Rescue of sarcoglycan mutations by inhibition of endoplasmic reticulum quality control is associated with minimal structural modifications.
Soheili T, Gicquel E, Poupiot J, N'Guyen L, Le Roy F, Bartoli M, Richard I.
Hum Mutat. 2012 Feb;33(2):429-39. doi: 10.1002/humu.21659. Epub 2011 Dec 22.
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D'Angelo MG, Gandossini S, Martinelli Boneschi F, Sciorati C, Bonato S, Brighina E, Comi GP, Turconi AC, Magri F, Stefanoni G, Brunelli S, Bresolin N,Cattaneo D, Clementi E.
E. Medea Scientific Institute, Bosisio Parini, Italy. Pharmacol Res. 2012 Apr;65(4):472-9. Epub 2012 Jan 25.
A dual acting compound releasing nitric oxide (NO) and ibuprofen, NCX 320, shows significant therapeutic effects in a mouse model of muscular dystrophy.
Sciorati C, Miglietta D, Buono R, Pisa V, Cattaneo D, Azzoni E, Brunelli S, Clementi E.
Pharmacol Res. 2011 Sep;64(3):210-7. Epub 2011 May 12.
Co-administration of ibuprofen and nitric oxide is an effective experimental therapy for muscular dystrophy, with immediate applicability to humans.
Sciorati C, Buono R, Azzoni E, Casati S, Ciuffreda P, D'Angelo G, Cattaneo D, Brunelli S, Clementi E.
Br J Pharmacol. 2010 Jul;160(6):1550-60.
Mannosidase I inhibition rescues the human alpha-sarcoglycan R77C recurrent mutation.
Bartoli M, Gicquel E, Barrault L, Soheili T, Malissen M, Malissen B, Vincent-Lacaze N, Perez N, Udd B, Danos O, Richard I.
Hum Mol Genet. 2008 May 1;17(9):1214-21. doi: 10.1093/hmg/ddn029. Epub 2008 Feb 5.
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