2008
DOI: 10.1038/mt.2008.23
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Abstract: Duchenne muscular dystrophy (DMD) is a fatal, progressive, muscle-wasting disease caused by defects in the dystrophin. No viral vector except the helper-dependent adenovirus vector (HDAdv) can package 14-kilobase (kb) full-length dystrophin complementary DNA (cDNA), and HDAdv is considerably safer than old-generation adenovirus vectors because of the large-size deletion in its genome. We have generated HDAdv that carries myc-tagged murine full-length dystrophin cDNA (HDAdv-myc-mFLdys). We injected it into mult… Show more

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Cited by 30 publications
(21 citation statements)
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“…Moreover, we were surprised at the increased longevity of mice after transplantation. No dko mouse survived longer than 20 weeks after birth 17 , but nearly all dko/MSC mice survived after 20 weeks, and some lived for 1 year (Fig. 1g).…”
Section: Resultsmentioning
confidence: 99%
“…Moreover, we were surprised at the increased longevity of mice after transplantation. No dko mouse survived longer than 20 weeks after birth 17 , but nearly all dko/MSC mice survived after 20 weeks, and some lived for 1 year (Fig. 1g).…”
Section: Resultsmentioning
confidence: 99%
“…Previous reports on mdx:utr Ϫ/Ϫ mouse life span is highly variable, ranging from as little as 4 wk to as many as 36 wk (5,8,10,13,15,17,24). This variability may reflect genetic drift within respective colonies and local environmental factors idiosyncratic to each housing facility (e.g., staff, cage sizes, husbandry, handling, food enrichment options, room noise, architecture, temperature, etc.).…”
Section: Discussionmentioning
confidence: 99%
“…Muscle function is compromised by the destabilization of the sarcolemma, a result of dystrophin deficiency (2), rendering skeletal muscle susceptible to contraction-induced injury (26). Without a cure, strategies to mitigate the disease progression and improve muscle function have been developed to compensate for dystrophin deficiency by boosting the presence of dystrophin-like cytoskeletal proteins (5,15,17,25). Specifically, utrophin, a protein homologue of dystrophin, sufficiently compensates for dystrophin and improves the phenotype of mdx mice, the primary animal model for DMD (11,28,30,33).…”
mentioning
confidence: 99%
“…Multiple proximal muscles of seven-day-old utrophin/dystrophin double knockout mice ( dko mice), which typically show symptoms similar to human DMD, were effectively transduced with the gutted adenovirus bearing full-length murine dystrophin cDNA [53] However, further extraordinary improvements would be required to regulate the adenovirus-associated severe inflammation before clinical trials might be considered, since the adenovirus proteins elicit a more potent immune response in vivo compared to the AAV capsids [39]. …”
Section: Gene-replacement Strategies Using Virus Vectorsmentioning
confidence: 99%