Mini-dystrophin gene transfer in mdx4cv diaphragm muscle fibers increases sarcolemmal stability

Decrouy, A.; Jm, Renaud; Hl, Davis; Ja, Lunde; Dickson, George; Bj, Jasmin

doi:10.1038/sj.gt.3300407

Cited by 44 publications

(33 citation statements)

References 9 publications

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“…These data are consistent with those recently reported by Decrouy et al, 30 who also found no significant alteration in the decline of force-generating capacity caused by eccentric contractions in adult mdx mouse diaphragms studied 3 weeks after plasmidmediated dystrophin minigene transfer. On the other hand, in neonatal mdx gastrocnemius muscles injected with AdV-Dys and studied 14 weeks later, 27 the percentage decline in force-generating capacity after eccentric contractions was significantly blunted.…”

Section: Discussionsupporting

confidence: 93%

Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice

et al. 1998

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Duchenne muscular dystrophy (DMD) and murine X-linked logy and muscle weakness. The main findings are as folmuscular dystrophy (mdx) are both due to absence of the lows: (1) acute myofiber toxicity and gene transfer subsarcolemmal protein dystrophin. Recombinant adenoefficiency are both AdV dose-dependent, such that the virus vectors (AdV) are considered a promising means for therapeutic margin of safety is fairly narrow; (2) immunodelivering a functional dystrophin gene to muscle. Howsuppressive therapy (FK506) prevents immune-mediated ever, the usefulness of AdV for this purpose is limited by elimination of dystrophin-positive fibers but not the dosevector toxicity as well as immune-mediated elimination of dependent toxic effects; (3) at the optimal vector dosage infected fibers. In addition, studies to date of AdV-mediated and with effective immunosuppression, AdV-mediated dysdystrophin gene transfer have either failed to examine trophin minigene transfer is capable of alleviating the loss effects on muscle strength or been performed in immunoof force-generating capacity as well as histopathological logically immature neonatal animals with little baseline evidence of disease progression normally seen in adult abnormality of force-generating capacity. In the present mdx muscles over a 2-month period. These findings have study, AdV-mediated dystrophin gene transfer was perforimportant implications for the eventual application of AdVmed in adult mdx mice with pre-existent dystrophic pathomediated dystrophin gene transfer in DMD patients.

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Section: Discussionsupporting

confidence: 93%

Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice

et al. 1998

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“…They are nonpathogenic in humans, have a wide host cell range, can transduce nondividing cells and are integrated or retained in target cells. 13 On the other hand, nonviral delivery systems such as cationic liposomes [14][15][16][17] and direct injection of plasmid vectors into skeletal muscle [18][19][20] are also established and offer safety and simplicity. Recently, Baudard et al 21 proposed liposome vehicles for transfection of AAV-based plasmid vectors; this combination overcomes their individual disadvantages, including the package size limit of AAV vectors and the transient nature of transfections using conventional liposome-mediated plasmid vectors.…”

Section: Introductionmentioning

confidence: 99%

Efficient coexpression and secretion of anti-atherogenic human apolipoprotein AI and lecithin-cholesterol acyltransferase by cultured muscle cells using adeno-associated virus plasmid vectors

et al. 1998

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Plasma apolipoprotein AI (apoAI) and lecithin-cholesterolThe secretion of apoAI or LCAT by transduced cultures acyltransferase (LCAT) play important roles in reverse was two-to five-fold higher using AAV-based plasmid veccholesterol transport, promoting the removal of excess tors than conventional plasmid vectors. Additionally, cells cholesterol from peripheral cells and reducing formation of transfected with a bicistronic AAV-based vector containing atherosclerotic lesions. Gene augmentation of either apoAI an internal ribosome entry site (IRES) efficiently expressed or LCAT, or both, are thus attractive targets for prevention both apoAI and LCAT simultaneously. Furthermore, AAVor treatment of atherosclerosis. With the eventual aim of based vector sequences were retained by host cells, safe and efficient gene delivery to skeletal muscle, our whereas those of conventional plasmid vectors were lost. chosen secretory platform for systemic delivery of antiThese studies indicate that ectopic overexpression of atherogenic proteins, we have constructed conventional apoAI and LCAT in muscle tissue using AAV-based and AAV-based plasmid vectors containing human apoAI plasmid vectors might provide a feasible anti-atherogenic or LCAT cDNAs; their efficacy was tested by lipoplex transstrategy in vivo. fection of mouse C2C12 muscle cells or human 293 cells.

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“…The main disadvantage of nonviral gene delivery, however, is the relative low efficiency of these methods in general. Development of delivery systems including direct plasmid DNA injection, 7 lipofection, 14,15 HVJ-liposomes, 16 ligand-directed gene delivery and particle bombardment, 17 have attempted to overcome this. In addition to problems of delivery, the plasmid-based eukaryotic expression vectors conventionally used do not replicate and so are not retained in the nucleus of rapidly dividing cells, with plasmid DNA being rapidly diluted.…”

Section: Introductionmentioning

confidence: 99%

“…[3][4][5][6] Various studies have shown that full-length and Becker-type mini-dystrophin (6.3 kb) cDNA gene transfer is able to prevent the onset of dystrophy in dystrophindeficient mdx mice. 3,[7][8][9][10] The mdx mouse has a point mutation in the dystrophin gene which results in the virtual complete absence of the protein. Using adenoviral and retroviral vectors for gene delivery several studies have shown that both full-length and truncated forms of dystrophin are able to locate to the sarcolemma of muscle fibres of mdx mice.…”

Section: Introductionmentioning

confidence: 99%

“…Recently, we have reported the successful direct injection of a mini-dystrophin plasmid (pRSVDy-B) into mdx 4 cv diaphragm muscle fibres. 7,8 In that study, expression of dystrophin in approximately 17% of diaphragm muscle fibres resulted in significant protection of fibres from the damaging effect of repetitive eccentric lengthening contraction, and the recovery of NO synthase at the sarcolemma. Previous studies in transgenic mdx mice have demonstrated that expression of recombinant dystrophin at levels as low as 20% of normal endogenous levels at the myofibre membrane can completely prevent the development of the dystrophic phenotype.…”

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confidence: 99%

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Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein–Barr virus (EBV)-based mini-chromosome vectors in mdx mice

et al. 1999

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Gene transfer by direct intramuscular injection of naked myotube cultures. Intramuscular injection of EBV-based plasmid DNA has been shown to be a safe, simple but dystrophin expression plasmids into nude/mdx mice relatively inefficient method for gene delivery in vivo. Eukaresulted in significant enhancement in the number of musryotic plasmid expression vectors incorporating the cle fibres expressing recombinant dystrophin compared Epstein-Barr virus (EBV) origin of replication (oriP) and with a conventional vector. This effect was observed for EBNA1 gene have been shown to act as autonomous epiover 10 weeks after a single administration. These results somally replicating gene transfer vectors which additionally indicate the potential advantage of EBV-based expression provide nuclear matrix retention functions. Prolonged vectors for focal plasmid-mediated gene augmentation expression of a LacZ reporter gene and recombinant therapy in Duchenne muscular dystrophy (DMD) and a human dystrophin was shown using EBV-based plasmid range of other gene therapeutic applications. vectors transfected into C2C12 mouse myoblast and

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Mini-dystrophin gene transfer in mdx4cv diaphragm muscle fibers increases sarcolemmal stability

Cited by 44 publications

References 9 publications

Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice

Adenovirus-mediated dystrophin minigene transfer improves muscle strength in adult dystrophic (MDX) mice

Efficient coexpression and secretion of anti-atherogenic human apolipoprotein AI and lecithin-cholesterol acyltransferase by cultured muscle cells using adeno-associated virus plasmid vectors

Enhanced expression of recombinant dystrophin following intramuscular injection of Epstein–Barr virus (EBV)-based mini-chromosome vectors in mdx mice

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