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Large-scale phenotyping of an accurate genetic mouse model of JNCL identifies novel early pathology outside the central nervous system.
Staropoli JF, Haliw L, Biswas S, Garrett L, Hölter SM, Becker L, Skosyrski S, Da Silva-Buttkus P, Calzada-Wack J, Neff F, Rathkolb B, Rozman J, Schrewe A, Adler T, Puk O, Sun M, Favor J, Racz I, Bekeredjian R, Busch DH, Graw J, Klingenspor M, Klopstock T, Wolf E, Wurst W, Zimmer A, Lopez E, Harati H, Hill E, Krause DS, Guide J, Dragileva E, Gale E, Wheeler VC, Boustany RM, Brown DE, Breton S, Ruether K, Gailus-Durner V, Fuchs H, de Angelis MH, Cotman SL. Staropoli JF, et al. Among authors: harati h. PLoS One. 2012;7(6):e38310. doi: 10.1371/journal.pone.0038310. Epub 2012 Jun 6. PLoS One. 2012. PMID: 22701626 Free PMC article.
Exogenous Galactosylceramide as Potential Treatment for CLN3 Disease.
El-Sitt S, Soueid J, Maalouf K, Makhoul N, Al Ali J, Makoukji J, Asser B, Daou D, Harati H, Boustany RM. El-Sitt S, et al. Among authors: harati h. Ann Neurol. 2019 Nov;86(5):729-742. doi: 10.1002/ana.25573. Epub 2019 Aug 28. Ann Neurol. 2019. PMID: 31393621
73 results