Viral vectors for gene transfer of micro-, mini-, or full-length dystrophin

Neuromuscular Disorders

Volumn 12, Issue SUPPL., 2002, Pages

  Scott, Jeannine M ^a   Li, Sheng ^a   Harper, Scott Q ^a   Welikson, Robert ^a   Bourque, Daniel ^a   DelloRusso, Christiana ^a   Hauschka, Stephen D ^a   Chamberlain, Jeffrey S ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

Adeno associated virus; Adenovirus; Dystrophin; Gene therapy; Lentivirus; mdx mouse

Indexed keywords

DYSTROPHIN; VIRUS VECTOR;

CONFERENCE PAPER; DUCHENNE MUSCULAR DYSTROPHY; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE SEQUENCE; GENE TRANSFER; HUMAN; NONHUMAN; PRIORITY JOURNAL; SKELETAL MUSCLE;

ADENOVIRIDAE; ANIMALS; DEPENDOVIRUS; DYSTROPHIN; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; LENTIVIRUS; MICE; MICE, INBRED MDX; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; MUSCULAR DYSTROPHY, DUCHENNE;

EID: 0036823730     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0960-8966(02)00078-0     Document Type: Conference Paper

References (39)

1. Koenig M., Hoffman E.P., Bertelson C.J., et al. Complete cloning of the Duchenne muscular dystrophy (DMD) cDNA and preliminary genomic organization of the DMD gene in normal and affected individuals. Cell. 50:1987;509-517.
2. Hoffman E.P., Brown R.H. Jr, Kunkel L.M. Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell. 51:1987;919-928.
3. Hartigan-O'Connor D., Chamberlain J.S. Developments in gene therapy for muscular dystrophy. Microsc Res Tech. 48:2000;23-238.
4. Kumar-Singh R., Chamberlain J.S. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Hum Mol Genet. 5:1996;913-921.
5. Kochanek S., Clemens P.R., Mitani K., et al. A new adenoviral vector: replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc Natl Acad Sci USA. 93:1996;5731-5736.
6. Hartigan-O'Connor D., Barjot C., Salvatori G., et al. Generation and growth of gutted adenoviral vectors. Methods Enzymol. 346:2002;224-246.
7. Naldini L., Verma I.M. Lentiviral vectors. Adv Virus Res. 55:2000;599-609.
8. Xiao X., Li J., Samulski R.J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol. 70:1996;8098-8108.
9. Snyder R.O., Spratt S.K., Lagarde C., et al. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum Gene Ther. 8:1997;1891-1900.
10. Fisher K.J., Jooss K., Alston J., et al. Recombinant adeno-associated virus for muscle directed gene therapy. Nat Med. 3:1997;306-312.
11. Koenig M., Monaco A.P., Kunkel L.M. The complete sequence of dystrophin predicts a rod-shaped cytoskeletal protein. Cell. 53:1988;219-226.
12. Koenig M., Kunkel L.M. Detailed analysis of the repeat domain of dystrophin reveals four potential hinge segments that may confer flexibility. J Biol Chem. 265:1990;4560-4566.
13. Hoffman E.P., Garcia C.A., Chamberlain J.S., et al. Is the carboxyl-terminus of dystrophin required for membrane association? A novel, severe case of Duchenne muscular dystrophy. Ann Neurol. 30:1991;605-610.
14. Passos-Bueno M.R., Vainzof M., Marie S.K., et al. Half the dystrophin gene is apparently enough for a mild clinical course: confirmation of its potential use for gene therapy. Hum Mol Genet. 3:1994;919-922.
15. England S.B., Nicholson L.V., Johnson M.A., et al. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature. 343:1990;180-182.
16. Cox G.A., Cole N.M., Matsumura K., et al. Overexpression of dystrophin in transgenic mdx mice eliminates dystrophic symptoms without toxicity [see comments]. Nature. 364:1993;725-729.
17. Phelps S.F., Hauser M.A., Cole N.M., et al. Expression of full-length and truncated dystrophin mini-genes in transgenic mdx mice. Hum Mol Genet. 4:1995;1251-1258.
18. Corrado K., Rafael J.A., Mills P.L., et al. Transgenic mdx mice expressing dystrophin with a deletion in the actin-binding domain display a 'mild Becker' phenotype. J Cell Biol. 134:1996;873-884.
19. Warner L.E., Dello Russo C., Crawford R., et al. Expression of Dp260 in muscle tethers the actin cytoskeletan to the dystrophin-glycoprotein complex. Hum Mol Genet. 11:2002;1095-1105.
20. Crawford G.E., Faulkner J.A., Crosbie R.H., et al. Assembly of the dystrophin-associated protein complex does not require the dystrophin COOH-terminal domain. J Cell Biol. 150:2000;1399-1410.
21. Rafael J.A., Cox G.A., Corrado K., et al. Forced expression of dystrophin deletion constructs reveals structure-function correlations. J Cell Biol. 134:1996;93-102.
22. Harper S.Q., Hauser M.A., Dello Russo C., et al. Modular flexibility of dystrophin: implications for gene therapy of Duchenne muscular dystrophy. Nat Med. 8:2002;253-261.
23. Hartigan-O'Connor D., Kirk C.J., Crawford R., et al. Immune evasion by muscle-specific gene expression in dystrophic muscle. Mol Ther. 4:2001;525-533.
24. Hauser M.A., Robinson A., Hartigan-O'Connor D., et al. Analysis of muscle creatine kinase regulatory elements in recombinant adenoviral vectors. Mol Ther. 2:2000;16-25.
25. Rutledge E.A., Halbert C.L., Russell D.W. Infectious clones and vectors derived from adeno-associated virus (AAV) serotypes other than AAV type 2. J Virol. 72:1998;309-319.
26. Naldini L., Blomer U., Gallay P., et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science. 272:1996;263-267.
27. Kafri T., Blomer U., Peterson D.A., et al. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet. 17:1997;314-317.
28. De Palma M., Naldini L. Transduction of a gene expression cassette using advanced generation lentiviral vectors. Methods Enzymol. 346:2002;514-529.
29. Barry S.C., Harder B., Brzezinski M., et al. Lentivirus vectors encoding both central polypurine tract and posttranscriptional regulatory element provide enhanced transduction and transgene expression. Hum Gene Ther. 12:2001;1103-1108.
30. Hauser M.A., Amalfitano A., Kumar-Singh R., et al. Improved adenoviral vectors for gene therapy of Duchenne muscular dystrophy. Neuromuscul Disord. 7:1997;277-283.
31. Barjot C, Hartigan-O'Connor D, Salvatori G, et al., Gutted adenoviral vector growth using E1/E2b/E3-deleted helper viruses. J Gene Med 2002; in press.
32. Dello Russo C, Scott J, Hartigan-O'Connor D, et al. Functional correction of adult mdx mouse muscle using gutted adenoviral vectors expressing full-length dystrophin. Submitted for publication.
33. Lu Q.L., Morris G.E., Wilton S.D., et al. Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion. J Cell Biol. 148:2000;985-996.
34. Crawford G.E., Lu Q.L., Partridge T.A., et al. Suppression of revertant fibers in mdx mice by expression of a functional dystrophin. Hum Mol Genet. 10:2001;2745-2750.
35. Bett A.J., Prevec L., Graham F.L. Packaging capacity and stability of human adenovirus type 5 vectors. J Virol. 67:1993;5911-5921.
36. Jackson K.A., Mi T., Goodell M.A. Hematopoietic potential of stem cells isolated from murine skeletal muscle. Proc Natl Acad Sci USA. 96:1999;14482-14486.
37. Gussoni E., Soneoka Y., Strickland C.D., et al. Dystrophin expression in the mdx mouse restored by stem cell transplantation. Nature. 401:1999;390-394.
38. Clegg C.H., Linkhart T.A., Olwin B.B., et al. Growth factor control of skeletal muscle differentiation: commitment to terminal differentiation occurs in G1 phase and is repressed by fibroblast growth factor. J Cell Biol. 105:1987;949-956.
39. Lumeng C.N., Phelps S.F., Rafael J.A., et al. Characterization of dystrophin and utrophin diversity in the mouse. Hum Mol Genet. 8:1999;593-599.