Abstract
Attempting gene transfer in muscle raises difficult problems: the nuclei of mature muscle fibers do not undergo division, thus excluding strategies involving replicative integration of exogenous DNA. As adenovirus has been reported to be an efficient vector for the transfer of an enzyme encoding gene in mice, we decided to explore its potential for muscle cells. Advantages of adenovirus vectors are their independence of host cell replication, broad host range, and potential capacity for large foreign DNA inserts. We constructed a recombinant adenovirus containing the beta-galactosidase reporter gene under the control of muscle-specific regulatory sequences. This recombinant virus was able to direct expression of the beta-galactosidase in myotubes in vitro. We report its in vivo expression in mouse muscles up to 75 days after infection. The efficiency and stability of expression we obtained compare very favorably with other strategies proposed for gene or myoblast transfer in muscle in vivo.
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- Alonso S., Garner I., Vandekerckhove J., Buckingham M. Genetic analysis of the interaction between cardiac and skeletal actin gene expression in striated muscle of the mouse. J Mol Biol. 1990 Feb 20;211(4):727–738. doi: 10.1016/0022-2836(90)90073-U. [DOI] [PubMed] [Google Scholar]
- Berkner K. L. Development of adenovirus vectors for the expression of heterologous genes. Biotechniques. 1988 Jul-Aug;6(7):616–629. [PubMed] [Google Scholar]
- Donoghue M., Ernst H., Wentworth B., Nadal-Ginard B., Rosenthal N. A muscle-specific enhancer is located at the 3' end of the myosin light-chain 1/3 gene locus. Genes Dev. 1988 Dec;2(12B):1779–1790. doi: 10.1101/gad.2.12b.1779. [DOI] [PubMed] [Google Scholar]
- England S. B., Nicholson L. V., Johnson M. A., Forrest S. M., Love D. R., Zubrzycka-Gaarn E. E., Bulman D. E., Harris J. B., Davies K. E. Very mild muscular dystrophy associated with the deletion of 46% of dystrophin. Nature. 1990 Jan 11;343(6254):180–182. doi: 10.1038/343180a0. [DOI] [PubMed] [Google Scholar]
- Garver R. I., Jr, Chytil A., Courtney M., Crystal R. G. Clonal gene therapy: transplanted mouse fibroblast clones express human alpha 1-antitrypsin gene in vivo. Science. 1987 Aug 14;237(4816):762–764. doi: 10.1126/science.3497452. [DOI] [PubMed] [Google Scholar]
- Graham F. L., Smiley J., Russell W. C., Nairn R. Characteristics of a human cell line transformed by DNA from human adenovirus type 5. J Gen Virol. 1977 Jul;36(1):59–74. doi: 10.1099/0022-1317-36-1-59. [DOI] [PubMed] [Google Scholar]
- Harding A. E. Neurological disease and mitochondrial genes. Trends Neurosci. 1991 Apr;14(4):132–138. doi: 10.1016/0166-2236(91)90081-5. [DOI] [PubMed] [Google Scholar]
- Herbomel P., Bourachot B., Yaniv M. Two distinct enhancers with different cell specificities coexist in the regulatory region of polyoma. Cell. 1984 Dec;39(3 Pt 2):653–662. doi: 10.1016/0092-8674(84)90472-0. [DOI] [PubMed] [Google Scholar]
- Law P. K., Bertorini T. E., Goodwin T. G., Chen M., Fang Q. W., Li H. J., Kirby D. S., Florendo J. A., Herrod H. G., Golden G. S. Dystrophin production induced by myoblast transfer therapy in Duchenne muscular dystrophy. Lancet. 1990 Jul 14;336(8707):114–115. doi: 10.1016/0140-6736(90)91628-n. [DOI] [PubMed] [Google Scholar]
- Lee F., Hall C. V., Ringold G. M., Dobson D. E., Luh J., Jacob P. E. Functional analysis of the steroid hormone control region of mouse mammary tumor virus. Nucleic Acids Res. 1984 May 25;12(10):4191–4206. doi: 10.1093/nar/12.10.4191. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Morgan J. E., Hoffman E. P., Partridge T. A. Normal myogenic cells from newborn mice restore normal histology to degenerating muscles of the mdx mouse. J Cell Biol. 1990 Dec;111(6 Pt 1):2437–2449. doi: 10.1083/jcb.111.6.2437. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Partridge T. A., Morgan J. E., Coulton G. R., Hoffman E. P., Kunkel L. M. Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature. 1989 Jan 12;337(6203):176–179. doi: 10.1038/337176a0. [DOI] [PubMed] [Google Scholar]
- Rosenfeld M. A., Siegfried W., Yoshimura K., Yoneyama K., Fukayama M., Stier L. E., Päkkö P. K., Gilardi P., Stratford-Perricaudet L. D., Perricaudet M. Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science. 1991 Apr 19;252(5004):431–434. doi: 10.1126/science.2017680. [DOI] [PubMed] [Google Scholar]
- Sanes J. R., Rubenstein J. L., Nicolas J. F. Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos. EMBO J. 1986 Dec 1;5(12):3133–3142. doi: 10.1002/j.1460-2075.1986.tb04620.x. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Smith B. F., Hoffman R. K., Giger U., Wolfe J. H. Genes transferred by retroviral vectors into normal and mutant myoblasts in primary cultures are expressed in myotubes. Mol Cell Biol. 1990 Jun;10(6):3268–3271. doi: 10.1128/mcb.10.6.3268. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Stratford-Perricaudet L. D., Levrero M., Chasse J. F., Perricaudet M., Briand P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum Gene Ther. 1990 Fall;1(3):241–256. doi: 10.1089/hum.1990.1.3-241. [DOI] [PubMed] [Google Scholar]
- Webster C., Blau H. M. Accelerated age-related decline in replicative life-span of Duchenne muscular dystrophy myoblasts: implications for cell and gene therapy. Somat Cell Mol Genet. 1990 Nov;16(6):557–565. doi: 10.1007/BF01233096. [DOI] [PubMed] [Google Scholar]
- Wigler M., Sweet R., Sim G. K., Wold B., Pellicer A., Lacy E., Maniatis T., Silverstein S., Axel R. Transformation of mammalian cells with genes from procaryotes and eucaryotes. Cell. 1979 Apr;16(4):777–785. doi: 10.1016/0092-8674(79)90093-x. [DOI] [PubMed] [Google Scholar]
- Wilson J. M., Danos O., Grossman M., Raulet D. H., Mulligan R. C. Expression of human adenosine deaminase in mice reconstituted with retrovirus-transduced hematopoietic stem cells. Proc Natl Acad Sci U S A. 1990 Jan;87(1):439–443. doi: 10.1073/pnas.87.1.439. [DOI] [PMC free article] [PubMed] [Google Scholar]
- Wolff J. A., Malone R. W., Williams P., Chong W., Acsadi G., Jani A., Felgner P. L. Direct gene transfer into mouse muscle in vivo. Science. 1990 Mar 23;247(4949 Pt 1):1465–1468. doi: 10.1126/science.1690918. [DOI] [PubMed] [Google Scholar]
- Worton R. G., Thompson M. W. Genetics of Duchenne muscular dystrophy. Annu Rev Genet. 1988;22:601–629. doi: 10.1146/annurev.ge.22.120188.003125. [DOI] [PubMed] [Google Scholar]
- Yaffe D., Saxel O. Serial passaging and differentiation of myogenic cells isolated from dystrophic mouse muscle. Nature. 1977 Dec 22;270(5639):725–727. doi: 10.1038/270725a0. [DOI] [PubMed] [Google Scholar]
- Yang N. S., Burkholder J., Roberts B., Martinell B., McCabe D. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc Natl Acad Sci U S A. 1990 Dec;87(24):9568–9572. doi: 10.1073/pnas.87.24.9568. [DOI] [PMC free article] [PubMed] [Google Scholar]