α2 and α3 helices of dystrophin R16 and R17 frame a microdomain in the α1 helix of dystrophin R17 for neuronal NOS binding

Proceedings of the National Academy of Sciences of the United States of America

Volumn 110, Issue 2, 2013, Pages 525-530

  Lai, Yi ^a   Zhao, Junling ^a   Yue, Yongping ^a   Duan, Dongsheng ^a  

^a University of Missouri   (United States)

Author keywords

Becker muscular dystrophy; BMD; DMD; Gene therapy; Microdystrophin

Indexed keywords

DYSTROPHIN; DYSTROPHIN RELATED PROTEIN 15; DYSTROPHIN RELATED PROTEIN 16; DYSTROPHIN RELATED PROTEIN 17; NEURONAL NITRIC OXIDE SYNTHASE; UNCLASSIFIED DRUG;

ALPHA HELIX; ANIMAL TISSUE; ARTICLE; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MUSCLE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN DOMAIN; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; PROTEIN PROTEIN INTERACTION; SARCOLEMMA; SEQUENCE ALIGNMENT;

ANIMALS; BINDING SITES; BLOTTING, WESTERN; CELL MEMBRANE; DYSTROPHIN; MICE; MICE, INBRED MDX; MICROSCOPY, FLUORESCENCE; MUSCLES; MUSCULAR DYSTROPHY, DUCHENNE; NEURONS; NITRIC OXIDE SYNTHASE TYPE I; PROTEIN BINDING; PROTEIN STRUCTURE, SECONDARY; SPECTRIN; TWO-HYBRID SYSTEM TECHNIQUES; UTROPHIN;

ADENO-ASSOCIATED VIRUS;

EID: 84872175513     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1211431109     Document Type: Article

References (25)

1. Djinovic-Carugo K, Gautel M, Ylänne J, Young P (2002) The spectrin repeat: A structural platform for cytoskeletal protein assemblies. FEBS Lett 513(1):119-123.
2. Le Rumeur E, Hubert JF, Winder SJ (2012) A new twist to coiled coil. FEBS Lett 586(17):2717-2722.
3. Kunkel LM (2005) 2004 William Allan Award address. Cloning of the DMD gene. Am J Hum Genet 76(2):205-214.
4. Lai Y, et al. (2009) Dystrophins carrying spectrin-like repeats 16 and 17 anchor nNOS to the sarcolemma and enhance exercise performance in a mouse model of muscular dystrophy. J Clin Invest 119(3):624-635.
5. Sander M, et al. (2000) Functional muscle ischemia in neuronal nitric oxide synthase-deficient skeletal muscle of children with Duchenne muscular dystrophy. Proc Natl Acad Sci USA 97(25):13818-13823.
6. Thomas GD, et al. (1998) Impaired metabolic modulation of alpha-adrenergic vasoconstriction in dystrophin-deficient skeletal muscle. Proc Natl Acad Sci USA 95(25):15090-15095.
7. Li D, Yue Y, Lai Y, Hakim CH, Duan D (2011) Nitrosative stress elicited by nNOSμ delocalization inhibits muscle force in dystrophin-null mice. J Pathol 223(1):88-98.
8. Hillier BJ, Christopherson KS, Prehoda KE, Bredt DS, Lim WA (1999) Unexpected modes of PDZ domain scaffolding revealed by structure of nNOS-syntrophin complex. Science 284(5415):812-815.
9. Tochio H, Zhang Q, Mandal P, Li M, Zhang M (1999) Solution structure of the extended neuronal nitric oxide synthase PDZ domain complexed with an associated peptide. Nat Struct Biol 6(5):417-421.
10. Lai Y, et al. (2005) Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat Biotechnol 23(11):1435-1439.
11. Yue Y, Liu M, Duan D (2006) C-terminal-truncated microdystrophin recruits dystrobrevin and syntrophin to the dystrophin-associated glycoprotein complex and reduces muscular dystrophy in symptomatic utrophin/dystrophin double-knockout mice. Mol Ther 14(1):79-87.
12. Judge LM, Haraguchiln M, Chamberlain JS (2006) Dissecting the signaling and mechanical functions of the dystrophin-glycoprotein complex. J Cell Sci 119(Pt 8):1537-1546.
13. Li D, et al. (2010) Sarcolemmal nNOS anchoring reveals a qualitative difference between dystrophin and utrophin. J Cell Sci 123(Pt 12):2008-2013.
14. Adams ME, et al. (2000) Absence of alpha-syntrophin leads to structurally aberrant neuromuscular synapses deficient in utrophin. J Cell Biol 150(6):1385-1398.
15. Kameya S, et al. (1999) alpha1-syntrophin gene disruption results in the absence of neuronal-type nitric-oxide synthase at the sarcolemma but does not induce muscle degeneration. J Biol Chem 274(4):2193-2200.
16. Harper SQ, et al. (2002) Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy. Nat Med 8(3):253-261.
17. Hancock JF, Paterson H, Marshall CJ (1990) A polybasic domain or palmitoylation is required in addition to the CAAX motif to localize p21ras to the plasma membrane. Cell 63(1):133-139.
18. Stabach PR, et al. (2009) The structure of the ankyrin-binding site of beta-spectrin reveals how tandem spectrin-repeats generate unique ligand-binding properties. Blood 113(22):5377-5384.
19. Ipsaro JJ, Mondragón A (2010) Structural basis for spectrin recognition by ankyrin. Blood 115(20):4093-4101.
20. Legrand B, Giudice E, Nicolas A, Delalande O, Le Rumeur E (2011) Computational study of the human dystrophin repeats: interaction properties and molecular dynamics. PLoS ONE 6(8):e23819.
21. Le Rumeur E, Winder SJ, Hubert JF (2010) Dystrophin: More than just the sum of its parts. Biochim Biophys Acta 1804(9):1713-1722.
22. Kobayashi YM, et al. (2008) Sarcolemma-localized nNOS is required to maintain activity after mild exercise. Nature 456(7221):511-515.
23. Chao DS, et al. (1996) Selective loss of sarcolemmal nitric oxide synthase in Becker muscular dystrophy. J Exp Med 184(2):609-618.
24. Wells KE, et al. (2003) Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle. Neuromuscul Disord 13(1):21-31.
25. Torelli S, et al. (2004) Absence of neuronal nitric oxide synthase (nNOS) as a pathological marker for the diagnosis of Becker muscular dystrophy with rod domain deletions. Neuropathol Appl Neurobiol 30(5):540-545.