Atelocollagen-mediated local and systemic applications of myostatin-targeting siRNA increase skeletal muscle mass

Gene Therapy

Volumn 15, Issue 15, 2008, Pages 1126-1130

  Kinouchi, N ^a   Ohsawa, Y ^b   Ishimaru, N ^a   Ohuchi, H ^a   Sunada, Y ^b   Hayashi, Y ^a   Tanimoto, Y ^a   Moriyama, K ^a   Noji, S ^a  

^a UNIVERSITY OF TOKUSHIMA   (Japan)

^b KAWASAKI MEDICAL SCHOOL   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

ATELOCOLLAGEN; MYOSTATIN; NANOPARTICLE; SMALL INTERFERING RNA;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; GENE SILENCING; GENE TARGETING; IN VIVO STUDY; MASSETER MUSCLE; MOUSE; MUSCLE ATROPHY; MUSCLE GROWTH; MUSCLE MASS; MUSCULAR DYSTROPHY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; QUADRICEPS FEMORIS MUSCLE; RNA INTERFERENCE; SKELETAL MUSCLE;

ANIMALS; COLLAGEN; GENE THERAPY; IMMUNOHISTOCHEMISTRY; INJECTIONS, INTRAMUSCULAR; INJECTIONS, INTRAVENOUS; MALE; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHY, ANIMAL; NANOPARTICLES; RNA INTERFERENCE; RNA, SMALL INTERFERING; TRANSFORMING GROWTH FACTOR BETA;

MUS;

EID: 47549101861     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2008.24     Document Type: Article

References (21)

1. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391: 806-811.
2. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411: 494-498.
3. de Fougerolles A, Vornlocher HP, Maraganore J, Lieberman J. Interfering with disease: A progress report on siRNA-based therapeutics. Nat Rev Drug Discov 2007; 6: 443-453.
4. Gary DJ, Puri N, Won YY. Polymer-based siRNA delivery: Perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery. J Control Release 2007; 121: 64-73.
5. Minakuchi Y, Takeshita F, Kosaka N, Sasaki H, Yamamoto Y, Kouno M et al. Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo. Nucleic Acids Res 2004; 32: E109.
6. Takeshita F, Minakuchi Y, Nagahara S, Honma K, Sasaki H, Hirai K et al. Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc Natl Acad Sci USA 2005; 102: 12177-12182.
7. Takeshita F, Ochiya T. Therapeutic potential of RNA interference against cancer. Cancer Sci 2006; 97: 689-696.
8. 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.
9. Deconinck N, Dan B. Pathophysiology of Duchenne muscular dystrophy: current hypotheses. Pediatr Neurol 2007; 36: 1-7.
10. Foster K, Foster H, Dickson JG. Gene therapy progress and prospects: Duchenne muscular dystrophy. Gene Therapy 2006; 13: 1677-1685.
11. Bogdanovich S, Krag TO, Barton ER, Morris LD, Whittemore LA, Ahima RS et al. Functional improvement of dystrophic muscle by myostatin blockade. Nature 2002; 420: 418-421.
12. Nishi M, Yasue A, Nishimatsu S, Nohno T, Yamaoka T, Itakura M et al A missense mutant myostatin causes hyperplasia without hypertrophy in the mouse muscle. Biochem Biophys Res Commun 2002; 293: 247-251.
13. Ohsawa Y, Hagiwara H, Nakatani M, Yasue A, Moriyama K, Murakami T et al. Muscular atrophy of caveolin-3-deficient mice is rescued by myostatin inhibition. J Clin Invest 2006; 116: 2924-2934.
14. Magee TR, Artaza JN, Ferrini MG, Vernet D, Zuniga FI, Cantini L et al Myostatin short interfering hairpin RNA gene transfer increases skeletal muscle mass. J Gene Med 2006; 8: 1171-1181.
15. Artaza JN, Bhasin S, Magee TR, Reisz-Porszasz S, Shen R, Groome NP et al. Myostatin inhibits myogenesis and promotes adipogenesis in C3H 10T(1/2) mesenchymal multipotent cells. Endocrinology 2005; 146 3547-3557.
16. Bulfield G, Siller WG, Wight PA, Moore KJ. X chromosome-linked muscular dystrophy (mdx) in the mouse. Proc Natl Acad Sci USA 1984; 81 1189-1192.
17. McPherron AC, Lee SJ. Suppression of body fat accumulation in myostatin-deficient mice. J Clin Invest 2002; 109: 595-601.
18. Takei Y, Kadomatsu K, Yuzawa Y, Matsuo S, Muramatsu T. A small interfering RNA targeting vascular endothelial growth factor as cancer therapeutics. Cancer Res 2004; 64: 3365-3370.
19. Honma K, Ochiya T, Nagahara. S, Sano A, Yamamoto H, Hirai K et al. Atelocollagen-based gene transfer in cells allows high-throughput screening of gene functions. Biochem Biophys Res Commun 2001; 289: 1075-1081.
20. Ochiya T, Nagahara S, Sano A, Itoh H, Terada. M. Biomaterials for gene delivery. atelocollagen-mediated controlled release of molecular medicines. Curr Gene Ther 2001; 1: 31-52.
21. Sano A, Maeda M, Nagahara S, Ochiya T, Honma K, Itoh H et al. Atelocollagen for protein and gene delivery. Adv Drug Deliv Rev 2003; 55: 1651-1677.