Expression of a recombinant dystrophin in mdx mice using adenovirus vector

Alameddine, Hala S.; Quantin, Beatrice; Cartaud, Annie; Dehaupas, Michèle; Mandel, Jean‐Louis; Fardeau, Michel

doi:10.1016/0960-8966(94)90020-5

Cited by 24 publications

(11 citation statements)

References 18 publications

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“…These have been shown to produce adequate levels of expression of reporter genes 11 and dystrophin. 12,13 However, only a relatively low percentage of positive fibres was achieved. In addition, the use of viral vectors could lead to recombinant events, for example during muscle regeneration after injury, and the activation of endogenous genes.…”

Section: Discussionmentioning

confidence: 99%

Myosin regulatory elements as vectors for gene transfer by intramuscular injection

et al. 1998

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Section: Discussionmentioning

confidence: 99%

Myosin regulatory elements as vectors for gene transfer by intramuscular injection

et al. 1998

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“…Dystro-phin was detected with a rabbit polyclonal antibody kindly provided by Dr A Cartaud (Institut J Monod, Université Paris VII, Paris, France) as previously described, 68 using a Cy3-labeled second antibody.…”

Section: Laminin-2 and Dystrophin Immunohistochemistrymentioning

confidence: 99%

Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies

Vilquin

Kennel²,

Paturneau-Jouas

et al. 2001

Gene Ther

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The electrotransfer of naked DNA has recently been adapted to the transduction of skeletal muscle fibers. We investigated the short-and long-term efficacy of this methodology in wild-type animals and in mouse models of congenital muscular dystrophy (dy/dy, dy 2J /dy 2J ), or Duchenne muscular dystrophy (mdx/mdx). Using a reporter construct, the short-term efficacy of fiber transduction reached 40% and was similar in wild-type, dy/dy and dy 2J /dy 2J animals, indicating that ongoing muscle fibrosis was not a major obstacle to the electrotransfer-mediated gene transfer. Although the complete rejection of transduced fibers was observed within 3 weeks in the absence of immunosuppression, the persistency was prolonged over 10 weeks when transient or continuous immunosuppressive regimens were used. Using

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“…In animal studies, replication-defective adenoviral vectors have been used for the transfer of genes encoding marker proteins (5), secretory proteins (6)(7)(8), and truncated forms of dystrophin to muscle (9)(10)(11)(12)(13)(14). T cell-mediated and B cell antibody immune responses to first generation adenoviral vectors have been observed in a variety of tissues, including skeletal muscle (7).…”

mentioning

confidence: 99%

Persistence in muscle of an adenoviral vector that lacks all viral genes

Chen

Mack

Kelly

et al. 1997

Proc. Natl. Acad. Sci. U.S.A.

276

153

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Genetic correction of inherited muscle diseases, such as Duchenne muscular dystrophy, will require long term expression of the recombinant protein following gene transfer. We have shown previously that a new adenoviral vector that lacks all viral genes expressed both full-length dystrophin and ␤-galactosidase in mdx (dystrophin-deficient) mouse muscle. We observed a significant histologic improvement of vector-transduced mdx muscle before the eventual loss of vector-encoded transgene expression. In this study, we investigated whether an immunological response against vector-encoded ␤-galactosidase contributed to the loss of vector expression and affected vector persistence in muscle. Intramuscular vector injection in control normal mice resulted in an early and complete loss of ␤-galactosidase expression accompanied by predominantly CD4 ؉ and CD8؉ lymphocytic infiltration and a significant loss of vector DNA. In contrast, intramuscular vector injection in lacZ transgenic mice resulted in persistent expression of ␤-galactosidase for at least 84 days with no evidence of inf lammation or significant loss of vector DNA. Our studies demonstrate that, in the absence of an immune response induced by ␤-galactosidase expression, an adenoviral vector lacking all viral genes is stably maintained in muscle.

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Expression of a recombinant dystrophin in mdx mice using adenovirus vector

Cited by 24 publications

References 18 publications

Myosin regulatory elements as vectors for gene transfer by intramuscular injection

Myosin regulatory elements as vectors for gene transfer by intramuscular injection

Electrotransfer of naked DNA in the skeletal muscles of animal models of muscular dystrophies

Persistence in muscle of an adenoviral vector that lacks all viral genes

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