Myostatin propeptide gene delivery by adeno-associated virus serotype 8 vectors enhances muscle growth and ameliorates dystrophic phenotypes in mdx mice

Human Gene Therapy

Volumn 19, Issue 3, 2008, Pages 241-253

  Qiao, Chunping ^a   Li, Jianbin ^a   Jiang, Jiangang ^a   Zhu, Xiaodong ^c   Wang, Bing ^b   Li, Juan ^a   Xiao, Xiao ^a,d  

^a UNIVERSITY OF NORTH CAROLINA   (United States)

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

^c UNIVERSITY OF PITTSBURGH   (United States)

^d University of North Carolina   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CREATINE KINASE; IMMUNOGLOBULIN FC FRAGMENT; MYOSTATIN; MYOSTATIN PROPEPTIDE; PARVOVIRUS VECTOR; PEPTIDE; UNCLASSIFIED DRUG;

AGED; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL PROLIFERATION; CONTROLLED STUDY; CREATININE BLOOD LEVEL; DUCHENNE MUSCULAR DYSTROPHY; EXERCISE TEST; FEMALE; GENE MUTATION; GENETIC TRANSCRIPTION; GRIP STRENGTH; HEART HYPERTROPHY; IMMUNOFLUORESCENCE TEST; MALE; MONONUCLEAR CELL; MORPHOMETRICS; MOUSE; MUSCLE GROWTH; MUSCLE HYPERTROPHY; MUSCLE STRENGTH; MUSCLE TETANIC CONTRACTION; MYOFIBROSIS; NEWBORN; NONHUMAN; NUCLEOTIDE SEQUENCE; PHENOTYPE; SKELETAL MUSCLE; STATISTICAL SIGNIFICANCE; TREADMILL EXERCISE; VIRAL GENE DELIVERY SYSTEM; X CHROMOSOME LINKED MUSCULAR DYSTROPHIC MOUSE;

ANIMALS; ANIMALS, NEWBORN; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC VECTORS; MICE; MICE, INBRED MDX; MUSCLE DEVELOPMENT; MUSCLE FIBERS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; PEPTIDES; PHENOTYPE; TRANSFORMING GROWTH FACTOR BETA;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MURINAE; MUS; RODENTIA;

EID: 41149152962     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2007.159     Document Type: Article

References (35)

1. BARTOLI, M., POUPIOT, J., VULIN, A., FOUGEROUSSE, F., ARANDEL, L., DANIELE, N., ROUDAUT, C., NOULET, F., GARCIA, L., DANOS, O., and RICHARD, I. (2007). AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not α-sarcoglycan deficiency. Gene Ther. 14, 733-740.
2. BOGDANOVICH, S., KRAG, T.O., BARTON, E.R., MORRIS, L.D., WHITTEMORE, L.A., AHIMA, R.S., and KHURANA, T.S. (2002). Functional improvement of dystrophic muscle by myostatin blockade. Nature 420, 418-421.
3. BOGDANOVICH, S., PERKINS, K.J., KRAG, T.O., WHITTEMORE, L.A., and KHURANA, T.S. (2005). Myostatin propeptide-mediated amelioration of dystrophic pathophysiology. FASEB J. 19, 543-549.
4. CLOP, A., MARCQ, F., TAKEDA, H., PIROTTIN, D., TORDOIR, X., BIBE, B., BOUIX, J., CAIMENT, F., ELSEN, J.M., EYCHENNE, F., LARZUL, C., LAVILLE, E., MEISH, F., MILENKOVIC, D., TOBIN, J., CHARLIER, C., and GEORGES, M. (2006). A mutation creating a potential illegitimate microRNA target site in the myostatin gene affects muscularity in sheep. Nat. Genet. 38, 813-818.
5. FRIBERG, P., ISGAARD, J., WAHLANDER, H., WICKMAN, A., GURON, G., and ADAMS, M.A. (1995). Cardiac hypertrophy and related growth processes: The role of insulin-like growth factor-I. Blood Press. Suppl. 2, 22-29.
6. HILL, J.J., DAVIES, M.V., PEARSON, A.A., WANG, J.H., HEWICK, R.M., WOLFMAN, N.M., and QIU, Y. (2002). The myostatin propeptide and the follistatin-related gene are inhibitory binding proteins of myostatin in normal serum. J. Biol. Chem. 277, 40735-40741.
7. HOFFMAN, E.P., BROWN, R.H., Jr., and KUNKEL, L.M. (1987). Dystrophin: The protein product of the Duchenne muscular dystrophy locus. Cell 51, 919-928.
8. HOFFMAN, E.P., PEGORARO, E., SCACHERI, P., BURNS, R.G., TABER, J.W., WEISS, L., SPIRO, A., and BLATTNER, P. (1996). Genetic counseling of isolated carriers of Duchenne muscular dystrophy. Am. J. Med. Genet. 63, 573-580.
9. HOLMES, J.H., ASHMORE, C.R., and ROBINSON, D.W. (1973). Effects of stress on cattle with hereditary muscular hypertrophy. J. Anim. Sci. 36, 684-694.
10. ITO, K., KIMURA, S., OZASA, S., MATSUKURA, M., IKEZAWA, M., YOSHIOKA, K., UENO, H., SUZUKI, M., ARAKI, K., YAMAMURA, K., MIWA, T., DICKSON, G., THOMAS, G.D., and MIIKE, T. (2006). Smooth muscle-specific dystrophin expression improves aberrant vasoregulation in mdx mice. Hum. Mol. Genet. 15, 2266-2275.
11. KOENIG, M., and KUNKEL, L.M. (1990). Detailed analysis of the repeat domain of dystrophin reveals four potential hinge segments that may confer flexibility. J. Biol. Chem. 265, 4560-4566.
12. KOOTSTRA, N.A., MATSUMURA, R., and VERMA, I.M. (2003). Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol. Ther. 7, 623-631.
13. LEE, S.J. (2004). Regulation of muscle mass by myostatin. Annu. Rev. Cell Dev. Biol. 20, 61-86.
14. LEE, S.J., and MCPHERRON, A.C. (2001). Regulation of myostatin activity and muscle growth. Proc. Natl. Acad. Sci. U.S.A. 98, 9306-9311.
15. LEE, S.J., REED, L.A., DAVIES, M.V., GIRGENRATH, S., GOAD, M.E., TOMKINSON, K.N., WRIGHT, J.F., BARKER, C., EHRMANTRAUT, G., HOLMSTROM, J., TROWELL, B., GERTZ, B., JIANG, M.S., SEBALD, S.M., MATZUK, M., LI, E., LIANG, L.F., QUATTLEBAUM, E., STOTISH, R.L., and WOLFMAN, N.M. (2005). Regulation of muscle growth by multiple ligands signaling through activin type II receptors. Proc. Natl. Acad. Sci. U.S.A. 102, 18117-18122.
16. LOUFRANI, L., DUBROCA, C., YOU, D., LI, Z., LEVY, B., PAULIN, D., and HENRION, D. (2004). Absence of dystrophin in mice reduces NO-dependent vascular function and vascular density: Total recovery after a treatment with the aminoglycoside gentamicin. Arterioscler. Thromb. Vasc. Biol. 24, 671-676.
17. MCMULLEN, J.R., and IZUMO, S. (2006). Role of the insulin-like growth factor 1 (IGF1)/phosphoinositide-3-kinase (PI3K) pathway mediating physiological cardiac hypertrophy. Novartis Found. Symp. 274, 90-111; discussion 111-117, 152-115, 272-116.
18. MCPHERRON, A.C., and LEE, S.J. (1997). Double muscling in cattle due to mutations in the myostatin gene. Proc. Natl. Acad. Sci. U.S.A. 94, 12457-12461.
19. MCPHERRON, A.C., LAWLER, A.M., and LEE, S.J. (1997). Regulation of skeletal muscle mass in mice by a new TGF-β superfamily member. Nature 387, 83-90.
20. MOSHER, D.S., QUIGNON, P., BUSTAMANTE, C.D., SUTTER, N.B., MELLERSH, C.S., PARKER, H.G., and OSTRANDER, E.A. (2007). A mutation in the myostatin gene increases muscle mass and enhances racing performance in heterozygote dogs. PLoS Genet. 3, e79.
21. NAKATANI, M., TAKEHARA, Y., SUGINO, H., MATSUMOTO, M., HASHIMOTO, O., HASEGAWA, Y., MURAKAMI, T., UEZUMI, A., TAKEDA, S., NOJI, S., SUNADA, Y., and TSUCHIDA, K. (2008). Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice. FASEB J. 22, 477-487.
22. NERI SERNERI, G.G., BODDI, M., MODESTI, P.A., CECIONI, I., COPPO, M., PADELETTI, L., MICHELUCCI, A., COLELLA, A., and GALANTI, G. (2001). Increased cardiac sympathetic activity and insulin-like growth factor-I formation are associated with physiological hypertrophy in athletes. Circ. Res. 89, 977-982.
23. NISHI, M., YASUE, A., NISHIMATU, S., NOHNO, T., YAMAOKA, T., ITAKURA, M., MORIYAMA, K., OHUCHI, H., and NOJI, S. (2002). A missense mutant myostatin causes hyperplasia without hypertrophy in the mouse muscle. Biochem. Biophys. Res. Commun. 293, 247-251.
24. OHSAWA, Y., HAGIWARA, H., NAKATANI, M., YASUE, A., MORIYAMA, K., MURAKAMI, T., TSUCHIDA, K., NOJI, S., and SUNADA, Y. (2006). Muscular atrophy of caveolin-3-deficient mice is rescued by myostatin inhibition. J. Clin. Invest. 116, 2924-2934.
25. 2-deficient congenital muscular dystrophy by somatic gene transfer of miniagrin. Proc. Natl. Acad. Sci. U.S.A. 102, 11999-12004.
26. SCHUELKE, M., WAGNER, K.R., STOLZ, L.E., HUBNER, C., RIEBEL, T., KOMEN, W., BRAUN, T., TOBIN, J.F., and LEE, S.J. (2004). Myostatin mutation associated with gross muscle hypertrophy in a child. N. Engl. J. Med. 350, 2682-2688.
27. TOBIN, J.F., and CELESTE, A.J. (2005). Myostatin, a negative regulator of muscle mass: Implications for muscle degenerative diseases. Curr. Opin. Pharmacol. 5, 328-332.
28. WAGNER, K.R., MCPHERRON, A.C., WINIK, N., and LEE, S.J. (2002). Loss of myostatin attenuates severity of muscular dystrophy in mdx mice. Ann. Neurol. 52, 832-836.
29. WANG, B., LI, J., and XIAO, X. (2000). Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc. Natl. Acad. Sci. U.S.A. 97, 13714-13719.
30. WANG, Z., ZHU, T., QIAO, C., ZHOU, L., WANG, B., ZHANG, J., CHEN, C., LI, J., and XIAO, X. (2005). Adeno-associated virus serotype 8 efficiently delivers genes to muscle and heart. Nat. Biotechnol. 23, 321-328.
31. WATCHKO, J., O'DAY, T., WANG, B., ZHOU, L., TANG, Y., LI, J., and XIAO, X. (2002). Adeno-associated virus vector-mediated minidystrophin gene therapy improves dystrophic muscle contractile function in mdx mice. Hum. Gene Ther. 13, 1451-1460.
32. WOLFMAN, N.M., MCPHERRON, A.C., PAPPANO, W.N., DAVIES, M.V., SONG, K., TOMKINSON, K.N., WRIGHT, J.F., ZHAO, L., SEBALD, S.M., GREENSPAN, D.S., and LEE, S.J. (2003). Activation of latent myostatin by the BMP-1/tolloid family of metalloproteinases. Proc. Natl. Acad. Sci. U.S.A. 100, 15842-15846.
33. XIAO, X., LI, J., and SAMULSKI, R.J. (1998). Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J. Virol. 72, 2224-2232.
34. ZHU, T., ZHOU, L., MORI, S., WANG, Z., MCTIERNAN, C.F., QIAO, C., CHEN, C., WANG, D.W., LI, J., and XIAO, X. (2005). Sustained whole-body functional rescue in congestive heart failure and muscular dystrophy hamsters by systemic gene transfer. Circulation 112, 2650-2659.
35. ZHU, X., HADHAZY, M., WEHLING, M., TIDBALL, J.G., and MCNALLY, E.M. (2000). Dominant negative myostatin produces hypertrophy without hyperplasia in muscle. FEBS Lett. 474, 71-75.