2016
DOI: 10.1016/j.nbd.2016.07.017
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Tolerability and efficacy study of P2X7 inhibition in experimental Charcot-Marie-Tooth type 1A (CMT1A) neuropathy

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Cited by 29 publications
(31 citation statements)
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“…n = 3-7 per group; t tests; # p < 0.05 CMT1A placebo versus WT placebo; *p < 0.05 CMT1A PXT3003 versus CMT1A placebo; dot plots and means (c and d) [Color figure can be viewed at wileyonlinelibrary.com] weight and to keep animal numbers as low as needed to minimize animal's burden for ethical reasons. Despite this limitation, using only one sex is considered sufficient since gender differences were not observed in CMT1A rats on disease severity with regard to molecular, electrophysiological, histological, and phenotypical characteristics (Chumakov et al, 2014;Fledrich et al, 2012Fledrich et al, , 2014Fledrich et al, , 2018Meyer zu Horste et al, 2007;Prukop et al, 2019;Sereda et al, 1996;Sereda, Meyer zu Hörste, Suter, Uzma, & Nave, 2003;Sociali et al, 2016). Moreover, no gender effect was reported in male and female CMT1A rats treated independently with Onapristone (Meyer zu Horste et al, 2007;Sereda et al, 2003), and no gender effect was observed in CMT1A patients after therapy with PXT3003 (Attarian et al, 2014) (Clinical Trials Register/Results, 2019.…”
Section: Discussionmentioning
confidence: 99%
“…n = 3-7 per group; t tests; # p < 0.05 CMT1A placebo versus WT placebo; *p < 0.05 CMT1A PXT3003 versus CMT1A placebo; dot plots and means (c and d) [Color figure can be viewed at wileyonlinelibrary.com] weight and to keep animal numbers as low as needed to minimize animal's burden for ethical reasons. Despite this limitation, using only one sex is considered sufficient since gender differences were not observed in CMT1A rats on disease severity with regard to molecular, electrophysiological, histological, and phenotypical characteristics (Chumakov et al, 2014;Fledrich et al, 2012Fledrich et al, , 2014Fledrich et al, , 2018Meyer zu Horste et al, 2007;Prukop et al, 2019;Sereda et al, 1996;Sereda, Meyer zu Hörste, Suter, Uzma, & Nave, 2003;Sociali et al, 2016). Moreover, no gender effect was reported in male and female CMT1A rats treated independently with Onapristone (Meyer zu Horste et al, 2007;Sereda et al, 2003), and no gender effect was observed in CMT1A patients after therapy with PXT3003 (Attarian et al, 2014) (Clinical Trials Register/Results, 2019.…”
Section: Discussionmentioning
confidence: 99%
“…At the present time, the most advanced strategy is a pharmacological treatment that reached the clinical phase III (NCT02579759). Another pharmacological treatment is reaching the clinical phase I (NCT03610334) and several others are in preclinical phases [12,16,34,37,51,58,66].…”
Section: Discussionmentioning
confidence: 99%
“…Probably best studied is its central role in NLRP3 (NOD-like receptor family, pyrin domain containing 3) inflammasome activation, cytokine maturation, and inflammation that was originally established in P2X7 knockout ( P2rx7 -/- ) mouse models ( Chessell et al, 2005 ; Solle et al, 2001 ; Di Virgilio et al, 2017 ). A growing body of evidence suggests that an increased P2X7 receptor function plays a role in various diseases of the central nervous system (CNS) and peripheral nervous system (PNS) and further supports its importance as a drug target ( Bhattacharya and Biber, 2016 ; Rassendren and Audinat, 2016 ; Sperlágh and Illes, 2014 ; Sociali et al, 2016 ).…”
Section: Introductionmentioning
confidence: 97%