Duchenne muscular dystrophy (DMD) is an X-linked genetic disease characterized by the absence of dystrophin (427 kDa). An approach to eventually restore this protein in patients with DMD is to introduce into their muscles a plasmid encoding dystrophin cDNA. Because the phenotype of the dystrophic dog is closer to the human phenotype than is the mdx mouse phenotype, we have studied the electrotransfer of a plasmid carrying the full-length dog dystrophin (FLDYSdog) in dystrophic dog muscle. To achieve this nonviral delivery, the FLDYSdog cDNA was cloned in two plasmids containing either a cytomegalovirus or a muscle creatine kinase promoter. In both cases, our results showed that the electrotransfer of these large plasmids (∼17 kb) into mouse muscle allowed FLDYSdog expression in the treated muscle. The electrotransfer of pCMV.FLDYSdog in a dystrophic dog muscle also led to the expression of dystrophin. In conclusion, introduction of the full-length dog dystrophin cDNA by electrotransfer into dystrophic dog muscle is a potential approach to restore dystrophin in patients with DMD. However, the electrotransfer procedure should be improved before applying it to humans. In this report by Picahavant et al., the authors examine the efficacy of electrotransfer of a plasmid containing dog dystrophin in a mouse and dog model of Duchenne muscular dystrophy (DMD). They demonstrate that this approach leads not only to the expression of dystrophin in dog muscle, but also to mononuclear cell infiltration.

mdx mouse

Utrophin

Muscle disorder

10.1089/hum.2010.024

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Citations (19)

Examination of Galectin Localization Using Confocal Microscopy

Methods in molecular biology (2014)

Daniel Giuliano Cerri Connie M. Arthur Lílian Cataldi Rodrigues Marise Lopes Fermino Lenaldo Branco Rocha

Immunofluorescence

Optical sectioning

10.1007/978-1-4939-1396-1_23

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Citations (5)

FAK deficient mast cells: response to stimulation via Fc'epsilon'RI

Journal of Histochemistry & Cytochemistry (2004)

Maria Célia Jamur Daniel Giuliano Cerri Constance Oliver

Mast (botany)

Source

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Leishmanicidal sesquiterpene lactones from Tithonia diversifolia

Planta Medica (2014)

SR Ambrósio JS de Toledo C.C. Borges Viviane Manfrim Daniel Giuliano Cerri

Sesquiterpene lactones (SLs) are recognized as a class of natural compounds with a wide spectrum of biological activities, including against Leishmania spp. In this work, a SL rich extract from Tithonia diversifolia (SLRE) was tested against promastigote forms of L. braziliensis. This result revealed that SLRE is a rich source of potent leishmanicidal compounds (LD50= 1.5 ± 0.50 µg/mL). Therefore eight sesquiterpene lactones from SLRE were investigated and three of them (1 – 3) showed potent leishmanicidal effect with high level of selectivity, displaying LD50 values (ranging from 21.2 ± 8.0 to 37.4 ± 7.1µM) similar to that observed for positive control (hygromycin B; 15.1 ± 0.9µM). Due to these results, compounds 1 – 3 were evaluated at their LD50 concentrations on intracellular amastigotes. After 48 hours of treatment these SLs reduced the internalization of parasites in about 58, 51 and 27%, respectively, in comparison with negative control. The results described herein bring new perspectives in the study of STL and may provide promising leads for the development of new drugs against leishmaniasis.

Tithonia

Amastigote

Sesquiterpene lactone

10.1055/s-0034-1382485

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G.P.6.04 Imatinib mesilate (Gleevec®) ameliorates the dystrophic phenotype in exercised mdx mice

Neuromuscular Disorders (2007)

João Carlos da Silva Bizário Daniel Giuliano Cerri Fabiana Matioli Paola Morales Lucélio Bernardes Couto

Creatine kinase

Muscle weakness

10.1016/j.nmd.2007.06.139

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