Drosophila as a starting point for developing therapeutics for the rare disease Duchenne Muscular Dystrophy

References

• Vaquer G, Rivière F, Mavris M, Bignami F, Llinares-Garcia J, Westermark K, et al. Animal models for metabolic, neuromuscular and ophthalmological rare diseases. Nat Rev Drug Discov 2013; 12:287 - 305; http://dx.doi.org/10.1038/nrd3831; PMID: 23493083
   PubMed Web of Science ®Google Scholar
• Shcherbata HR, Yatsenko AS, Patterson L, Sood VD, Nudel U, Yaffe D, et al. Dissecting muscle and neuronal disorders in a Drosophila model of muscular dystrophy. EMBO J 2007; 26:481 - 93; http://dx.doi.org/10.1038/sj.emboj.7601503; PMID: 17215867
   PubMed Web of Science ®Google Scholar
• Christoforou CP, Greer CE, Challoner BR, Charizanos D, Ray RP. The detached locus encodes Drosophila Dystrophin, which acts with other components of the Dystrophin Associated Protein Complex to influence intercellular signalling in developing wing veins. Dev Biol 2008; 313:519 - 32; http://dx.doi.org/10.1016/j.ydbio.2007.09.044; PMID: 18093579
   PubMed Web of Science ®Google Scholar
• Kucherenko MM, Pantoja M, Yatsenko AS, Shcherbata HR, Fischer KA, Maksymiv DV, et al. Genetic modifier screens reveal new components that interact with the Drosophila dystroglycan-dystrophin complex. PLoS One 2008; 3:e2418; http://dx.doi.org/10.1371/journal.pone.0002418; PMID: 18545683
   PubMedGoogle Scholar
• Kucherenko MM, Marrone AK, Rishko VM, Magliarelli H de F, Shcherbata HR. Stress and muscular dystrophy: a genetic screen for dystroglycan and dystrophin interactors in Drosophila identifies cellular stress response components. Dev Biol 2011; 352:228 - 42; http://dx.doi.org/10.1016/j.ydbio.2011.01.013; PMID: 21256839
   PubMed Web of Science ®Google Scholar
• Pantoja M, Fischer KA, Ieronimakis N, Reyes M, Ruohola-Baker H. Genetic elevation of sphingosine 1-phosphate suppresses dystrophic muscle phenotypes in Drosophila. Development 2013; 140:136 - 46; http://dx.doi.org/10.1242/dev.087791; PMID: 23154413
   PubMed Web of Science ®Google Scholar
• Matsumura K, Shimizu T, Nonaka I, Mannen T. Immunochemical study of connectin (titin) in neuromuscular diseases using a monoclonal antibody: connectin is degraded extensively in Duchenne muscular dystrophy. J Neurol Sci 1989; 93:147 - 56; http://dx.doi.org/10.1016/0022-510X(89)90185-8; PMID: 2592979
   PubMed Web of Science ®Google Scholar
• Bruni P, Donati C. Pleiotropic effects of sphingolipids in skeletal muscle. Cell Mol Life Sci 2008; 65:3725 - 36; http://dx.doi.org/10.1007/s00018-008-8236-6; PMID: 18668202
   PubMed Web of Science ®Google Scholar
• Rapizzi E, Donati C, Cencetti F, Nincheri P, Bruni P. Sphingosine 1-phosphate differentially regulates proliferation of C2C12 reserve cells and myoblasts. Mol Cell Biochem 2008; 314:193 - 9; http://dx.doi.org/10.1007/s11010-008-9780-y; PMID: 18454302
   PubMed Web of Science ®Google Scholar
• Ieronimakis N, Pantoja M, Hays A, Dosey TL, Qi J, Fischer KA, et al. Increased Sphingosine-1-Phosphate improves muscle regeneration in acutely injured mdx mice. Skeletal Muscle 2013; In press
   Google Scholar
• Loh KC, Leong W-I, Carlson ME, Oskouian B, Kumar A, Fyrst H, et al. Sphingosine-1-phosphate enhances satellite cell activation in dystrophic muscles through a S1PR2/STAT3 signaling pathway. PLoS One 2012; 7:e37218; http://dx.doi.org/10.1371/journal.pone.0037218; PMID: 22606352
   PubMedGoogle Scholar
• Bagdanoff JT, Donoviel MS, Nouraldeen A, Carlsen M, Jessop TC, Tarver J, et al. Inhibition of sphingosine 1-phosphate lyase for the treatment of rheumatoid arthritis: discovery of (E)-1-(4-((1R,2S,3R)-1,2,3,4-tetrahydroxybutyl)-1H-imidazol-2-yl)ethanone oxime (LX2931) and (1R,2S,3R)-1-(2-(isoxazol-3-yl)-1H-imidazol-4-yl)butane-1,2,3,4-tetraol (LX2932). J Med Chem 2010; 53:8650 - 62; http://dx.doi.org/10.1021/jm101183p; PMID: 21090716
   PubMed Web of Science ®Google Scholar
• Pandey UB, Nichols CD. Human disease models in Drosophila melanogaster and the role of the fly in therapeutic drug discovery. Pharmacol Rev 2011; 63:411 - 36; http://dx.doi.org/10.1124/pr.110.003293; PMID: 21415126
   PubMed Web of Science ®Google Scholar