Myostatin short interfering hairpin RNA gene transfer increases skeletal muscle mass

Journal of Gene Medicine

Volumn 8, Issue 9, 2006, Pages 1171-1181

  Magee, Thomas R ^a,b,c,d   Artaza, Jorge N ^c   Ferrini, Monica G ^a,b,c   Vernet, Dolores ^b   Zuniga, Freddi I ^b   Cantini, Liliana ^b   Reisz Porszasz, Suzanne ^c   Rajfer, Jacob ^a,b   Gonzalez Cadavid, Nestor F ^a,b,c  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b HARBOR UCLA MEDICAL CENTER   (United States)

^c CHARLES R DREW UNIVERSITY OF MEDICINE AND SCIENCE   (United States)

^d LABioMed   (United States)

Author keywords

Electroporation; Fibrosis; Gene theraphy; Muscle atrophy; Muscle dystrophy; Sarcopenia

Indexed keywords

BETA GALACTOSIDASE; MESSENGER RNA; MYOSIN HEAVY CHAIN; MYOSTATIN; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; TRANSCRIPTION FACTOR PAX7;

ANIMAL TISSUE; ARTICLE; CELL COUNT; CELL PROLIFERATION; CELL SIZE; CONTROLLED STUDY; ELECTROPORATION; EXPRESSION VECTOR; GENE EXPRESSION; GENE SILENCING; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; MALE; MOLECULAR CLONING; MUSCLE ATROPHY; MUSCLE CELL; MUSCLE MASS; NONHUMAN; NUCLEOTIDE SEQUENCE; PLASMID; PRIORITY JOURNAL; PROTEIN ANALYSIS; PROTEIN DETERMINATION; PROTEIN EXPRESSION; PROTEIN TARGETING; RAT; REAL TIME POLYMERASE CHAIN REACTION; SKELETAL MUSCLE; SKELETAL MUSCLE SATELLITE CELL; TIBIALIS ANTERIOR MUSCLE; TREATMENT INDICATION; WESTERN BLOTTING;

ANIMALS; BASE SEQUENCE; CELL LINE; ELECTROPORATION; GENE EXPRESSION; GENE SILENCING; GENE TRANSFER TECHNIQUES; GENES, REPORTER; GENETIC VECTORS; HUMANS; LAC OPERON; MALE; MICE; MUSCLE, SKELETAL; PLASMIDS; RATS; RATS, INBRED F344; RNA, SMALL INTERFERING; TRANSFECTION; TRANSFORMING GROWTH FACTOR BETA;

EID: 33749249252     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.946     Document Type: Article

References (51)

1. Patel K, Amthor H. The function of myostatin and strategies of myostatin blockade-new hope for therapies aimed at promoting growth of skeletal muscle. Neuromuscul Disord 2005; 15: 117-126.
2. Gonzalez-Cadavid NF, Bhasin S. Role of myostatin in metabolism. Curr Opin Clin Nutr Metab Care 2004; 7: 451-457.
3. Lee SJ. Regulation of muscle mass by myostatin. Annu Rev Cell Dev Biol 2004; 20: 61-86.
4. McPherron AC, Lawler AM, Lee SJ. Regulation of skeletal muscle mass in mice by a new TGF-beta superfamily member. Nature 1997; 387: 83-90.
5. Grobet L, Pirottin D, Farnir F, et al. Modulating skeletal muscle mass by postnatal, muscle-specific inactivation of the myostatin gene. Genesis 2003; 35: 227-238.
6. Thomas M, Langley B, Berry C, et al. Myostatin, a negative regulator of muscle growth, functions by inhibiting myoblast proliferation. J Biol Chem 2000; 275: 40235-40243.
7. Taylor WE, Bhasin S, Artaza J, et al. Myostatin inhibits cell proliferation and protein synthesis in C2C12 muscle cells. Am J Physiol Endocrinol Metab 2001; 280: E221-E228.
8. McPherron AC, Lee SJ. Double muscling in cattle due to mutations in the myostatin gene. Proc Natl Acad Sci U S A 1997; 94: 12457-12461.
9. Schuelke M, Wagner KR, Stolz LE, et al. Myostatin mutation associated with gross muscle hypertrophy in a child. N Engl J Med 2004; 350: 2682-2688.
10. Reisz-Porszasz S, Bhasin S, Artaza JN, et al. Lower skeletal muscle mass in male transgenic mice with muscle-specific overexpression of myostatin. Am J Physiol Endocrinol Metab 2003; 285: E876-E888.
11. Wolfman NM, McPherron AC, Pappano WN, et al. Activation of latent myostatin by the BMP-1/tolloid family of metalloproteinases. Proc Natl Acad Sci USA 2003; 100: 15842-15846.
12. Whittemore LA, Song K, Li X, et al. Inhibition of myostatin in adult mice increases skeletal muscle mass and strength. Biochem Biophys Res Commun 2003; 300: 965-971.
13. Wiener P, Smith JA, Lewis AM, et al. Muscle-related traits in cattle: The role of the myostatin gene in the South Devon breed. Genet Sel Evol 2002; 34: 221-232.
14. Hanset R. The major gene of muscular hypertrophy in the Belgian Blue cattle breed. In Breeding for Disease Resistance in Farm Animals, Owen JB, Axford RFE (eds). CAB International: Bangor, UK, 1991; 467-478.
15. Chupin D. Analysis of reproduction problems in double muscle females. In Muscle Hypertrophy of Genetic Origin and Its Use to Improve Beef Production, King JWB, Menissier F (eds). Kluwer: Dordrecht, 1982.
16. Bogdanovich S, Krag TO, Barton ER, et al. Functional improvement of dystrophic muscle by myostatin blockade. Nature 2002; 420: 418-421.
17. McCroskery S, Thomas M, Platt L, et al. Improved muscle healing through enhanced regeneration and reduced fibrosis in myostatin-null mice. J Cell Sci 2005; 118: 3531-3541.
18. Wagner KR, McPherron AC, Winik N, et al. Loss of myostatin attenuates severity of muscular dystrophy in mdx mice. Ann Neurol 2002; 52: 832-836.
19. Liu M, Yue Y, Harper SQ, et al. Adeno-associated virus-mediated microdystropbin expression protects young mdx muscle from contraction-induced injury. Mol Ther 2005; 11: 245-256.
20. Uchida Y, Maeda Y, Kimura E, et al. Effective repetitive dystrophin gene transfer into skeletal muscle of adult mdx mice using a helper-dependent adenovirus vector expressing the coxsackievirus and adenovirus receptor (CAR) and dystrophin. J Gene Med 2005; 7: 1010-1022.
21. Wells DJ. Gene therapy progress and prospects: electroporation and other physical methods. Gene Ther 2004; 11: 1363-1369.
22. Somia N, Verma IM. Gene therapy: trials and tribulations. Nat Rev Genet 2000; 1: 91-99.
23. Gronevik E, von Steyern FV, Kalhovde JM, et al. Gene expression and immune response kinetics using electroporation-mediated DNA delivery to muscle. J Gene Med 2005; 7: 218-227.
24. Satkauskas S, Bureau MF, Mahfoudi A, et al. Slow accumulation of plasmid in muscle cells: supporting evidence for a mechanism of DNA uptake by receptor-mediated endocytosis. Mol Ther 2001; 4: 317-323.
25. Mir LM, Bureau MF, Gehl J, et al. High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc Natl Acad Sci U S A 1999; 96: 4262-4267.
26. McMahon JM, Wells DJ. Electroporation for gene transfer to skeletal muscles: current status. BioDrugs 2004; 18: 155-165.
27. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411: 494-498.
28. Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002; 296: 550-553.
29. Yin C, Xi L, Wang X, et al. Silencing heat shock factor 1 by small interfering RNA abrogates heat shock-induced cardioprotection against ischemia-reperfusion injury in mice. J Mol Cell Cardiol 2005; 39: 681-689.
30. Golzio M, Mazzolini L, Moller P, et al. Inhibition of gene expression in mice muscle by in vivo electrically mediated siRNA delivery. Gene Ther 2005; 12: 246-251.
31. Stegmeier F, Hu G, Rickles RJ, et al. A lentiviral microRNA-based system for single-copy polymerase II-regulated RNA interference in mammalian cells. Proc Natl Acad Sci U S A 2005; 102: 13212-13217.
32. Sioud M. Therapeutic siRNAs. Trends Pharmacol Sci 2004; 25: 22-28.
33. Seibler J, Kuter-Luks B, Kem H, et al. Single copy shRNA configuration for ubiquitous gene knockdown in mice. Nucleic Acids Res 2005; 33: e67.
34. Faurie C, Golzio M, Moller P, et al. Cell and animal imaging of electrically mediated gene transfer. DNA Cell Biol 2003; 22: 777-783.
35. Reddy GK, Enwemeka CS. A simplified method for the analysis of hydroxyproline in biological tissues. Clin Biochem 1996; 29: 225-229.
36. Ferrini MG, Vernet D, Magee TR, et al. Antifibrotic role of inducible nitric oxide synthase. Nitric Oxide 2002; 6: 283-294.
37. Magee TR, Ferrini M, Garban HJ, et al. Gene therapy of erectile dysfunction in the rat with penile neuronal nitric oxide synthase. Biol Reprod 2002; 67: 1033-1041.
38. T method. Methods 2001; 25: 402-408.
39. Artaza JN, Bhasin S, Magee TR, et al. Myostatin inhibits myogenesis and promotes adipogenesis in C3H 10T(1/2) mesenchymal multipotent cells. Endocrinology 2005; 146: 3547-3557.
40. Staron RS, Kraemer WJ, Hikida RS, et al. Fiber type composition of four hindlimb muscles of adult Fisher 344 rats. Histochem Cell Biol 1999; 111: 117-123.
41. Bogdanovich S, Perkins KJ, Krag TO, et al. Myostatin propeptide-mediated amelioration of dystrophic pathophysiology. FASEB J 2005; 19: 543-549.
42. Tobin JF, Celeste AJ. Myostatin, a negative regulator of muscle mass: implications for muscle degenerative diseases. Curr Opin Pharmacol 2005; 5: 328-332.
43. Acosta J, Carpio Y, Borroto I, Gonzalez O, Estrada MP. Myostatin gene silenced by RNAi show a zebrafish giant phenotype. J Biotechnol 2005; 119: 324-331.
44. Nishi M, Yasue A, Nishimatu S, et al. A missense mutant myostatin causes hyperplasia without hypertrophy in the mouse muscle. Biochem Biophys Res Commun 2002; 293: 247-251.
45. Satkauskas S, Bureau MF, Puc M, et al. Mechanisms of in vivo DNA electrotransfer: respective contributions of cell electropermeabilization and DNA electrophoresis. Mol Ther 2002; 5: 133-140.
46. Cappelletti M, Zampaglione I, Rizzuto G, et al. Gene electro-transfer improves transduction by modifying the fate of intramuscular DNA. J Gene Med 2003; 5: 324-332.
47. Yarasheski KE, Bhasin S, Sinha-Hikim I, et al. Serum myostatin-immunoreactive protein is increased in 60-92 year old women and men with muscle wasting. J Nutr Health Aging 2002; 6: 343-348.
48. Taylor WE, Bhasin S, Lalani R, et al. Alteration of gene expression profiles in skeletal muscle of rats exposed to microgravity during a spaceflight. J Gravit Physiol 2002; 9: 61-70.
49. Jackman RW, Kandarian SC. The molecular basis of skeletal muscle atrophy. Am J Physiol Cell Physiol 2004; 287: C834-C843.
50. Gonzalez-Cadavid NF, Taylor WE, Yarasheski K, et al. Organization of the human myostatin gene and expression in healthy men and HIV-infected men with muscle wasting. Proc Natl Acad Sci U S A 1998; 95: 14938-14943.
51. Frimel TN, Kapadia F, Gaidosh GS, et al. A model of muscle atrophy using cast immobilization in mice. Muscle Nerve 2005; 32: 672-674.