Correction of the Dystrophic Phenotype by In Vivo Targeting of Muscle Progenitor Cells

Human Gene Therapy

Volumn 14, Issue 15, 2003, Pages 1441-1449

  Kobinger, Gary P ^a,c   Louboutin, Jean Pierre ^a,c   Barton, Elisabeth R ^b   Sweeney, H Lee ^b   Wilson, James M ^a,c  

^a Institute for Human Gene Therapy   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c WISTAR INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; LENTIVIRUS VECTOR; RECOMBINANT DNA;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CELL DIVISION; CELL POPULATION; CELL PROLIFERATION; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; ENZYME ENGINEERING; EXPERIMENTAL MODEL; EXPRESSION VECTOR; GENE FUNCTION; GENE TARGETING; MOUSE; MUSCLE CELL; MUSCULAR DYSTROPHY; NONHUMAN; PROTEIN EXPRESSION; SATELLITE CELL; SKELETAL MUSCLE; STEM CELL; VIRAL GENE THERAPY;

ANIMALS; BETA-GALACTOSIDASE; CELL DIFFERENTIATION; COLORING AGENTS; DYSTROPHIN; EVANS BLUE; GENE THERAPY; GENE TRANSFER TECHNIQUES; IMMUNOHISTOCHEMISTRY; LENTIVIRUS; MICE; MICE, INBRED C57BL; MICE, INBRED MDX; MUSCLE FIBERS; MUSCLE, SKELETAL; MUSCULAR DYSTROPHIES; NECROSIS; PHENOTYPE; STEM CELLS;

ANIMALIA; LENTIVIRUS; VECTORS;

EID: 0142042481     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/104303403769211655     Document Type: Article

References (24)

1. BARTON-DAVIS, E.R., SHOTURMA, D.I., MUSARO, A., ROSENTHAL, N., and SWEENEY, H.L. (1998). Viral mediated expression of insulin-like growth factor I blocks the aging-related loss of skeletal muscle function. Proc. Natl. Acad. Sci. U.S.A. 95, 15603-15607.
2. BEYER, W.R., MILETIC, H., OSTERTAG, W., and VON LAER, D. (2001). Recombinant expression of lymphocytic choriomeningitis virus strain WE glycoproteins: A single amino acid makes the difference. J. Virol. 75, 1061-1064.
3. BLOMER, U., NALDINI, L., KAFRI, T., TRONO, D., VERMA, I.M., and GAGE, F.H. (1997). Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J. Virol. 71, 6641-6649.
4. BROOKS, S.V., and FAULKNER, J.A. (1988). Contractile properties of skeletal muscles from young, adult and aged mice. J. Physiol. 404, 71-82.
5. DANGAIN, J., and VRBOVA, G. (1984). Muscle development in mdx mutant mice. Muscle Nerve 7, 700-704.
6. DUBOIS, C., FIGARELLA-BRANGER, D., PASTORET, C., RAMPINI, C., KARPATI, G., and ROUGON, G. (1994). Expression of NCAM and its polysialylated isoforms during mdx mouse muscle regeneration and in vitro myogenesis. Neuromuscul. Disord. 4, 171-182.
7. FOLLENZI, A., AILLES, L.E., BAKOVIC, S., GEUNA, M., and NALDINI, L. (2000). Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25, 217-222.
8. GILBERT, R., LIU, A., PETROF, B., NALBANTOGLU, J., and KARPATI, G. (2002). Improved performance of a fully gutted adenovirus vector containing two full-length dystrophin cDNAs regulated by a strong promoter. Mol. Ther. 6, 501.
9. GOLDRING, K., PARTRIDGE, T., and WATT, D. (2002). Muscle stem cells. J. Pathol. 197, 457-467.
10. HAECKER, S.E., STEDMAN, H.H., BALICE-GORDON, R.J., SMITH, D.B., GREELISH, J.P., MITCHELL, M.A., WELLS, A., SWEENEY, H.L., and WILSON, J.M. (1996). In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes. Hum. Gene Ther. 7, 1907-1914.
11. HARPER, S.Q., HAUSER, M.A., DELLORUSSO, C., DUAN, D., CRAWFORD, R.W., PHELPS, S.F., HARPER, H.A., ROBINSON, A.S., ENGELHARDT, J.F., BROOKS, S.V., and CHAMBERLAIN, J.S. (2002). Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy [see comments]. Nat. Med. 8, 253-261.
12. KOBINGER, G.P., WEINER, D.J., YU, Q.C., and WILSON, J.M. (2001). Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat. Biotechnol. 19, 225-230.
13. KOCHANEK, S., CLEMENS, P.R., MITANI, K., CHEN, H.H., CHAN, S., and CASKEY, C.T. (1996). A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc. Natl. Acad. Sci. U.S.A. 93, 5731-5736.
14. LODGE, R., SUBBRAMANIAN, R.A., FORGET, J., LEMAY, G., and COHEN, E.A. (1998). MuLV-based vectors pseudotyped with truncated HIV glycoproteins mediate specific gene transfer in CD4+ peripheral blood lymphocytes. Gene Ther. 5, 655-664.
15. LOUBOUTIN, J.P., ROUGER, K., TINSLEY, J.M., HALLDORSON, J., and WILSON, J.M. (2001). iNOS expression in dystrophinopathies can be reduced by somatic gene transfer of dystrophin or utrophin. Mol. Med. 7, 355-364.
16. MOCHIZUKI, H., SCHWARTZ, J.P., TANAKA, K., BRADY, R.O., and REISER, J. (1998). High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J. Virol. 72, 8873-8883.
17. NALDINI, L., BLOMER, U., GALLAY, P., ORY, D., MULLIGAN, R., GAGE, F.H., VERMA, I.M., and TRONO, D. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267.
18. PETROF, B.J., SHRAGER, J.B., STEDMAN, H.H., KELLY, A.M., and SWEENEY, H.L. (1993). Dystrophin protects the sarcolemma from stresses developed during muscle contraction. Proc. Natl. Acad. Sci. U.S.A. 90, 3710-3714.
19. RAGOT, T., VINCENT, N., CHAFEY, P., VIGNE, E., GILGENKRANTZ, H., COUTON, D., CARTAUD, J., BRIAND, P., KAPLAN, J.C., and PERRICAUDET, M. (1993). Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361, 647-650.
20. STEDMAN, H.H., SWEENEY, H.L., SHRAGER, J.B., MAGUIRE, H.C., PANETTIERI, R.A., PETROF, B., NARUSAWA, M., LEFEROVICH, J.M., SLADKY, J.T., and KELLY, A.M. (1991). The mdx mouse diaphragm reproduces the degenerative changes of Duchenne muscular dystrophy. Nature 352, 536-539.
21. TORRES, L.F., and DUCHEN, L.W. (1987). The mutant mdx: Inherited myopathy in the mouse. Morphological studies of nerves, muscles and end-plates. Brain 110, 269-299.
22. WANG, B., LI, J., and XIAO, X. (2000). Adeno-associated virus vector carrying human minidystrophin genes effectively ameliorates muscular dystrophy in mdx mouse model. Proc. Natl. Acad. Sci. U.S.A. 97, 13714-13719.
23. WATSON, D.J., KOBINGER, G.P., PASSINI, M.A., WILSON, J.M., and WOLFE, J.H. (2002). Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mol. Ther. 5, 528-537.
24. WOOL-LEWIS, R.J., and BATES, P. (1998). Characterization of Ebola virus entry by using pseudotyped viruses: Identification of receptor-deficient cell lines. J. Virol. 72, 3155-3160.