[18]在亨廷顿氏病小鼠模型中,用鞘氨醇-1-磷酸裂解酶(SGPL1)抑制剂THI治疗可调节鞘氨醇脂糖代谢,并取得有效的治疗效果

G. Pepe, L. Capocci, F. Marracino, N. Realini, P. Scarselli, Clotilde Di Cicco, A. Armirotti, R. Parlato, A. D. Pardo, V. Maglione
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I18 Treatment with THI, an inhibitor of sphingosine-1-phosphate lyase (SGPL1), modulates glycosphingolipid metabolism and results therapeutically effective in a mouse model of Huntington’s disease
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I01 Msh3-targeting antisense oligonucleotides halt CAG repeat expansions in Huntington’s disease IPSC-derived neurons I12 The novel alpha-2 adrenoceptor inhibitor beditin reduces cytotoxicity and huntingtin aggregates in cell models of Huntington’s disease I05 CRISPR-Cas9 nickase-mediated gene editing to treat Huntington’s disease I08 Passive immunization, with the anti-huntingtin aggregate antibody EM48, is beneficial in R6/2 mouse model I17 Sustained benefits of brain-permeable cholesterol-loaded nanoparticles in zQ175DN mouse model
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