High-level dystrophin expression after adenovirus-mediated dystrophin minigene transfer to skeletal muscle of dystrophic dogs: Prolongation of expression with immunosuppression

Human Gene Therapy

Volumn 9, Issue 5, 1998, Pages 629-634

  Howell, John McC ^a,b   Lochmüller, Hanns ^a,e   O'Hara, Amanda ^a,b   Fletcher, Susan ^b   Kakulas, Byron A ^b   Massie, Bernard ^c   Nalbantoglu, Josephine ^d   Karpati, George ^d  

^a MURDOCH UNIVERSITY   (Australia)

^b PATHWEST LABORATORY MEDICINE   (Australia)

^c NATIONAL RESEARCH COUNCIL CANADA   (Canada)

^d MCGILL UNIVERSITY   (Canada)

^e UNIVERSITY OF MUNICH   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIGEN; CYCLOSPORIN; DYSTROPHIN; VIRUS VECTOR;

ADENOVIRUS; ANIMAL MODEL; ARTICLE; CELLULAR IMMUNITY; CONTROLLED STUDY; DNA TRANSFER; DOG; DUCHENNE MUSCULAR DYSTROPHY; FEMALE; GENE EXPRESSION REGULATION; GENE TRANSFER; HUMORAL IMMUNITY; IMMUNOMODULATION; MALE; MOUSE; NONHUMAN; PHENOTYPE; PROTEIN EXPRESSION; SKELETAL MUSCLE; TRANSGENE;

ADENOVIRIDAE; ANIMALIA; CANIS FAMILIARIS;

EID: 0032549685     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1998.9.5-629     Document Type: Article

References (10)

1. ACSADI, G., LOCHMÜLLER, H., JANI, A., HUARD, J., MASSIE, B., PRESCOTT, S., SIMONEAU, M., PETROF, B., and KARPATI, G. (1996). Dystrophin expression in muscles of mdx mice after adenovirus-mediated in vivo gene transfer. Hum. Gene Ther. 7, 129-140.
2. CLEMENS, P.R., KOCHANEK, S., SUNADA, Y., CHAN, S., CHEN, H.H., CAMPBELL, K.P., and CASKEY, C.T. (1996). In vivo muscle gene transfer of full length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther. 3, 965-972.
3. COOPER, B.J., WINAND, N.J., STEDMAN, H., VALENTINE, B.A., HUFFMAN, E.P., KUNKEL, L.M., SCOTT, M.O., FISCHBECK, K.H., KORNEGAY, J.N., AVERY, R.J., WILLIAMS, J.R., SCHMICKEL, R.D., and SYLVESTER, J.E. (1988). The homologue of the Duchenne locus is defective in X-linked muscular dystrophy of dogs. Nature 334, 154-156.
4. TRIPATHY, S., BLACK, H., GOLDWASSER, E., and LEIDEN, J.M. (1996). Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 545-550.
5. VILQUIN, J., WAGNER, E., KINOSHITA, I., ROY, R., and TREMBLAY, J.P. (1995). Successful histocompatible myoblast transplantation in dystrophin-deficient mdx mouse despite the production of antibodies against dystrophin. J. Cell. Biol. 131, 975-988.
6. WANG, Q., JIA, X., and FINER, M. (1995). A packaging cell line for propagation of recombinant adenovirus vectors containing two lethal gene-region deletions. Gene Then 2, 775-783.
7. XIAO, X.A., LI, J.A., and SAMULSKI, R.J. (1996). Efficient long term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70, 8098-8108.
8. YANG, Y., TRINCHIERI, G., and WILSON, J.M. (1995). Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nature Med. 1, 890-893.
9. YANG, Y.P., HAECKER, S.E., SU, Q., and WILSON, J.M. (1996). Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum. Mol. Genet. 5, 1703-1712.
10. YANG, L., LOCHMÜLLER, H., LUO, J., MASSIE, B., NALBANTOGLU, J., KARPATI, G., and PETROF, B. (1997). Adenovirusmediated dystrophin minigene transfer improves muscle strength in adult dystrophic (mdx) mice. Gene Then (in press).