Exon-specific dystrophin antibodies for studies of duchenne muscular dystrophy

Translational Neuroscience

Volumn 1, Issue 3, 2010, Pages 233-237

  Lam, Le Thanh ^a   Nguyen, Giang H ^a,d   Man, Nguyen Thi ^a   Sewry, Caroline A ^a,b   Morris, Glenn E ^a,c  

^a ROBERT JONES AND AGNES HUNT ORTHOPAEDIC HOSPITAL   (United Kingdom)

^b UNIVERSITY COLLEGE LONDON   (United Kingdom)

^c KEELE UNIVERSITY   (United Kingdom)

^d NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH SCIENCES   (United States)

Author keywords

Cell therapy; Dystrophin; Epitope mapping; Gene therapy; Monoclonal antibody; Muscular dystrophy

Indexed keywords

DYSTROPHIN; MONOCLONAL ANTIBODY; TISSUE EXTRACT;

ANIMAL CELL; ANTIBODY DETECTION; ANTIBODY PRODUCTION; ANTIBODY SPECIFICITY; ARTICLE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; EPITOPE MAPPING; EXON; HUMAN; HUMAN TISSUE; IMMUNOHISTOCHEMISTRY; MOLECULAR CLONING; MOUSE; NONHUMAN; PRIORITY JOURNAL; TISSUE SECTION; WESTERN BLOTTING;

EID: 79952348849     PISSN: 20813856     EISSN: 20816936     Source Type: Journal    
DOI: 10.2478/v10134-010-0034-7     Document Type: Article

References (21)

1. Le TT, Nguyen thi Man, Hori S, Sewry CA, Dubowitz V, Morris GE Characterization of genetic deletions in Becker Muscular Dystrophy using monoclonal antibodies against a deletion-prone region of dystrophin.Amer. J. Med. Genet. 58, 1995, 177-86
2. Lederfein D, Levy N, Augier N, Leger J, Morris GE, Fuchs O, Yaffe D, Nudel U 71kD protein is a major product of the Duchenne Muscular Dystrophy gene in brain and other non-muscle tissues.Proc Natl Acad Sci.USA, 89, 1992, 5346-50.
3. Hugnot JP, Gilgenkrantz H, Vincent N, Chafey P, Morris GE, Monaco T, Berwald-Netter Y, Koulakoff A, Kaplan JC, Kahn A, Chelly J. Novel products of the dystrophin gene: A distal transcript initiated from a unique alternative first exon encoding a 75 kDa protein widely distributed in nonmuscle tissues.Proc Natl Acad Sci.USA, 89, 1992, 7506-10.
4. Blake DJ, Love DR, Tinsley J, Morris GE, Turley H, Gatter K, Dickson G, Morgan J, Edwards YH, Davies KE Characterization of a 4.8kb transcript from the Duchenne muscular dystrophy locus expressed in Schwannoma cells.Hum. Mol. Genet. 1, 1992, 103-9.
5. Morris GE, Simmons C, Nguyen thi Man, Apo-dystrophins (Dp140 and Dp71) and dystrophin splicing isoforms in developing brain. Biochem. Biophys. Res. Commun. 215, 1995, 361-7.
6. d'Souza VN, Nguyen thi Man, Morris GE, Karges W, Pillers DM, Ray PN A novel dystrophin isoform is required for normal retinal electrophysiology Hum. Mol.. Genet. 4, 1995, 837-42.
7. Le TT, Nguyen thi Man, Helliwell TR, Morris GE Characterization of revertant muscle fibres in Duchenne Muscular Dystrophy using exon-specific monoclonal antibodies against dystrophin. Amer. J. Hum. Genet. 56, 1995, 725-31.
8. Lu QL, Morris GE,. Wilton SD, Ly T, Artem'yeva OV, Strong P, Partridge TA Massive Idiosyncratic Exon Skipping Corrects The Nonsense Mutation In Dystrophic Mouse Muscle And Produces Functional Revertant Fibres By Clonal Expansion J. Cell Biol. 148, 2000, 985-96.
9. Partridge TA, Lu QL, Morris GE, Hoffmann EP Is myoblast transplantation effective?Nature Med. 4, 1998, 1208-9.
10. Gussoni E, Bennett RR, Gilgoff I, Stein J, Chan Y, Muskiewicz KR, Lidov HG, Bönneman C, von Moers A, Morris GE, Chamberlain JS, Kunkel LM, Weinberg K Detection of donor nuclei in the muscles of a Duchenne muscular dystrophy patient 13 years following bone marrow transplantation J. Clin. Invest., 110, 2002, 807-14.
11. Nguyen thi Man, Morris GE A rapid method for generating large numbers of high-affinity monoclonal antibodies from a single mouse, in "The Protein Protocols Handbook" 3rd Edn. (ed. Walker JM) Humana Press, Totowa, NJ (2009) pp 1961-74.
12. Tunnah D., Sewry CA Vaux D, Schirmer EC, Morris GE The apparent absence of lamin B1 and emerin in many tissue nuclei is due to epitope masking. J. Mol. Histol., 36, 2005, 337-44. (Pubitemid 41740145)
13. Hori S, Ohtani S, Nguyen thi Man, Morris GE The N-terminal half of dystrophin is protected from proteolysis in situ. Biochem. Biophys. Res. Commun., 209, 1995, 1062-7.
14. Mendell JR, Kissel JT, Amato AA, King W, Signore L, Prior, TW et al. Myoblast transfer in the treatment of Duchenne's Muscular Dystrophy. New Engl. J. Med. 333, 1993, 832-8.
15. Romero NB, Braun S, Benveniste O, Leturcq F, Hogrel JY, Morris GE. et al Phase I study of dystrophin plasmid-based gene therapy in Duchenne/ Becker muscular dystrophy. Hum. Gene Therapy, 15, 2004, 1065-76. (Pubitemid 39552288)
16. Welch EM, Barton ER, Zhuo J, Tomizawa Y, Friesen WJ, Trifillis P et al. PTC124 targets genetic disorders caused by nonsense mutations. Nature 447, 2007, 87-91. (Pubitemid 46685839)
17. Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, Garralda ME, Rutherford M, McCulley C, Popplewell L, Graham IR, Dickson G, Wood MJ, Wells DJ, Wilton SD, Kole R, Straub V, Bushby K, Sewry C, Morgan JE, Muntoni F Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: A single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol. 8, 2009, 918-28.
18. Braun S, Thioudellet C, Rodriguez P, Ali-Hadji D, Perraud F, Accart N et al Immune rejection of human dystrophin following intramuscular injections of naked DNA in mdx mice. Gene Ther. 7, 2000, 1447-57.
19. Nguyen thi Man, Ellis JM, Love DR, Davies KE, Gatter KC, Dickson G, Morris, GE Localization of the DMDL-gene-encoded dystrophinrelated protein using a panel of 19 monoclonal antibodies. Presence at neuromuscular junctions, in the sarcolemma of dystrophic skeletal muscle, in vascular and other smooth muscles and in proliferating brain cell lines. J. Cell Biol. 115, 1991, 1695-700. (Pubitemid 21910009)
20. Burton EA, Tinsley JM, Holzfeind PJ, Rodrigues NR, Davies KE A second promoter provides an alternative target for therapeutic upregulation of utrophin in Duchenne muscular dystrophy. Proc Natl Acad Sci U S A. 96, 1999, 14025-30.
21. Malik V, Rodino-Klapac LR, Viollet L, Wall C, King W, Al-Dahhak R, Lewis S, Shilling CJ, Kota J, Serrano-Munuera C, Hayes J, Mahan JD, Campbell KJ, Banwell B, Dasouki M, Watts V, Sivakumar K, Bien- Willner R, Flanigan KM, Sahenk Z, Barohn RJ, Walker CM, Mendell JR Gentamicin-induced readthrough of stop codons in Duchenne muscular dystrophy. Ann Neurol. 67, 2010, 771-80.