P2RX7 Purinoceptor: A Therapeutic Target for Ameliorating the Symptoms of Duchenne Muscular Dystrophy

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dc.cclicenceCC-BYen
dc.contributor.authorSinadinos, Anthonyen
dc.contributor.authorYoung, Christopher N. J.en
dc.contributor.authorAl-Khalidi, Rashaen
dc.contributor.authorTeti, Annaen
dc.contributor.authorKalinski, Pawelen
dc.contributor.authorMohamad, Shafinien
dc.contributor.authorFloriot, Léonoreen
dc.contributor.authorHenry, Tiphaineen
dc.contributor.authorTozzi, Gianlucaen
dc.contributor.authorJiang, Taiwenen
dc.contributor.authorWurtz, Olivieren
dc.contributor.authorLefebvre, Alexisen
dc.contributor.authorShugay, Mikhailen
dc.contributor.authorTong, Jieen
dc.contributor.authorVaudry, Daviden
dc.contributor.authorArkle, Stephenen
dc.contributor.authordoRego, Jean-Claudeen
dc.contributor.authorGórecki, Dariuszen
dc.date.acceptance2015-09-01en
dc.date.accessioned2017-12-18T16:24:24Z
dc.date.available2017-12-18T16:24:24Z
dc.date.issued2015-10-13
dc.descriptionopen access articleen
dc.description.abstractDuchenne muscular dystrophy (DMD) is the most common inherited muscle disease, leading to severe disability and death in young men. Death is caused by the progressive degeneration of striated muscles aggravated by sterile inflammation. The pleiotropic effects of the mutant gene also include cognitive and behavioral impairments and low bone density. Current interventions in DMD are palliative only as no treatment improves the long-term outcome. Therefore, approaches with a translational potential should be investigated, and key abnormalities downstream from the absence of the DMD product, dystrophin, appear to be strong therapeutic targets. We and others have demonstrated that DMD mutations alter ATP signaling and have identified P2RX7 purinoceptor up-regulation as being responsible for the death of muscles in the mdx mouse model of DMD and human DMD lymphoblasts. Moreover, the ATP–P2RX7 axis, being a crucial activator of innate immune responses, can contribute to DMD pathology by stimulating chronic inflammation. We investigated whether ablation of P2RX7 attenuates the DMD model mouse phenotype to assess receptor suitability as a therapeutic target.en
dc.funderDuchenne Parents Project NL (https://www. duchenne.nl/) to DCG, Muscular Dystrophy Association USA (MDA294571; http://mda.org/) to DCG, and the EU Interreg IV (TC2N; http://www. interreg4a-2mers.eu/en) to DV.en
dc.identifier.citationSinadinos, A., et al. (2015) P2X7 purinoceptor ablation ameliorates muscle and non-muscle pathology in the mdx mouse model of Duchenne muscular dystrophy. PLOS Medicine. 12 (10): e1001888.en
dc.identifier.doihttps://doi.org/10.1371/journal.pmed.1001888
dc.identifier.urihttp://hdl.handle.net/2086/15015
dc.language.isoenen
dc.peerreviewedYesen
dc.projectidMDA294571en
dc.publisherPLOS ONEen
dc.researchinstituteLeicester Institute for Pharmaceutical Innovation - From Molecules to Practice (LIPI)en
dc.titleP2RX7 Purinoceptor: A Therapeutic Target for Ameliorating the Symptoms of Duchenne Muscular Dystrophyen
dc.typeArticleen

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