Persistent expression of FLAG-tagged micro dystrophin in nonhuman primates following intramuscular and vascular delivery

Molecular Therapy

Volumn 18, Issue 1, 2010, Pages 109-117

  Rodino Klapac, Louise R ^a,b   Montgomery, Chrystal L ^a,b   Bremer, William G ^a,b   Shontz, Kimberly M ^a,b   Malik, Vinod ^a,b   Davis, Nancy ^a,b   Sprinkle, Spencer ^a,b   Campbell, Katherine J ^a,b   Sahenk, Zarife ^a,b   Clark, K Reed ^a,b   Walker, Christopher M ^a,b   Mendell, Jerry R ^a,b   Chicoine, Louis G ^a,b  

^a OHIO STATE UNIVERSITY   (United States)

^b NATIONWIDE CHILDREN S HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; EPITOPE; GAMMA INTERFERON; MICRODYSTROPHIN; PARVOVIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CARBOXY TERMINAL SEQUENCE; CONTROLLED STUDY; DIAPHRAGM MUSCLE; DUCHENNE MUSCULAR DYSTROPHY; ENZYME LINKED IMMUNOSORBENT ASSAY; FLUOROSCOPY; GASTROCNEMIUS MUSCLE; GENE CASSETTE; IMMUNE RESPONSE; MOUSE; MUSCLE FATIGUE; NONHUMAN; RHESUS MONKEY; TIBIALIS ANTERIOR MUSCLE; VIRAL GENE THERAPY; VIRUS CAPSID;

ANIMALS; BLOTTING, WESTERN; DEPENDOVIRUS; DYSTROPHIN; ENZYME-LINKED IMMUNOSORBENT ASSAY; GENE THERAPY; GENETIC VECTORS; HUMANS; INJECTIONS, INTRA-ARTERIAL; INJECTIONS, INTRAMUSCULAR; MACACA MULATTA; MICE; MICE, INBRED C57BL; MUSCULAR DYSTROPHY, ANIMAL; PEPTIDES;

ANIMALIA; MACACA; MACACA MULATTA; MIRIDAE; PRIMATES;

EID: 74149085535     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2009.254     Document Type: Article

References (27)

1. Rodino-Klapac, LR, Chicoine, LG, Kaspar, BK and Mendell, JR (2007). Gene therapy for duchenne muscular dystrophy: expectations and challenges. Arch Neurol 64: 1236-1241.
2. Wang, Z, Chamberlain, JS, Tapscott, SJ and Storb, R (2009). Gene therapy in large animal models of muscular dystrophy. ILAR J 50: 187-198.
3. Harper, SQ, Hauser, MA, DelloRusso, C, Duan, D, Crawford, RW, Phelps, SF et al. (2002). Modular fexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med 8: 253-261.
4. Liu, M, Yue, Y, Harper, SQ, Grange, RW, Chamberlain, JS and Duan, D (2005). Adeno-associated virus-mediated microdystrophin expression protects young mdx muscle from contraction-induced injury. Mol Ther 11: 245-256.
5. Rodino-Klapac, LR, Janssen, PM, Montgomery, CL, Coley, BD, Chicoine, LG, Clark, KR et al. (2007). A translational approach for limb vascular delivery of the micro-dystrophin gene without high volume or high pressure for treatment of Duchenne muscular dystrophy. J Transl Med 5: 45.
6. Bostick, B, Yue, Y, Lai, Y, Long, C, Li, D and Duan, D (2008). Adeno-associated virus serotype-9 microdystrophin gene therapy ameliorates electrocardiographic abnormalities in mdx mice. Hum Gene Ther 19: 851-856.
7. Bostick, B, Yue, Y, Long, C, Marschalk, N, Fine, DM, Chen, J et al. (2009). Cardiac expression of a mini-dystrophin that normalizes skeletal muscle force only partially restores heart function in aged Mdx mice. Mol Ther 17: 253-261.
8. Townsend, D, Blankinship, MJ, Allen, JM, Gregorevic, P, Chamberlain, JS and Metzger, JM (2007). Systemic administration of micro-dystrophin restores cardiac geometry and prevents dobutamine-induced cardiac pump failure. Mol Ther 15: 1086-1092.
9. Blankinship, MJ, Gregorevic, P, Allen, JM, Harper, SQ, Harper, H, Halbert, CL et al. (2004). Effcient transduction of skeletal muscle using vectors based on adeno-associated virus serotype 6. Mol Ther 10: 671-678.
10. Mah, C, Fraites, TJ Jr, Cresawn, KO, Zolotukhin, I, Lewis, MA and Byrne, BJ (2004). A new method for recombinant adeno-associated virus vector delivery to murine diaphragm. Mol Ther 9: 458-463.
11. Gregorevic, P, Allen, JM, Minami, E, Blankinship, MJ, Haraguchi, M, Meuse, L et al. (2006). rAAV6-microdystrophin preserves muscle function and extends lifespan in severely dystrophic mice. Nat Med 12: 787-789.
12. Yue, Y, Ghosh, A, Long, C, Bostick, B, Smith, BF, Kornegay, JN et al. (2008). A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol Ther 16: 1944-1952.
13. Wherry, EJ, Barber, DL, Kaech, SM, Blattman, JN and Ahmed, R (2004). Antigen-independent memory CD8 T cells do not develop during chronic viral infection. Proc Natl Acad Sci USA 101: 16004-16009.
14. Hopp, T P, Prickett, KS, Price, VL, Libby, RT, March, CJ, Cerretti, DP et al. (1988). A short polypeptide marker sequence useful for recombinant protein identifcation and purifcation. Biotechnology (N Y) 6: 1204-1210.
15. Louboutin, JP, Reyes, BA, Agrawal, L, Van Bockstaele, E and Strayer, DS (2007). Strategies for CNS-directed gene delivery: in vivo gene transfer to the brain using SV40-derived vectors. Gene Ther 14: 939-949.
16. Terpe, K (2003). Overview of tag protein fusions: from molecular and biochemical fundamentals to commercial systems. Appl Microbiol Biotechnol 60: 523-533.
17. Ishizaki, M, Suga, T, Kimura, E, Shiota, T, Kawano, R, Uchida, Y et al. (2008). Mdx respiratory impairment following fbrosis of the diaphragm. Neuromuscul Disord 18: 342-348.
18. Stedman, HH, Sweeney, HL, Shrager, JB, Maguire, HC, Panettieri, RA, Petrof, B et al. (1991). The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 352: 536-539.
19. Fisher, KJ, Jooss, K, Alston, J, Yang, Y, Haecker, SE, High, K et al. (1997). Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med 3: 306-312.
20. Kessler, PD, Podsakoff, GM, Chen, X, McQuiston, SA, Colosi, PC, Matelis, LA et al. (1996). Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci USA 93: 14082-14087.
21. Corrado, K, Rafael, JA, Mills, PL, Cole, NM, Faulkner, JA, Wang, K et al. (1996). Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a "mild Becker" phenotype. J Cell Biol 134: 873-884.
22. Gao, G P, Alvira, MR, Wang, L, Calcedo, R, Johnston, J and Wilson, JM (2002). Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99: 11854-11859.
23. Li, H, Lin, SW, Giles-Davis, W, Li, Y, Zhou, D, Xiang, ZQ et al. (2009). A preclinical animal model to assess the effect of pre-existing immunity on AAV-mediated gene transfer. Mol Ther 17: 1215-1224.
24. + T cells are unable to eliminate AAV-transduced hepatocytes. Mol Ther 15: 792-800.
25. Mendell, JR, Rodino-Klapac, LR, Rosales-Quintero, X, Kota, J, Coley, BD, Galloway, G et al. (2009). Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins. Ann Neurol 66: 290-297.
26. Rabinowitz, JE, Rolling, F, Li, C, Conrath, H, Xiao, W, Xiao, X et al. (2002). Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specifcity. J Virol 76: 791-801.
27. Clark, KR, Liu, X, McGrath, JP and Johnson, PR (1999). Highly purifed recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther 10: 1031-1039.