJ, Draghia-Akli R. Synthetic muscle promoters: activities exceeding naturally occurring regulatory sequences. Nat Biotechnol. 1999;17:241-245.
38. Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol. 1998;72:2224-2232.
39. Kadokami T, McTiernan CF, Kubota T, Frye CS, Bounoutas GS, Robbins PD, Watkins SC, Feldman AM. Effects of soluble TNF receptor treatment on lipopolysaccharide-induced myocardial cytokine expression. Am J Physiol Heart Circ Physiol. 2001;280:H2281-H2291.
40. Nakai H, Fuess S, Storm TA, Muramatsu S, Nara Y, Kay MA. Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice. J Virol. 2005;79:214-224.
41. Kay MA, High K. Gene therapy for the hemophilias. Proc Natl Acad Sci U S A. 1999;96:9973-9975.
42. Xiao W, Chirmule N, Schnell MA, Tazelaar J, Hughes JV, Wilson JM. Route of administration determines induction of T-cell-independent humoral responses to adeno-associated virus vectors. Mol Ther. 2000;1:323-329.
43. Dressman D, Araishi K, Imamura M, Sasaoka T, Liu LA, Engvall E, Hoffman EP. Delivery of alpha- and beta-sarcoglycan by recombinant adeno-associated virus: efficient rescue of muscle, but differential toxicity. Hum Gene Ther. 2002;13:1631-1646.
44. Dunant P, Larochelle N, Thirion C, Stucka R, Ursu D, Petrof BJ, Wolf E, Lochmuller H. Expression of dystrophin driven by the 1.35-kb MCK promoter ameliorates muscular dystrophy in fast, but not in slow muscles of transgenic mdx mice. Mol Ther. 2003;8:80-89.
45. Larochelle N, Lochmuller H, Zhao J, Jani A, Hallauer P, Hastings KE, Massie B, Prescott S, Petrof BJ, Karpati G, Nalbantoglu J. Efficient muscle-specific transgene expression after adenovirus- mediated gene transfer in mice using a 1.35 kb muscle creatine kinase promoter/enhancer. Gene Ther. 1997;4:465-472.
46. Jaynes JB, Johnson JE, Buskin JN, Gartside CL, Hauschka SD. The muscle creatine kinase gene is regulated by multiple upstream elements, including a muscle-specific enhancer. Mol Cell Biol. 1988;8:62-70.